Trial Outcomes & Findings for STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS (NCT NCT04878432)

NCT ID: NCT04878432

Last Updated: 2025-10-16

Results Overview

An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a participant or clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 39 participants
• Primary outcome timeframe: Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months.
• Results posted on: 2025-10-16

Participant Flow

Participant milestones

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Overall Study STARTED	39
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	37

Reasons for withdrawal

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Overall Study Adverse Event	1
Overall Study Death	2
Overall Study Unsatisfactory Therapeutic Effect	3
Overall Study Withdrawal by Subject	6
Overall Study Progressive Disease	7
Overall Study New Therapy for Study Indication	7
Overall Study Physician Decision	11

Baseline Characteristics

STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS

Baseline characteristics by cohort

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=5 Participants
Age, Categorical >=65 years	25 Participants n=5 Participants
Age, Continuous	67.8 Years STANDARD_DEVIATION 11.49 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	25 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	31 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months.

Population: Safety set - The SS included all patients who received at least one dose of any component of the study medication (sabatolimab+HMA).

An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a participant or clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Adverse events (AEs)	39 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Treatment-related Adverse events	30 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events AEs with grade ≥ 3	35 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Treatment-related AEs with grade ≥ 3	25 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Serious Adverse Events (SAEs)	23 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Treatment-related SAEs	8 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Fatal SAEs	0 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Treatment-related Fatal SAEs	0 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events AEs leading to discontinuation	3 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Treatment-related AEs leading to discontinuation	3 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events AEs leading to dose adjustment/interruption	8 Participants
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events AEs requiring additional therapy	32 Participants

SECONDARY outcome

Timeframe: up to Month 12

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Complete remission rate was assessed according to International Working Group (IWG) for MDS (2006) with MBG453 (sabatolimab) in combination with HMAs (IV/SC/Oral) in Participants with intermediate, high, or very high risk MDS by 12 months.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Number of Participants With Complete Remission According to International Working Group (IWG) for MDS (2006) With MBG453 (Sabatolimab) in Combination With HMAs (IV/SC/Oral) by 12 Months.	1 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Defined as time from enrollment to disease progression (including transformation to leukemia per WHO 2016 classification), relapse from CR according to IWG-MDS or death due to any cause, whichever occurs first, as per investigator assessment by 12 months post last patient first treatment (LPFT).

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Number of Participants With Progression Free Survival - Death and Disease Progression Death	7 Participants
Number of Participants With Progression Free Survival - Death and Disease Progression Disease Progression	9 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Defined as time from enrollment to disease progression (including transformation to leukemia per WHO 2016 classification), relapse from CR according to IWG-MDS or death due to any cause, whichever occurs first, as per investigator assessment by 12 months post last patient first treatment (LPFT).

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Progression Free Survival - 50th Percentile	11.56 months Interval 4.24 to 16.59

SECONDARY outcome

Timeframe: Months 6 and 12

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Defined as time from enrollment to disease progression (including transformation to leukemia per WHO 2016 classification), relapse from CR according to IWG-MDS or death due to any cause, whichever occurs first, as per investigator assessment by 12 months post last patient first treatment (LPFT).

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Progression Free Survival - Percent Probability - Kaplan-Meier Probability Estimates 6 months	65.28 percent probability Interval 43.18 to 80.53
Progression Free Survival - Percent Probability - Kaplan-Meier Probability Estimates 12 months	43.27 percent probability Interval 19.45 to 65.14

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Overall Survival (OS) is defined as the time from the first dose of study medication to death due to any cause.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Overall Survival - Number of Participants Who Died	20 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Overall Survival (OS) is defined as the time from the first dose of study medication to death due to any cause.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Overall Survival - 50th Percentile	15.77 months Interval 8.74 to 19.19

