Trial Outcomes & Findings for Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission (NCT NCT01757535)

Last Updated: 2025-07-08

Results Overview

Overall survival was defined as time from randomization to death from any cause; participants surviving at the end of the follow-up period, or who withdraw consent, or who were lost to follow up were censored at the date last known alive.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

472 participants

Primary outcome timeframe

Day 1 (randomization) up to data cut off date of 15 July 2019; median follow-up for OS estimated by the reverse K-M method was 41.2 months for all participants.

Results posted on

2025-07-08

Participant Flow

472 Participants Randomized, 469 participants treated

Participant milestones

Participant milestones
Measure	Oral Azacitidine Plus Best Supportive Care Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Overall Study STARTED	238	234
Overall Study Received Treatment	236	233
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	238	234

Reasons for withdrawal

Reasons for withdrawal
Measure	Oral Azacitidine Plus Best Supportive Care Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Overall Study Disease Relapse	151	181
Overall Study Other Reasons	28	27
Overall Study Adverse Event	31	11
Overall Study Withdrew Consent	19	13
Overall Study Physician Decision	7	0
Overall Study Death	2	2

Baseline Characteristics

Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Oral Azacitidine Plus Best Supportive Care n=238 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=234 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.	Total n=472 Participants Total of all reporting groups
Age, Continuous	67.9 Years STANDARD_DEVIATION 5.72 • n=5 Participants	68.0 Years STANDARD_DEVIATION 5.62 • n=7 Participants	67.9 Years STANDARD_DEVIATION 5.66 • n=5 Participants
Age, Customized 18 to 64 Years	66 Participants n=5 Participants	68 Participants n=7 Participants	134 Participants n=5 Participants
Age, Customized 65 to 84 Years	171 Participants n=5 Participants	166 Participants n=7 Participants	337 Participants n=5 Participants
Age, Customized ≥ 85 years	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Female	120 Participants n=5 Participants	107 Participants n=7 Participants	227 Participants n=5 Participants
Sex: Female, Male Male	118 Participants n=5 Participants	127 Participants n=7 Participants	245 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	20 Participants n=5 Participants	14 Participants n=7 Participants	34 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	196 Participants n=5 Participants	202 Participants n=7 Participants	398 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	22 Participants n=5 Participants	18 Participants n=7 Participants	40 Participants n=5 Participants
Race/Ethnicity, Customized White	216 Participants n=5 Participants	197 Participants n=7 Participants	413 Participants n=5 Participants
Race/Ethnicity, Customized Black or African-American	2 Participants n=5 Participants	6 Participants n=7 Participants	8 Participants n=5 Participants
Race/Ethnicity, Customized Asian	6 Participants n=5 Participants	20 Participants n=7 Participants	26 Participants n=5 Participants
Race/Ethnicity, Customized Other	12 Participants n=5 Participants	11 Participants n=7 Participants	23 Participants n=5 Participants
Race/Ethnicity, Customized Missing	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Initial Acute Myeloid Leukemia (AML) Classification AML with Recurrent Genetic Abnormalities	39 Participants n=5 Participants	46 Participants n=7 Participants	85 Participants n=5 Participants
Initial Acute Myeloid Leukemia (AML) Classification AML with Myelodysplasia - Related Changes	49 Participants n=5 Participants	42 Participants n=7 Participants	91 Participants n=5 Participants
Initial Acute Myeloid Leukemia (AML) Classification Therapy-related Myeloid Neoplasms	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Initial Acute Myeloid Leukemia (AML) Classification AML not Otherwise Specified	148 Participants n=5 Participants	145 Participants n=7 Participants	293 Participants n=5 Participants
Initial Acute Myeloid Leukemia (AML) Classification Missing	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Type of Acute Myeloid Leukemia (AML) Primary (de novo)	213 Participants n=5 Participants	216 Participants n=7 Participants	429 Participants n=5 Participants
Type of Acute Myeloid Leukemia (AML) Secondary	25 Participants n=5 Participants	18 Participants n=7 Participants	43 Participants n=5 Participants
Cytogenetic Risk Category at Diagnosis Intermediate	203 Participants n=5 Participants	203 Participants n=7 Participants	406 Participants n=5 Participants
Cytogenetic Risk Category at Diagnosis Poor	35 Participants n=5 Participants	31 Participants n=7 Participants	66 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 0	116 Participants n=5 Participants	111 Participants n=7 Participants	227 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 1	101 Participants n=5 Participants	106 Participants n=7 Participants	207 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 2	21 Participants n=5 Participants	15 Participants n=7 Participants	36 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Grade 3	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 (randomization) up to data cut off date of 15 July 2019; median follow-up for OS estimated by the reverse K-M method was 41.2 months for all participants.

