Trial Outcomes & Findings for The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS) (NCT NCT01566695)

NCT ID: NCT01566695

Last Updated: 2025-01-07

Results Overview

RBC transfusion (tfx) independence was defined as the absence of any RBC transfusion during any consecutive "rolling" 56 days within the treatment period. Participants who did not receive any RBC transfusion during a consecutive rolling 56 days (i.e., day 1 to day 56, day 2 to day 57) were considered as a 56-day RBC transfusion independent responder.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 216 participants
• Primary outcome timeframe: Each participant was assessed for at least 56 days or more; from the date of randomization of study drug up to the data cut-off date of 25 January 2019, approximately 5 months.
• Results posted on: 2025-01-07

Participant Flow

Participants were randomized at 101 sites globally. The sites were located in: Europe (76), North America (13), Asia/Pacific (10), and Latin America (2). Results are reported as of the data cut-off date of 25 January 2019.

Participants were stratified by: average baseline (BL) Red Blood Cell (RBC) transfusion requirement (≤ 4 units versus > 4 units of RBC per 28 days), BL platelet transfusion status (dependent or independent), country of enrollment and Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) (0 to 1 versus 2).

Participant milestones

Measure	Oral Azacitidine Plus Best Supportive Care Participants received 300 mg oral azacitidine tablets daily (QD) on days 1 to 21 of each 28-day treatment cycle and best supportive care (BSC) which included and was not limited to packed RBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Overall Study STARTED	107	109
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	107	109

Reasons for withdrawal

Measure	Oral Azacitidine Plus Best Supportive Care Participants received 300 mg oral azacitidine tablets daily (QD) on days 1 to 21 of each 28-day treatment cycle and best supportive care (BSC) which included and was not limited to packed RBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Overall Study Lack of Efficacy	1	0
Overall Study Adverse Event	3	0
Overall Study Death	79	86
Overall Study Withdrawal by Subject	13	12
Overall Study Lost to Follow-up	0	3
Overall Study Other reasons	11	8

Baseline Characteristics

The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS)

Baseline characteristics by cohort

Measure	Oral Azacitidine Plus Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily (QD) on days 1 to 21 of each 28-day treatment cycle and best supportive care (BSC) which included and was not limited to packed RBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.	Total n=216 Participants Total of all reporting groups
Race/Ethnicity, Customized American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	73.0 Years STANDARD_DEVIATION 9.23 • n=5 Participants	73.1 Years STANDARD_DEVIATION 8.36 • n=7 Participants	73.0 Years STANDARD_DEVIATION 8.78 • n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants	30 Participants n=7 Participants	58 Participants n=5 Participants
Sex: Female, Male Male	79 Participants n=5 Participants	79 Participants n=7 Participants	158 Participants n=5 Participants
Race/Ethnicity, Customized White	96 Participants n=5 Participants	99 Participants n=7 Participants	195 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Asian	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islanders	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Japanese	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Other	8 Participants n=5 Participants	7 Participants n=7 Participants	15 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	4 Participants n=5 Participants	9 Participants n=7 Participants	13 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	91 Participants n=5 Participants	93 Participants n=7 Participants	184 Participants n=5 Participants
Race/Ethnicity, Customized Not Reported	12 Participants n=5 Participants	7 Participants n=7 Participants	19 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification RA = Refractory Anemia	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification RN = Refractory Neutropenia	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification RT = Refractory Thrombocytopenia	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification RARS = RA with Ringed Sideroblasts	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification RCMD = R Cytopenia w/ Multilineage Dysplasia	80 Participants n=5 Participants	73 Participants n=7 Participants	153 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification RAEB-1 RA with Excess Blasts - 1	17 Participants n=5 Participants	29 Participants n=7 Participants	46 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification RAEB-2 RA with Excess Blasts - 2	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification MDS-U (MDS-unclassified)	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Myelodysplastic Syndrome (MDS) World Health Organization (WHO) 2008 Classification del (5q) MDS Associated with Isolated del 5q	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
International Prognostic Scoring System (IPSS) Low	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
International Prognostic Scoring System (IPSS) Intermediate 1 (0.5-1.0)	106 Participants n=5 Participants	109 Participants n=7 Participants	215 Participants n=5 Participants
International Prognostic Scoring System (IPSS) Intermediate 2 (1.5-2.0)	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
International Prognostic Scoring System (IPSS) High	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Average Red Blood Cell Transfusion Requirement (units per 28 days)	3.33 units per 28 days n=5 Participants	3.33 units per 28 days n=7 Participants	3.33 units per 28 days n=5 Participants
Hemoglobin	8.22 g/dL STANDARD_DEVIATION 0.988 • n=5 Participants	8.04 g/dL STANDARD_DEVIATION 0.960 • n=7 Participants	8.13 g/dL STANDARD_DEVIATION 0.976 • n=5 Participants
Platelet Count	27.0 10^9 cells/L STANDARD_DEVIATION 15.97 • n=5 Participants	27.9 10^9 cells/L STANDARD_DEVIATION 18.11 • n=7 Participants	27.5 10^9 cells/L STANDARD_DEVIATION 17.05 • n=5 Participants
Platelet Transfusion Status Dependent	30 Participants n=5 Participants	35 Participants n=7 Participants	65 Participants n=5 Participants
Platelet Transfusion Status Independent	77 Participants n=5 Participants	74 Participants n=7 Participants	151 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 0-1	91 Participants n=5 Participants	94 Participants n=7 Participants	185 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 2	16 Participants n=5 Participants	15 Participants n=7 Participants	31 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 3	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 4	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 5	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Each participant was assessed for at least 56 days or more; from the date of randomization of study drug up to the data cut-off date of 25 January 2019, approximately 5 months.

Population: The intent-to-treat (ITT) population included all participants who were randomized, regardless of whether they received treatment or not.

RBC transfusion (tfx) independence was defined as the absence of any RBC transfusion during any consecutive "rolling" 56 days within the treatment period. Participants who did not receive any RBC transfusion during a consecutive rolling 56 days (i.e., day 1 to day 56, day 2 to day 57) were considered as a 56-day RBC transfusion independent responder.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days	30.8 Percentage of Participants Interval 22.1 to 39.6	11.9 Percentage of Participants Interval 5.8 to 18.0

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the data cut-off date of 25 January 2019

Population: Intent to Treat population. Participants who achieved RBC transfusion independence of ≥ 56 days on treatment.