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Overall Survival (OS) is defined as the time from the first dose of study medication to death due to any cause.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates 6 months	82.90 percent probability Interval 65.77 to 91.95
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates 9 months	65.66 percent probability Interval 46.24 to 79.5
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates 12 months	61.56 percent probability Interval 41.79 to 76.35
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates 15 months	53.35 percent probability Interval 33.57 to 69.65
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates 18 months	35.57 percent probability Interval 16.4 to 55.37
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates 21 months	28.45 percent probability Interval 10.67 to 49.37
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates 24 months	21.34 percent probability Interval 6.07 to 42.68

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Leukemia-free survival (LFS) is the time from the date of the first dose of medication to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or death due to any cause.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Leukemia-free Survival - Number of Participants Who Died and Number of Participants Who Progressed to Leukemia Death	16 Participants
Leukemia-free Survival - Number of Participants Who Died and Number of Participants Who Progressed to Leukemia Progressed to leukemia	6 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Leukemia-free survival (LFS) is the time from the date of the first dose of medication to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or death due to any cause.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Leukemia-free Survival - 50th Percentile	11.30 months Interval 6.01 to 16.59

SECONDARY outcome

Timeframe: Months 6 and 12

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Leukemia-free survival (LFS) is the time from the date of the first dose of medication to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or death due to any cause.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Leukemia-free Survival - Percent Probability - Kaplan-Meier Probability Estimates 6 months	70.58 percent probability Interval 50.67 to 83.65
Leukemia-free Survival - Percent Probability - Kaplan-Meier Probability Estimates 12 months	48.04 percent probability Interval 27.46 to 65.98

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

Percentage of complete remission, marrow complete remission, and/or partial remission according to IWG-MDS remission criteria as per investigator assessment

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission Complete Remission (CR)	1 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission Marrow Complete Remission (mCR)	6 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission mCR with hematological improvement (HI)	4 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission Partial Remission (PR)	2 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission Stable Disease (SD)	11 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission SD with hematological improvement (HI)	1 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission CR+mCR+PR	9 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission CR+PR+HI	8 Participants
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission CR+mCR+PR+HI	10 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication and who had complete remission.

Time to Complete Remission is defined as the time from the date of the first dose of study medication to the first documented CR.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=1 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Time to Complete Remission	5.91 months Standard Deviation NA Not applicable for a single patient who met this condition.

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

RBC/Platelets transfusion independence is defined as a participant having received <0 units RBC/Platelets transfusions during at least 8 consecutive weeks after enrollment.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Improvement in Packed RBCs Transfusion Independence - Mean Total Duration (Weeks) of Transfusion Independence Post-baseline	27.02 weeks Standard Deviation 21.194

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

RBC/Platelets transfusion independence rate is defined as the percentage of participants having received <0 units RBC/Platelets transfusions during at least 8 consecutive weeks after enrollment.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Number of Participants With Improvement in Packed RBCs Transfusion Independence - At Least One Period of Transfusion Independence Post Baseline	25 Participants

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

RBC/Platelets transfusion independence is defined as a participant having received <0 units RBC/Platelets transfusions during at least 8 consecutive weeks after enrollment

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Improvement in Platelets Transfusion Independence - Mean Total Duration (Weeks) of Transfusion Independence Post-baseline	27.41 weeks Standard Deviation 22.397

SECONDARY outcome

Timeframe: up to Month 24

Population: Full analysis set - The FAS comprised all enrolled patients who received any component of the study medication.

RBC/Platelets transfusion independence rate is defined as the proportion of participants having received <0 units RBC/Platelets transfusions during at least 8 consecutive weeks after enrollment.

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Number of Participants With Improvement in Platelets Transfusion Independence - At Least One Period of Transfusion Independence Post Baseline	27 Participants

POST_HOC outcome

Timeframe: Deaths are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months.

Population: Safety set - The SS included all patients who received at least one dose of any component of the study medication (sabatolimab+HMA).