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

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=238 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=234 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Kaplan-Meier (K-M) Estimate for Overall Survival (OS)	24.7 Months Interval 18.7 to 30.5	14.8 Months Interval 11.7 to 17.6

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: The intent to treat population includes participants who were randomized, regardless of whether they received treatment or not.

RFS was defined as the time from the date of randomization to the date of documented relapse or death from any cause, whichever occurred first. Participants who were still alive without documented relapse, or who were lost to follow-up or withdrew consent without documented relapse, were censored at the date of their last bone marrow assessment, prior to receiving any other therapy for AML. Documented relapse was defined as the earliest date of the following: • ≥ 5% bone marrow blasts (myeloblasts) from Central Pathology report, or • appearance of \> 0% blasts in the peripheral blood with a later bone marrow confirmation (bone marrow blast \[myeloblasts\] ≥ 5%) within 100 days, or • at least 2 peripheral blasts ≥ 5% within 30 days.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=238 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=234 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Kaplan-Meier Estimate of Relapse Free Survival (RFS)	10.2 Months Interval 7.9 to 12.9	4.8 Months Interval 4.6 to 6.4

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: The intent to treat population includes participants who were randomized, regardless of whether they received treatment or not.

Time to relapse was defined as the interval (in months) from the date of randomization to the date of documented relapse. Estimates of relapse rate were based on the cumulative incidence function from a competing risk analysis with death as a competing risk for relapse from complete remission (CR)/ complete remission with incomplete blood count recovery (CRi). Documented relapse was defined as, the earliest date of the following: • ≥ 5% bone marrow blasts (myeloblasts) from Central Pathology report, or • appearance of \> 0% blasts in the peripheral blood with a later bone marrow confirmation (bone marrow blast \[myeloblasts\] ≥ 5%) within 100 days, or • at least 2 peripheral blasts ≥ 5% within 30 days.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=238 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=234 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Kaplan-Meier Estimate of Time to Relapse	10.2 months Interval 8.3 to 13.4	4.9 months Interval 4.6 to 6.4

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: The intent to treat population includes participants who were randomized, regardless of whether they received treatment or not.

Time to discontinuation from treatment was assessed and defined as the interval from the date of randomization to the date of discontinuation from study drug. Participants who were receiving treatment at the time of study closure were censored at the date of last visit. Estimates of relapse rate were based on the cumulative incidence function from a competing risk analysis with death as a competing risk for relapse from CR/ CRi.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=238 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=234 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Kaplan-Meier Estimates of Time to Discontinuation From Treatment	14.6 months Interval 11.3 to 20.1	6.9 months Interval 5.3 to 7.9

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: The safety population included all randomized participants who received at least 1 dose of study drug.

TEAEs include AEs that started between first dose date and 28 days after the last dose of study drug. A serious adverse event (SAE) is: • Death • Life-threatening event • Inpatient hospitalization or prolongation of existing hospitalization • Persistent or significant disability or incapacity • Congenital anomaly or birth defect • Other important medical event The severity of AEs were assessed by the investigator according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0: Grade 1 (Mild): asymptomatic/mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 (Moderate): minimal, local or noninvasive intervention indicated; limiting age-appropriate activities of daily living. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care Grade 4: Life-threatening; urgent intervention indicated. Grade 5: Death due to AE.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=236 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=233 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 TEAE	235 Participants	233 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 TEAE Related to Study Treatment	213 Participants	121 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 Serious TEAE	110 Participants	109 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 Treatment Related Serious TEAE	22 Participants	5 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 Grade 3/4 TEAE	207 Participants	201 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 Treatment Related Grade 3/4 TEAE	113 Participants	55 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 TEAE Leading to Death	15 Participants	11 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 TEAE Leading to Dose Reduction (Red)	37 Participants	6 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 TEAE Leading to Dose Interruption	107 Participants	43 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 TEAE Leading to Dose Red and Interruption	25 Participants	3 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) ≥ 1 TEAE Leading to Study Drug Discontinuation	155 Participants	170 Participants

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: All treated participants with FACIT-Fatigue measurement by EOT

The functional assessment of chronic illness therapy (FACIT-Fatigue Scale V 4.0) is a subscale of the FACIT-F and has been validated in the oncology setting. The FACIT-Fatigue Scale is a short, 13-item, self-administered tool that measures the level of fatigue in an individual during usually daily activities over the past week. The level of fatigue is measured on a 5-point Likert scale (0 = not at all; 4 = very much. The scores range from 0 to 52, with higher scores indicating less fatigue. If there were missing items, but the participant answered at least 50% of the items, then subscores were prorated.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=165 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=150 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Mean Change in the Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale V 4.0) Score From Baseline	-3.7 units on a scale Standard Deviation 10.92	-2.5 units on a scale Standard Deviation 9.93