Duration of RBC transfusion independence was analyzed only for participants who achieved RBC transfusion independence of ≥ 56 days on treatment. Duration of RBC transfusion independence was defined as the time from the date transfusion independence is first observed (day 1 of a ≥ 56 days period without a transfusion) until the date the participants had a subsequently documented RBC transfusion. In the event a participant had more than one ≥56 days rolling periods which met the RBC independence criteria, the duration with the longest rolling period was used in the analysis. Participants who maintained RBC TI through the end of the treatment period were censored at the date of treatment discontinuation, death, or 1 day before the start of the subsequent MDS treatment (if any), whichever occurred first, or the particiapnts latest available assessment date in the database if the treatment was still on-going.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=33 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=13 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 56 Days	11.1 months Interval 8.2 to 26.0	12.0 months Interval 2.3 to Could not be estimated due to the low number of events at the time of the analysis.

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: Responders in the intent to treat population who achieved a 56-day TI response

Time to RBC transfusion independence of ≥ 56 days was defined as the time between randomization and the date onset of transfusion independence was first observed (ie, Day 1 of 56 without any RBC transfusions).

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=33 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=13 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Time to RBC Transfusion Independence for at Least 56 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 56 Days	2.37 Months Interval 0.0 to 10.9	2.04 Months Interval 0.0 to 14.3

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: Intent to Treat population; includes participants who achieved RBC transfusion reduction of at least 4 units for at least 8 weeks.

A participant was considered as a RBC transfusion reduction responder if the participant had at least 4 units reduction in transfusion units over any consecutive 56 days period compared to the baseline transfusion units in 56 days.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=45 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=34 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Duration of RBC Transfusion Reduction for Participants Who Achieved RBC Transfusion Reduction of at Least 4 Units of RBCs for at Least 8 Weeks	10.0 months Interval 7.1 to 13.3	2.3 months Interval 2.0 to 5.0

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: The ITT population includes all participants who were randomized, regardless of whether they received treatment or not.

RBC transfusion independence was defined as the absence of any red blood cell (RBC) transfusion during any consecutive "rolling" 84 days within the treatment period. Participants who did not receive any RBC transfusion during a consecutive rolling 84 days (i.e., day 1 to day 84, day 2 to day 85) were considered as a 84-day RBC transfusion independent responder.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants Who Achieved RBC Transfusion Independence for ≥ 84 Days	28.0 Percentage of Participants Interval 19.5 to 36.5	6.4 Percentage of Participants Interval 1.8 to 11.0

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: Intent to Treat population. Participants who achieved RBC transfusion independence for at least 84 days on treatment.

Duration of RBC transfusion independence was analyzed only for participants who achieved RBC transfusion independence of ≥ 84 days on treatment. Duration of RBC transfusion independence was defined as the time from the date transfusion independence is first observed (day 1 of a ≥ 84 days period without a transfusion) until the date the participants had a subsequently documented RBC transfusion. In case a participant had more than one ≥84 days rolling periods which met the RBC independence criteria, the duration with the longest rolling period was used in the analysis.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=30 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=7 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 84 Days	11.1 months Interval 8.2 to 26.0	NA months Interval 5.0 to Could not be estimated due to the low number of events at the time of analysis.

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: Participants who achieved a 84-day TI response. Responders in the intent to treat population.

Time to RBC transfusion independence of ≥ 84 days was defined as the time between randomization and the date onset of transfusion independence was first observed (ie, Day 1 of 84 without any RBC transfusions).

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=30 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=7 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Time to RBC Transfusion Independence for at Least 84 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 84 Days	2.64 Months Interval 0.0 to 9.9	4.01 Months Interval 0.5 to 14.3

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: The ITT population included all participants who were randomized, regardless of whether they received treatment or not.

Erythroid HI-E improvement was defined as a hemoglobin increase of ≥ 1.5 g/dL; or a reduction in units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a hemoglobin of ≤ 9.0 g/dL on treatment were counted in the RBC transfusion response evaluation.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With an Erythroid Hematological Improvement (HI-E) Response According to 2006 IWG Criteria HI-E Response	43.0 Percentage of Participants Interval 33.6 to 52.4	32.1 Percentage of Participants Interval 23.3 to 40.9
Percentage of Participants With an Erythroid Hematological Improvement (HI-E) Response According to 2006 IWG Criteria ≥ 1.5 g/dL Hemoglobin Increase	23.4 Percentage of Participants Interval 15.3 to 31.4	5.5 Percentage of Participants Interval 1.2 to 9.8
Percentage of Participants With an Erythroid Hematological Improvement (HI-E) Response According to 2006 IWG Criteria RBC Transfusion Reduction	42.1 Percentage of Participants Interval 32.7 to 51.4	31.2 Percentage of Participants Interval 22.5 to 39.9

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: The ITT population included all participants who were randomized, regardless of whether they received treatment or not.

HI-P response was defined according to IWG 2006 criteria (Cheson, 2006) and as: 1. Absolute increase of ≥ 30 X 10^9/L for participants^ starting with > 20 X 10^9/L platelets; 2. Increase from < 20 X 10^9/L to > 20 X 10^9/L and by at least 100%. HI-P must have lasted at least 8 weeks.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Hematological Improvement Response in Platelets (HI-P) According to 2006 IWG Criteria	24.3 Percentage of Participants Interval 16.2 to 32.4	7.3 Percentage of Participants Interval 2.4 to 12.2

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: ITT population; includes participants who were baseline platelet transfusion dependent.

Platelet transfusion independence was defined as the absence of any platelet transfusion during any consecutive "rolling" 56 days during the treatment period, (ie, Day 1 to 56, Day 2 to 57, Days 3 to 58, etc.). Participants were considered platelet transfusion dependent at baseline if they had received ≥ 2 platelet transfusions during the 56 days immediately preceding randomization and had no consecutive 28-day period during which no platelet transfusions were administered.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=30 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=35 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants Who Achieved Platelet Transfusion Independence With a Duration of ≥ 8 Weeks (56 Days)	16.7 Percentage Participants	14.3 Percentage Participants

SECONDARY outcome

Timeframe: From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: Intent to Treat population; includes participants who achieved platelet transfusion independence for at least 56 days.

Time to platelet transfusion independence was defined as the time between randomization and the first documented date of onset of transfusion independence (ie, Day 1 of 56 without any platelet transfusions).

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=5 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=5 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Time to Platelet Transfusion Independence	9.6 Months Interval 9.6 to 10.9	NA Months Median, lower and upper range not calculated due to insufficient number of events.

SECONDARY outcome

Timeframe: From randomization up to death from any cause; up to a maximum of approximately 10 years on study; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: Intent to Treat population

Overall survival was defined as the time from randomization to death from any cause and was calculated using randomization date and date of death, or date of last follow-up for censored participants. All subjects were followed until drop out (withdrawal of consent from further data collection or lost to follow-up), death, or study closure. Participants who dropped out or were alive at study closure (or at the time of the interim analysis) had their OS times censored at the time of last contact, as appropriate.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Overall Survival (OS)	17.3 Months Interval 12.9 to 20.8	16.7 Months Interval 12.8 to 24.0

SECONDARY outcome

Timeframe: Response was assessed every 3 cycles; up to the data cut-off date of 25 Jan 2019; median duration of exposure to oral azacitidine was 86.0 days and 119.0 days for placebo

Population: Population includes participants with a CR, PR and mCR who had baseline bone marrow blasts \> 5%. ITT population.

Hematologic response was defined as: • A complete response (CR): <5% myeloblasts, and normal maturation of all cell lines; Peripheral blood (PB) shows: hemoglobin >10 g/dL, neutrophils ≥1.0x10^9/L, platelets ≥100x10^9/dL, blasts (0%) • Partial Response (PR): same as CR bone marrow (BM) shows blasts decreased by ≥ 50% over pre-treatment but still > 5%; Cellularity and morphology not relevant • Marrow CR: BM: ≤ 5% myeloblasts and decrease by ≥ 50% over pre-treatment PB • Stable disease (SD): failure to achieve at least PR, but no evidence of progression for > 8 wks • Failure: death during treatment or disease progression • Disease Progression for those with: - Less than 5% blasts: ≥ 50% increase in blasts to > 5% blasts - 5%-10% blasts:≥ 50% increase to > 10% blasts - 10%-20% blasts:≥ 50% increase to > 20% blasts - 20%-30% blasts ≥ 50% increase to > 30% blasts Any of the following: - ≥ 50% decrease from maximum remission/response in granulocytes or platelets

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS Complete Response (CR)	7.7 Percentage of Participants	0 Percentage of Participants
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS Partial Response	0 Percentage of Participants	0 Percentage of Participants
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS Marrow CR	23.1 Percentage of Participants	4.2 Percentage of Participants
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS Stable Disease (SD)	2.8 Percentage of Participants	30.3 Percentage of Participants
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS Disease Progression	62.6 Percentage of Participants	46.8 Percentage of Participants
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS Failure due to Death	0.9 Percentage of Participants	0.9 Percentage of Participants

SECONDARY outcome

Timeframe: From randomization of study drug to the end up to final data cut-off date of 25 January 2019; maximum follow-up time was 67.9 months for azacitidine and 64.8 months for placebo group

Population: The intent-to-treat (ITT) population included all participants who were randomized, regardless of whether they received treatment or not.

Participants with a documented diagnosis of AML arising from previous MDS documented diagnosis.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants Who Progressed to Acute Myeloid Leukemia (AML)	7.5 Percentage of Participants	16.5 Percentage of Participants

SECONDARY outcome

Timeframe: From randomization of study drug to progression of AML; up to a maximum of approximately 10 years on study; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: The intent-to-treat (ITT) population who progressed to AML.

Time to AML progression was defined as the time from the date of randomization until the date the subject has documented progression to AML. For participants who had progression to AML documented in MLL central lab report, the earliest sample collection date with the diagnosis of "s-AML arising from previous MDS" was used as the date to AML progression.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=8 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=18 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Time to Progression to Acute Myeloid Leukemia (AML) Among Participants Who Progressed to AML	NA Months The Time to AML was not evaluable due to insufficient number of events.	NA Months The Time to AML was not evaluable due to insufficient number of events.

SECONDARY outcome

Timeframe: From date of randomization until 28 days after the last dose of IP; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo

Population: The intent-to-treat (ITT) population included all participants who were randomized, regardless of whether they received treatment or not.

Clinically significant bleeding event was defined as: any intracranial or retroperitoneal bleed; bleeding requiring transfusions of > 2 units of blood/blood products; bleeding associated with a decrease in hemoglobin of > 2 g/dL; or bleeding from any site requiring transfusions of > 2 units of blood.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Significant Bleeding Events	8.4 Percentage of Participants	9.2 Percentage of Participants

SECONDARY outcome

Timeframe: From first dose of IP up to 28 days after the last dose of IP or until the last study visit; up to a maximum of approximately 6 months on study

Population: The safety population includes all randomized participants who received at least one dose of study drug.

A TEAE was defined as an adverse event that begins or worsens in intensity of frequency on or after the first dose of study drug through 28 days after last dose of study drug. A serious adverse event (SAE) is any: • Death; • Life-threatening event; • Any inpatient hospitalization or prolongation of existing hospitalization; • Persistent or significant disability or incapacity; • Congenital anomaly or birth defect; • Any other important medical event The investigator determined the relationship of an AE to study drug based on the timing of the AE relative to drug administration and whether or not other drugs, therapeutic interventions, or underlying conditions could provide a sufficient explanation for the event. The severity of an AE was evaluated by the investigator according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) (Version 4.0) where Grade 1 = Mild, Grade 2 = Moderate, Grade 3 = Severe, Grade 4 = Life-threatening and Grade 5 = Death.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE	107 Participants	108 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE Related to Study Drug	102 Participants	54 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 Serious TEAE	83 Participants	69 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 Serious TEAE Related to Study Drug	38 Participants	8 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 Grade (GR) 3-4 TEAE	98 Participants	81 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 Grade 3-4 TEAE Related to Study Drug	73 Participants	20 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 Grade (GR) 3-4 Serious TEAE	79 Participants	56 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 GR 3-4 Serious TEAE Related to Study Drug	38 Participants	5 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE Leading to Death	25 Participants	14 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE Related to Study Drug Leading to Death	9 Participants	2 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE Leading to Dose Reduction	31 Participants	4 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE Leading to Dose Interruption	68 Participants	40 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE Leading to Dose Interruption/Reduction	29 Participants	2 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) ≥ 1 TEAE Leading to Treatment Discontinuation	34 Participants	31 Participants

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: The Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being (PWG), social/family (SWB), emotional well being (EWB) and Functional Well-Being (FWB) and an additional 20-item anemia questionnaire that measures fatigue associated items and 7 non-fatigue items. The scales are formatted on 1 to 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general health related quality of life (HRQoL), the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various therapeutic areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 indicates the poorest QOL and 100 denotes the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Physical Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia (FACT-An) Endpoints at Cycle 6	0.2 Units on a Scale Standard Deviation 4.12	-0.8 Units on a Scale Standard Deviation 3.91