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: All treated participants with a EQ-5D-3L measurement at the end of treatment

The EQ-5D-3L is a self-administered questionnaire consisting of 5 questions, pertaining to specific health dimensions (ie, mobility, self-care, pain, usual activities, and anxiety/depression) and a health status scale. Each question has 3 levels of severity, corresponding to no problems, moderate problems and severe problems. Canadian population sample weights were used to derive health utility scores. A higher utility score represents a better health state. A clinically meaningful improvement or worsening was defined as at least 0.08 points of improvement or 0.10 points of worsening from baseline, respectively, for the EQ-5D-3L Health Utility Index. The instrument is scored using the United Kingdom (UK) index ranges from -0.594 to 1, where 0 equates to death and 1 equates to full health; -0.594 is considered 'worse than death'.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=164 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=150 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Mean Change in the European Quality of Life-Five Dimensions-Three Levels (EQ-5D-3L) Score From Baseline	-0.0416 Units on a scale Standard Deviation 0.15467	-0.0152 Units on a scale Standard Deviation 0.14799

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months

Population: The Health Related Quality of Life (HRQoL) evaluable population was defined as the ITT participants with a non-missing EQ-5D-3L score at baseline (C1D1) and at least one post-baseline visit. Results are presented for each post-baseline visit with sample size ≥ 25 in both arms. Death was censored at the date of the last HRQoL assessment visit.

Clinically meaningful deterioration was defined at least 0.10 point of deterioration from baseline for at least 2 consecutive visits for the EQ-5D Health Utility Index. The EQ-5D-3L is a self-administered questionnaire consisting of 5 questions, pertaining to specific health dimensions (ie, mobility, self-care, pain, usual activities, and anxiety/depression) and a health status scale. Each question has 3 levels of severity, corresponding to no problems, moderate problems and severe problems. Canadian population sample weights were used to derive health utility scores. A higher utility score represents a better health state. A clinically meaningful improvement or worsening was defined as at least 0.08 points of improvement or 0.10 points of worsening from baseline, respectively, for the EQ-5D-3L Health Utility Index. The EQ-5D-3L is scored using the UK index ranges from -0.594 to 1, where 0 equates to death and 1 equates to full health; -0.594 is considered 'worse than death'.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=225 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=217 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Time to Definitive Clinically Meaningful Deterioration for ≥ 2 Consecutive Visits as Measured Using the EQ-5D HRQoL Scale	NA Weeks Interval 135.1 to Not estimable because the survival curve does not drop to or below 0.5, i.e. the cumulative probability of developing clinically meaningful deterioration does not reach or exceed 50%, during the observation period	NA Weeks Interval 122.9 to Not estimable because the survival curve does not drop to or below 0.5, i.e. the cumulative probability of developing clinically meaningful deterioration does not reach or exceed 50%, during the observation period

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: Safety population includes all participants who received at least 1 dose of study drug.

HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=236 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=233 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Healthcare Resource Utilization (HRU): Rate of Hospital Events Per Person Year	0.36 Hospitalizations per person-years Interval 0.31 to 0.42	0.63 Hospitalizations per person-years Interval 0.54 to 0.74

SECONDARY outcome

Timeframe: From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months

Population: Safety population includes all participants who received at least 1 dose of study drug.

Outcome measures

Outcome measures
Measure	Oral Azacitidine Plus Best Supportive Care n=236 Participants Participants received 300 mg azacitidine tablets once a day (QD) for the first 14 days of each 28-day treatment cycle until discontinuation, which includes the following reasons: disease relapse, withdrawal of consent, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, death, lost to follow-up, or protocol violation or until the end of the study.	Placebo Plus Best Supportive Care n=233 Participants Participants received identically matching placebo tablets QD for the first 14 days of each 28-day treatment cycle until no longer receiving benefit, withdrawal of consent, disease relapse, adverse events, participant became eligible for allogeneic bone marrow or stem cell transplantation during the treatment period, lost to follow-up, or protocol violation or until the end of the study.
Healthcare Resource Utilization (HRU): Number of Days Hospitalized Per Person-Year	6.00 Days per person-years Interval 5.78 to 6.22	13.13 Days per person-years Interval 12.68 to 13.6

Adverse Events

Oral Azacitidine Plus Best Supportive Care

Serious events: 110 serious events

Other events: 235 other events

Deaths: 172 deaths

Placebo Plus Best Supportive Care

Serious events: 109 serious events

Other events: 224 other events

Deaths: 176 deaths

Serious adverse events

Other adverse events

Additional Information

Bristol-Myers Squibb Study Director

Bristol-Myers Squibb

Phone: Please email

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Bristol-Myers Squibb Co. agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing, but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
Publication restrictions are in place

Restriction type: OTHER