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: The Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being (PWG), social/family (SWB), emotional well being (EWB) and Functional Well-Being (FWB) and an additional 20-item anemia questionnaire that measures fatigue associated items and 7 non-fatigue items. The scales are formatted on 1 to 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general health related quality of life (HRQoL), the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various therapeutic areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 indicates the poorest QOL and 100 denotes the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Social Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia Instrument at Cycle 6	-0.4 Units on a Scale Standard Deviation 3.96	-1.1 Units on a Scale Standard Deviation 4.69

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: The Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being (PWG), social/family (SWB), emotional well being (EWB) and Functional Well-Being (FWB) and an additional 20-item anemia questionnaire that measures fatigue associated items and 7 non-fatigue items. The scales are formatted on 1 to 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general health related quality of life (HRQoL), the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various therapeutic areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 indicates the poorest QOL and 100 denotes the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Emotional Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia Instrument at Cycle 6	1.3 Units on a Scale Standard Deviation 4.33	0.2 Units on a Scale Standard Deviation 4.35

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: The FACT-Anemia evaluable population was defined as all ITT participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being (PWG), social/family (SWB), emotional well being (EWB) and Functional Well-Being (FWB) and an additional 20-item anemia questionnaire that measures fatigue associated items and 7 non-fatigue items. The scales are formatted on 1 to 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general health related quality of life (HRQoL), the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various therapeutic areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 indicates the poorest QOL and 100 denotes the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Functional Well-Being Component of the FACT-An Instrument at Cycle 6	0.5 Units on a Scale Standard Deviation 3.95	-1.2 Units on a Scale Standard Deviation 4.45

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being (PWG), social/family (SWB), emotional well being (EWB) and Functional Well-Being (FWB) and an additional 20-item anemia questionnaire that measures fatigue associated items and 7 non-fatigue items. The scales are formatted on 1 to 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general health related quality of life (HRQoL), the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various therapeutic areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 indicates the poorest QOL and 100 denotes the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Anemia Subscale Within FACT-An Instrument at Cycle 6	2.9 Units on a Scale Standard Deviation 11.81	-0.6 Units on a Scale Standard Deviation 10.39

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being (PWG), social/family (SWB), emotional well being (EWB) and Functional Well-Being (FWB) and an additional 20-item anemia questionnaire that measures fatigue associated items and 7 non-fatigue items. The scales are formatted on 1 to 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general health related quality of life (HRQoL), the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various therapeutic areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 indicates the poorest QOL and 100 denotes the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Fatigue-Related Subscale Within the FACT-An Instrument at Cycle 6	2.1 Units on a Scale Standard Deviation 8.74	-0.6 Units on a Scale Standard Deviation 7.84

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-G and FACT-An score are summed to form the FACT-An total score. The FACT-An Trial Outcome Index (TOI) consists of the summation of a "summary scale" and includes the Physical Well-being, (PWB; 7 items; score range, 0-28), the Functional Well-being (7 items; score range, 0-28) and the Anemia subscale consisting of 20 items on the same five-point scale, with 13 of them measuring fatigue related symptoms (FS) and seven measuring non-FS. The FACT-An TOI has been demonstrated to be a sensitive indicator of clinical outcomes in a number of diseases including MDS. The Fact-TOI score ranges from 0 to 136. Higher scores on all scales of the Fact-An and subscales on the FACT-TOI reflect better quality of life or fewer symptoms.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia Trial Outcome Index (FACT-An TOI) Summary Scale Within the FACT-An Instrument at Cycle 6	3.7 Units on a Scale Standard Deviation 17.29	-2.7 Units on a Scale Standard Deviation 15.45

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-An is a 47-item, cancer-specific questionnaire consisting of a core 27-item general questionnaire (i.e., the Functional Assessment of Cancer Therapy-General [FACT-G]) The FACT-G measures the 4 domains on a 5-point scale ranging from 0 (not at all) to 4 (very much). The 4 domains are: • Physical Well-being (PWB; 7 items; score range, 0-28), • Social/Family Well-being (SWB; 7 items; score range, 0-28), • Emotional Well-being (EWB; 6 items; score range, 0-24), and • Functional Well-being (7 items; score range, 0-28). The FACT-G is a summation composed of a "summary scale" including the PWB, SWB, EWB and FWB. The FACT-G score range is from 0 to 108. For all summary scales including FACT-G, a higher score indicates better HRQoL or lower level of symptoms.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia-General (FACT-G) Summary Scale Within the FACT-An Instrument at Cycle 6	1.6 Units on a Scale Standard Deviation 12.00	-2.9 Units on a Scale Standard Deviation 12.11

SECONDARY outcome

Timeframe: Baseline to Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

The FACT-G and the anemia subscale (AnS) are summed to form the FACT-An total score and the total score ranges from 0 to 188. The FACT-G measures the 4 domains on a 5-point scale ranging from 0 (not at all) to 4 (very much). The 4 domains are: • Physical Well-being (PWB; 7 items; score range, 0-28), • Social/Family Well-being (SWB; 7 items; score range, 0-28), • Emotional Well-being (EWB; 6 items; score range, 0-24), and • Functional Well-being (7 items; score range, 0-28). The AnS consists of 20 items on the same 5-point scale, with 13 of them measuring fatigue-related symptoms (FS) and 7 measuring non-FS. The AnS and FS scores can range from 0-80 and 0-52, respectively. For all domains and summary subscales, a higher score indicates better HRQoL or lower level of symptoms.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=42 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=49 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia-Total Score at Cycle 6	4.5 Units on a Scale Standard Deviation 21.88	-3.5 Units on a Scale Standard Deviation 20.62

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Physical Well-Being Domain Within the FACT-An Instrument at Cycle 6	17.3 Percentage of Participants	13.7 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Social Well-Being Domain Within the FACT-An Instrument at Cycle 6	11.1 Percentage of Participants	14.7 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Emotional Well-Being Domain Within the FACT-An Instrument at Cycle 6	23.5 Percentage of Participants	15.8 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Functional Well-Being Domain Within the FACT-An Instrument at Cycle 6	14.8 Percentage of Participants	8.4 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Anemia Subscale Domain Within the FACT-An Instrument at Cycle 6	27.2 Percentage of Participants	15.8 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Fatigue Related Symptoms Subscale Domain Within the FACT-An Instrument at Cycle 6	27.2 Percentage of Participants	18.9 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Functional Assessment of Cancer Therapy-Anemia Trial Outcome Index Subscale Domain Within the FACT-An Instrument at Cycle 6	19.8 Percentage of Participants	12.6 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Functional Assessment of Cancer Therapy-Anemia-General Subscale Domain Within the FACT-An Instrument at Cycle 6	23.5 Percentage of Participants	13.7 Percentage of Participants

SECONDARY outcome

Timeframe: Cycle 6 Day 1

Population: Health Related Quality of Life (HR-QoL) evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score at C1D1 and at least one post-baseline assessment visit.

A clinically meaningful improvement or deterioration was defined by domain specific thresholds of change from baseline. The FACT-An questionnaire is a 47-item, cancer specific questionnaire consisting of a core 27 items measuring 4 general domains physical well being, social/family, emotional well being and Functional Well-Being and an additional 20-item anemia questionnaire that measures fatigue and 7 non-fatigue items. The scales are formatted on 4 pages for self-administration using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a Bit and 4 = Very much). Also, general HRQoL measures the impact of fatigue and other anemia-related symptoms on patient functioning and is used to assess the effect of treatments in various areas, including MDS. The instrument and the fatigue and non-fatigue subscales are scored by summing points from all questions, then converting this sum to a 100 point scale; 0 = the poorest QOL and 100 = the highest QOL.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With a Clinically Meaningful Improvement (CMI) From Baseline in the Functional Assessment of Cancer Therapy Anemia-Total Score Domain Within the FACT-An Instrument at Cycle 6	19.8 Percentage of Participants	11.6 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 2 Day 1 (C2D1)

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved [i.e., change score from 1 to 4], no change [0], worsened by one level [-1], worsened by ≥2 levels [-2 to -4], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 2 Day 1 (C2D1) Improved	2.5 Percentage of Participants	10.5 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 2 Day 1 (C2D1) No Change	30.9 Percentage of Participants	49.5 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 2 Day 1 (C2D1) Worsened by 1 Level	25.9 Percentage of Participants	23.2 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 2 Day 1 (C2D1) Worsened by 2 Levels	23.5 Percentage of Participants	6.3 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 2 Day 1 (C2D1) Missing	17.3 Percentage of Participants	10.5 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 3 Day 1 (C3D1)

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved [i.e., change score from 1 to 4], no change [0], worsened by one level [-1], worsened by ≥2 levels [-2 to -4], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 3 Day 1 (C3D1) Improved	7.4 Percentage of Participants	10.5 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 3 Day 1 (C3D1) No Change	24.7 Percentage of Participants	41.1 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 3 Day 1 (C3D1) Worsened by 1 Level	16.0 Percentage of Participants	18.9 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 3 Day 1 (C3D1) Worsened by 2 Levels	23.5 Percentage of Participants	13.7 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 3 Day 1 (C3D1) Missing	28.4 Percentage of Participants	15.8 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 4 Day 1 (C4D1)

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved [i.e., change score from 1 to 4], no change [0], worsened by one level [-1], worsened by ≥2 levels [-2 to -4], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 4 Day 1 (C4D1) Improved	2.5 Percentage of Participants	9.5 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 4 Day 1 (C4D1) No Change	32.1 Percentage of Participants	37.9 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 4 Day 1 (C4D1) Worsened by 1 Level	16.0 Percentage of Participants	14.7 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 4 Day 1 (C4D1) Worsened by 2 Levels	14.8 Percentage of Participants	6.3 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 4 Day 1 (C4D1) Missing	34.6 Percentage of Participants	31.6 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 5 Day 1 (C5D1)

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved [i.e., change score from 1 to 4], no change [0], worsened by one level [-1], worsened by ≥2 levels [-2 to -4], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 5 Day 1 (C5D1) Improved	2.5 Percentage of Participants	7.4 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 5 Day 1 (C5D1) No Change	25.9 Percentage of Participants	34.7 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 5 Day 1 (C5D1) Worsened by 1 Level	13.6 Percentage of Participants	12.6 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 5 Day 1 (C5D1) Worsened by 2 Levels	8.6 Percentage of Participants	5.3 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 5 Day 1 (C5D1) Missing	49.4 Percentage of Participants	40.0 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 6 Day 1 (C6 D1)

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved [i.e., change score from 1 to 4], no change [0], worsened by one level [-1], worsened by ≥2 levels [-2 to -4], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 6 Day 1 (C6 D1) Improved	1.2 Percentage of Participants	4.2 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 6 Day 1 (C6 D1) No Change	25.9 Percentage of Participants	27.4 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 6 Day 1 (C6 D1) Worsened by 1 Level	9.9 Percentage of Participants	12.6 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 6 Day 1 (C6 D1) Worsened by 2 Levels	14.8 Percentage of Participants	7.4 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 6 Day 1 (C6 D1) Missing	48.1 Percentage of Participants	48.4 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 7 Day 1 (C7D1)

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved [i.e., change score from 1 to 4], no change [0], worsened by one level [-1], worsened by ≥2 levels [-2 to -4], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 7 Day 1 (C7D1) Improved	1.2 Percentage of Participants	1.1 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 7 Day 1 (C7D1) No Change	25.9 Percentage of Participants	21.1 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 7 Day 1 (C7D1) Worsened by 1 Level	11.1 Percentage of Participants	3.2 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 7 Day 1 (C7D1) Worsened by 2 Levels	7.4 Percentage of Participants	3.2 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - Cycle 7 Day 1 (C7D1) Missing	54.3 Percentage of Participants	71.6 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to End of Treatment

Population: The HRQoL evaluable population was defined as participants with a non-missing FACT-An Trial Outcome Index (TOI) score baseline visit and at least one post-baseline assessment visit.

The distribution (frequency and percentage) of the observed responses (i.e., "Not at all (0)," "A little bit (1)," "Somewhat (2)," "Quite a bit (3)," "Very much (4)," and missing) to Item GP-5 ("I am bothered by side effects of treatment" in the past seven days) of the FACT-An at each scheduled visit were summarized for each treatment group. The denominator for the percentage calculation per treatment group was based on the number of the FACT-An evaluable population at baseline. The distribution of change in responses (improved [i.e., change score from 1 to 4], no change [0], worsened by one level [-1], worsened by ≥2 levels [-2 to -4], and missing) from baseline at each post-baseline scheduled visit were summarized by treatment group.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - End of Treatment Improved	2.5 Percentage of Participants	6.3 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - End of Treatment No Change	14.8 Percentage of Participants	25.3 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - End of Treatment Worsened by 1 Level	9.9 Percentage of Participants	8.4 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - End of Treatment Worsened by 2 Levels	9.9 Percentage of Participants	12.6 Percentage of Participants
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5 - End of Treatment Missing	63.0 Percentage of Participants	47.4 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 6 Day 1

Population: The HRQoL evaluable population was defined as participants with a EQ-5D-3L health utility at baseline and at least one post-baseline assessment visit.

The EQ-5D-3L is a generic, self-administered questionnaire that consists of 5 dimensions: mobility, self-care, pain, usual activities, and anxiety/depression. Each dimension has 3 levels of severity corresponding to no problems, some problems, and extreme problems. It also includes a Visual Analog Scale that recorded the respondent's self-rated health on a vertical, 0-100 scale, where 100 = Best imaginable health state and 0 = Worst imaginable health state. Distribution of the observed responses (i.e., no problems, moderate problems, severe problems, and missing) of the 5 dimensions at each visit was summarized per arm. The denominator for the percentage calculation per group was based on the number of the EQ-5D-3L evaluable population at baseline. The distribution of change in responses (i.e., improved [by ≥1 level], no change, worsened [by ≥1 level], and missing) from baseline are reported.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level (EQ-5D-3L) Mobility Dimension Responses at Cycle 6 Improved	8.6 Percentage of Participants	8.4 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level (EQ-5D-3L) Mobility Dimension Responses at Cycle 6 No Change	35.8 Percentage of Participants	33.7 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level (EQ-5D-3L) Mobility Dimension Responses at Cycle 6 Worsened	7.4 Percentage of Participants	9.5 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level (EQ-5D-3L) Mobility Dimension Responses at Cycle 6 Missing	48.1 Percentage of Participants	48.4 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 6 Day 1

Population: The HRQoL evaluable population was defined as participants with a EQ-5D-3L health utility at baseline and at least one post-baseline assessment visit.

The EQ-5D-3L is a generic, self-administered questionnaire that consists of 5 dimensions: mobility, self-care, pain, usual activities, and anxiety/depression. Each dimension has 3 levels of severity corresponding to no problems, some problems, and extreme problems. It also includes a Visual Analog Scale that recorded the respondent's self-rated health on a vertical, 0-100 scale, where 100 = Best imaginable health state and 0 = Worst imaginable health state. Distribution of the observed responses (i.e., no problems, moderate problems, severe problems, and missing) of the 5 dimensions at each visit was summarized per arm. The denominator for the percentage calculation per group was based on the number of the EQ-5D-3L evaluable population at baseline. The distribution of change in responses (i.e., improved [by ≥1 level], no change, worsened [by ≥1 level], and missing) from baseline are reported.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level of Self-Care Dimension Responses at Cycle 6 Improved	2.5 Percentage of Participants	4.2 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level of Self-Care Dimension Responses at Cycle 6 No Change	42.0 Percentage of Participants	44.2 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level of Self-Care Dimension Responses at Cycle 6 Worsened	7.4 Percentage of Participants	3.2 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level of Self-Care Dimension Responses at Cycle 6 Missing	48.1 Percentage of Participants	48.4 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 6 Day 1

Population: The HRQoL evaluable population was defined as participants with a EQ-5D-3L health utility at baseline and at least one post-baseline assessment visit.

TThe EQ-5D-3L is a generic, self-administered questionnaire that consists of 5 dimensions: mobility, self-care, pain, usual activities, and anxiety/depression. Each dimension has 3 levels of severity corresponding to no problems, some problems, and extreme problems. It also includes a Visual Analog Scale that recorded the respondent's self-rated health on a vertical, 0-100 scale, where 100 = Best imaginable health state and 0 = Worst imaginable health state. Distribution of the observed responses (i.e., no problems, moderate problems, severe problems, and missing) of the 5 dimensions at each visit was summarized per arm. The denominator for the percentage calculation per group was based on the number of the EQ-5D-3L evaluable population at baseline. The distribution of change in responses (i.e., improved [by ≥1 level], no change, worsened [by ≥1 level], and missing) from baseline are reported.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level Usual Activities Dimension Responses at Cycle 6 Improved	11.1 Percentage of Participants	3.2 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level Usual Activities Dimension Responses at Cycle 6 No Change	28.4 Percentage of Participants	41.1 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level Usual Activities Dimension Responses at Cycle 6 Worsened	12.3 Percentage of Participants	7.4 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level Usual Activities Dimension Responses at Cycle 6 Missing	48.1 Percentage of Participants	48.4 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 6 Day 1

Population: The HRQoL evaluable population was defined as participants with a EQ-5D-3L health utility at baseline and at least one post-baseline assessment visit.

The EQ-5D-3L is a generic, self-administered questionnaire that consists of 5 dimensions: mobility, self-care, pain, usual activities, and anxiety/depression. Each dimension has 3 levels of severity corresponding to no problems, some problems, and extreme problems. It also includes a Visual Analog Scale that recorded the respondent's self-rated health on a vertical, 0-100 scale, where 100 = Best imaginable health state and 0 = Worst imaginable health state. Distribution of the observed responses (i.e., no problems, moderate problems, severe problems, and missing) of the 5 dimensions at each visit was summarized per arm. The denominator for the percentage calculation per group was based on the number of the EQ-5D-3L evaluable population at baseline. The distribution of change in responses (i.e., improved [by ≥1 level], no change, worsened [by ≥1 level], and missing) from baseline are reported.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Pain/Discomfort Dimension Responses at Cycle 6 Improved	13.6 Percentage of Participants	8.4 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Pain/Discomfort Dimension Responses at Cycle 6 No Change	33.3 Percentage of Participants	32.6 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Pain/Discomfort Dimension Responses at Cycle 6 Worsened	4.9 Percentage of Participants	10.5 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Pain/Discomfort Dimension Responses at Cycle 6 Missing	48.1 Percentage of Participants	48.4 Percentage of Participants

SECONDARY outcome

Timeframe: From Baseline to Cycle 6 Day 1

Population: The HRQoL evaluable population was defined as participants with a EQ-5D-3L health utility at baseline and at least one post-baseline assessment visit.

The EQ-5D-3L is a generic, self-administered questionnaire that consists of 5 dimensions: mobility, self-care, pain, usual activities, and anxiety/depression. Each dimension has 3 levels of severity corresponding to no problems, some problems, and extreme problems. It also includes a Visual Analog Scale that recorded the respondent's self-rated health on a vertical, 0-100 scale, where 100 = Best imaginable health state and 0 = Worst imaginable health state. Distribution of the observed responses (i.e., no problems, moderate problems, severe problems, and missing) of the 5 dimensions at each visit was summarized per arm. The denominator for the percentage calculation per group was based on the number of the EQ-5D-3L evaluable population at baseline. The distribution of change in responses (i.e., improved [by ≥1 level], no change, worsened [by ≥1 level], and missing) from baseline are reported.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=81 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=95 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Anxiety/Depression Dimension Responses at Cycle 6 Improved	4.9 Percentage of Participants	7.4 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Anxiety/Depression Dimension Responses at Cycle 6 No Change	35.8 Percentage of Participants	37.9 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Anxiety/Depression Dimension Responses at Cycle 6 Worsened	11.1 Percentage of Participants	6.3 Percentage of Participants
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Anxiety/Depression Dimension Responses at Cycle 6 Missing	48.1 Percentage of Participants	48.4 Percentage of Participants

SECONDARY outcome

Timeframe: From date of randomization up to 28 days after the last dose of study drug; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo

Population: The safety population includes all randomized participants who received at least one dose of study drug.

The number of reasons for hospitalizations and hospital admissions during the treatment period were monitored and include those associated with: AEs, protocol-driven procedures, transfusions, non-protocol procedures, elective procedures or those associated with social, practical or technical reasons in the absence of AEs. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized Adverse Events	79 Participants	65 Participants
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized Protocol Driven Procedures	2 Participants	7 Participants
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized Non-Protocol Driven Procedures	9 Participants	19 Participants
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized Transfusion	32 Participants	33 Participants
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized Procedure Planned Prior to Signing Consent	0 Participants	4 Participants
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized Elective Procedures	4 Participants	10 Participants
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized Social, Technical or Practical Reason except AEs	4 Participants	6 Participants

SECONDARY outcome

Timeframe: From date of randomization up to 28 days after the last dose of study drug; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo

Population: The safety population includes all randomized participants who received at least one dose of study drug.

The total number of days hospitalized due to any reason during the treatment period was monitored. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Healthcare Resource Utilization (HRU): Total Number of Days Hospitalized Due to Any Reason	3513 Days	2688 Days

SECONDARY outcome

Timeframe: From date of randomization up to 28 days after the last dose of study drug; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo

Population: The safety population includes all randomized participants who received at least one dose of study drug.

The number of days hospitalized per total patient years. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

Outcome measures

Measure	Oral Azacitidine and Best Supportive Care n=107 Participants Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 Participants Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Healthcare Resource Utilization (HRU): Total Number of Days Hospitalized Per Total Patient-Years	41.44 Days Per Total Patient Years	40.53 Days Per Total Patient Years

Adverse Events

Oral Azacitidine and Best Supportive Care

Serious events: 83 serious events

Other events: 107 other events

Deaths: 83 deaths

Placebo Plus Best Supportive Care

Serious events: 69 serious events

Other events: 104 other events

Deaths: 86 deaths

Serious adverse events

Measure	Oral Azacitidine and Best Supportive Care n=107 participants at risk Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 participants at risk Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Blood and lymphatic system disorders Anaemia	6.5% 7/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	4.6% 5/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Blood loss anaemia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Bone marrow failure	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Febrile neutropenia	27.1% 29/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	8.3% 9/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Haemolytic anaemia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Neutropenia	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Pancytopenia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Thrombocytopenia	4.7% 5/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Acute coronary syndrome	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Acute myocardial infarction	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Angina pectoris	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Angina unstable	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Atrial fibrillation	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Atrioventricular block complete	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Atrioventricular block first degree	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Cardiac arrest	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Cardiac failure	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Cardiac failure congestive	2.8% 3/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Cardio-respiratory arrest	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Cardiogenic shock	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Long QT syndrome	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Myocardial infarction	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Tachyarrhythmia	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Ventricular tachycardia	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Abdominal pain	2.8% 3/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Abdominal pain upper	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Colitis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Constipation	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Diarrhoea	2.8% 3/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Gastritis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Gastrointestinal haemorrhage	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Haemorrhoidal haemorrhage	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Intestinal obstruction	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Intestinal perforation	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Intra-abdominal haemorrhage	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Melaena	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Nausea	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Neutropenic colitis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Oesophageal achalasia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Oesophageal varices haemorrhage	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Oral mucosal blistering	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Rectal haemorrhage	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Upper gastrointestinal haemorrhage	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Vomiting	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Fatigue	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Gait disturbance	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders General physical health deterioration	2.8% 3/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Hypothermia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Multiple organ dysfunction syndrome	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Pyrexia	7.5% 8/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	3.7% 4/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Sudden death	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Hepatobiliary disorders Biliary colic	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Hepatobiliary disorders Cholecystitis	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Hepatobiliary disorders Hyperbilirubinaemia	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Immune system disorders Haemophagocytic lymphohistiocytosis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Abscess limb	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Arteriovenous fistula site infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Atypical pneumonia	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Bacteraemia	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Bronchitis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Bronchopulmonary aspergillosis	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations COVID-19 pneumonia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Cellulitis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Clostridium difficile colitis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Coronavirus infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Cystitis escherichia	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Device related infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Diverticulitis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Epididymitis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Escherichia sepsis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Febrile infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Gastroenteritis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Gastroenteritis clostridial	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Groin abscess	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Haemophilus infection	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Infection	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Influenza	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Klebsiella infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Klebsiella sepsis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Lower respiratory tract infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	3.7% 4/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Lymph gland infection	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Meningitis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Meningitis bacterial	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Myringitis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Neutropenic sepsis	4.7% 5/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pharyngeal abscess	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pharyngitis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pneumonia	12.1% 13/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	11.0% 12/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pneumonia aspiration	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pneumonia fungal	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pneumonia pneumococcal	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Prostatic abscess	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pseudomonal sepsis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Respiratory tract infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Respiratory tract infection bacterial	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Sepsis	7.5% 8/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Septic shock	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Skin infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Staphylococcal infection	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Staphylococcal sepsis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Tooth abscess	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Upper respiratory tract infection	3.7% 4/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Urinary tract infection	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Urinary tract infection bacterial	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Viral sepsis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Arteriovenous fistula site haemorrhage	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Cystitis radiation	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Fall	3.7% 4/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Febrile nonhaemolytic transfusion reaction	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Head injury	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Hip fracture	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Periorbital haematoma	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Subdural haematoma	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Subdural haemorrhage	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Transfusion reaction	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Upper limb fracture	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Investigations Blood urea increased	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Investigations Gamma-glutamyltransferase increased	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Investigations Weight decreased	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Dehydration	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Diabetes mellitus	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Diabetes mellitus inadequate control	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Diabetic metabolic decompensation	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Hyperkalaemia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Hypoglycaemia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Hyponatraemia	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Musculoskeletal and connective tissue disorders Back pain	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Musculoskeletal and connective tissue disorders Muscular weakness	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Musculoskeletal and connective tissue disorders Polychondritis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Acute myeloid leukaemia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Adenocarcinoma of colon	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal cell carcinoma	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Bone neoplasm	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Bowen's disease	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Carcinoma in situ of skin	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Central nervous system leukaemia	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Chronic myelomonocytic leukaemia	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colon cancer	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colorectal adenoma	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Diffuse large B-cell lymphoma	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lip squamous cell carcinoma	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Malignant neoplasm of unknown primary site	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Mantle cell lymphoma recurrent	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastases to liver	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Myelodysplastic syndrome	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Myelodysplastic syndrome with excess blasts	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Small intestine carcinoma	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Spinal cord neoplasm	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous cell carcinoma of skin	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Transformation to acute myeloid leukaemia	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	5.5% 6/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Central nervous system lesion	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Cerebral haemorrhage	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Cerebral ischaemia	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Epilepsy	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Generalised tonic-clonic seizure	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Guillain-Barre syndrome	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Haemorrhage intracranial	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders IIIrd nerve paresis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Lethargy	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Presyncope	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Sciatica	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Status epilepticus	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Syncope	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Transient ischaemic attack	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Psychiatric disorders Bipolar disorder	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Psychiatric disorders Confusional state	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Acute kidney injury	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Chronic kidney disease	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Haematuria	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Pollakiuria	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Prerenal failure	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Renal colic	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Renal failure	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Urinary retention	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Urinary tract obstruction	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Reproductive system and breast disorders Prostatitis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Epistaxis	1.9% 2/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Haemoptysis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Laryngeal oedema	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Pleurisy	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Pleuritic pain	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Pulmonary oedema	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Skin and subcutaneous tissue disorders Cutaneous vasculitis	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Skin and subcutaneous tissue disorders Rash	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Vascular disorders Arteritis	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Vascular disorders Haematoma	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Vascular disorders Polyarteritis nodosa	0.00% 0/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Vascular disorders Shock haemorrhagic	0.93% 1/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo

Other adverse events

Measure	Oral Azacitidine and Best Supportive Care n=107 participants at risk Participants received 300 mg oral azacitidine tablets daily on days 1 to 21 of each 28-day treatment cycle and BSC included and was not limited to pRBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.	Placebo Plus Best Supportive Care n=109 participants at risk Participants received identically matching placebo tablets QD on days 1 to 21 of each 28-day treatment cycle and BSC which included but was not limited to, pRBC and whole blood, platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and G-CSF for participants who experienced neutropenic fever/infections.
Blood and lymphatic system disorders Anaemia	21.5% 23/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	15.6% 17/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Leukopenia	9.3% 10/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Neutropenia	48.6% 52/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	14.7% 16/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Blood and lymphatic system disorders Thrombocytopenia	28.0% 30/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	16.5% 18/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Atrial fibrillation	6.5% 7/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Cardiac disorders Cardiac failure	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Abdominal pain	15.0% 16/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	12.8% 14/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Constipation	47.7% 51/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	22.0% 24/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Diarrhoea	68.2% 73/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	23.9% 26/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Gastrooesophageal reflux disease	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Gingival bleeding	6.5% 7/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	3.7% 4/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Haemorrhoids	3.7% 4/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	5.5% 6/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Mouth haemorrhage	9.3% 10/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	6.4% 7/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Nausea	75.7% 81/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	22.9% 25/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Rectal haemorrhage	2.8% 3/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	7.3% 8/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Gastrointestinal disorders Vomiting	62.6% 67/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	10.1% 11/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Asthenia	23.4% 25/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	18.3% 20/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Fatigue	23.4% 25/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	20.2% 22/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Oedema peripheral	28.0% 30/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	15.6% 17/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
General disorders Pyrexia	29.9% 32/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	13.8% 15/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Cellulitis	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Oral herpes	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Pneumonia	9.3% 10/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	3.7% 4/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Upper respiratory tract infection	6.5% 7/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	3.7% 4/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Infections and infestations Urinary tract infection	11.2% 12/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	4.6% 5/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Contusion	15.0% 16/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Injury, poisoning and procedural complications Fall	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Investigations Alanine aminotransferase increased	9.3% 10/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	5.5% 6/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Investigations Serum ferritin increased	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	4.6% 5/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Investigations Weight decreased	10.3% 11/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Decreased appetite	25.2% 27/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	9.2% 10/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Hyperglycaemia	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Hyperkalaemia	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.00% 0/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Hypokalaemia	11.2% 12/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	9.2% 10/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Hypomagnesaemia	10.3% 11/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	4.6% 5/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Metabolism and nutrition disorders Iron overload	6.5% 7/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	10.1% 11/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Musculoskeletal and connective tissue disorders Arthralgia	10.3% 11/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	11.0% 12/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Musculoskeletal and connective tissue disorders Back pain	14.0% 15/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	11.9% 13/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Musculoskeletal and connective tissue disorders Pain in extremity	7.5% 8/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	3.7% 4/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Dizziness	7.5% 8/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	8.3% 9/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Nervous system disorders Syncope	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Psychiatric disorders Anxiety	8.4% 9/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	3.7% 4/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Psychiatric disorders Confusional state	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	0.92% 1/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Psychiatric disorders Depression	6.5% 7/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	1.8% 2/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Psychiatric disorders Insomnia	10.3% 11/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	5.5% 6/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Renal and urinary disorders Haematuria	3.7% 4/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	5.5% 6/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Cough	15.0% 16/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	13.8% 15/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Dyspnoea	13.1% 14/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	13.8% 15/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Respiratory, thoracic and mediastinal disorders Epistaxis	26.2% 28/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	19.3% 21/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Skin and subcutaneous tissue disorders Ecchymosis	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	9.2% 10/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Skin and subcutaneous tissue disorders Petechiae	19.6% 21/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	18.3% 20/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Skin and subcutaneous tissue disorders Pruritus	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	7.3% 8/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Vascular disorders Haematoma	10.3% 11/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	10.1% 11/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Vascular disorders Hypertension	4.7% 5/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	6.4% 7/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Vascular disorders Hypotension	5.6% 6/107 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo	2.8% 3/109 • Participants were assessed for all-cause mortality from their randomization to study completion, (up to approximately 10 years). SAEs and Other AEs were assessed from first dose to 28 days following last dose (up to approximately 6 months) Median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo

Additional Information

Bristol-Myers Squibb Study Director

Bristol-Myers Squibb

Phone: Please Email

Email: Clinical.Trials@bms.com

Results disclosure agreements

• Principal investigator is a sponsor employee Results from a center cannot be submitted for publication before results of multicenter study are published unless it is \> 1 year since study completion. Then, Investigator can publish if manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides publication would hinder drug development, Investigator must delay submission for up to 90 additional days. Investigator must delete confidential information before submission and defer publication to permit patent applications.
• Publication restrictions are in place

Restriction type: OTHER