Outcome measures

Measure	MBG453 (Sabatolimab) + Hypomethylating Agents (HMA) n=39 Participants MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
All Collected Deaths On-treatment Deaths	2 Participants
All Collected Deaths Off-treatment Deaths	18 Participants
All Collected Deaths Total Deaths	20 Participants

Adverse Events

MBG453+HMA

Serious events: 23 serious events

Other events: 35 other events

Deaths: 20 deaths

Serious adverse events

Measure	MBG453+HMA n=39 participants at risk MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Blood and lymphatic system disorders Febrile neutropenia	35.9% 14/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Blood and lymphatic system disorders Immune thrombocytopenia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Cardiac disorders Angina pectoris	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Cardiac disorders Atrial fibrillation	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Cardiac disorders Sinus tachycardia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Cardiac disorders Tachycardia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Eye disorders Photopsia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Gastrointestinal haemorrhage	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Haematochezia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Proctitis	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Small intestinal obstruction	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
General disorders Malaise	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
General disorders Physical deconditioning	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
General disorders Pyrexia	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Bacteraemia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations COVID-19	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Cellulitis	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Device related infection	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Enterocolitis infectious	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Pneumonia	12.8% 5/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Sepsis	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Urinary tract infection	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Injury, poisoning and procedural complications Fall	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Injury, poisoning and procedural complications Post procedural haemorrhage	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Hypervolaemia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Back pain	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Brain neoplasm	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Nervous system disorders Encephalopathy	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Psychiatric disorders Delirium	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Renal and urinary disorders Haematuria	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Respiratory, thoracic and mediastinal disorders Epistaxis	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Respiratory, thoracic and mediastinal disorders Hypoxia	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Skin and subcutaneous tissue disorders Penile ulceration	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Vascular disorders Embolism	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Vascular disorders Hypotension	2.6% 1/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.

Other adverse events

Measure	MBG453+HMA n=39 participants at risk MBG453 400 mg/4 ml + HMA (azacitidine 75 mg/m\^2, decitabine 20 mg/m\^2, or INQOVI (oral decitabine) 100 mg cedazuridine/35 mg)
Blood and lymphatic system disorders Anaemia	41.0% 16/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Blood and lymphatic system disorders Febrile neutropenia	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Blood and lymphatic system disorders Neutropenia	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Blood and lymphatic system disorders Thrombocytopenia	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Anorectal discomfort	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Constipation	38.5% 15/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Diarrhoea	20.5% 8/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Dry mouth	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Gastrooesophageal reflux disease	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Nausea	28.2% 11/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Stomatitis	15.4% 6/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Gastrointestinal disorders Vomiting	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
General disorders Chills	15.4% 6/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
General disorders Fatigue	30.8% 12/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
General disorders Oedema peripheral	17.9% 7/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
General disorders Pyrexia	12.8% 5/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Sinusitis	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Infections and infestations Tooth infection	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Injury, poisoning and procedural complications Contusion	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Injury, poisoning and procedural complications Fall	15.4% 6/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Injury, poisoning and procedural complications Infusion related reaction	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Injury, poisoning and procedural complications Skin abrasion	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations Blood bilirubin increased	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations Blood creatinine increased	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations Lipase increased	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations Lymphocyte count decreased	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations Neutrophil count decreased	38.5% 15/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations Platelet count decreased	41.0% 16/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations Weight decreased	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Investigations White blood cell count decreased	41.0% 16/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Abnormal loss of weight	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Decreased appetite	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Gout	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Hyperphosphataemia	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Hypoalbuminaemia	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Hypokalaemia	15.4% 6/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Hypomagnesaemia	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Metabolism and nutrition disorders Hyponatraemia	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Arthralgia	12.8% 5/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Back pain	15.4% 6/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Bone pain	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Joint swelling	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Muscular weakness	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Myalgia	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Neck pain	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Musculoskeletal and connective tissue disorders Pain in extremity	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Nervous system disorders Dizziness	17.9% 7/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Nervous system disorders Dysgeusia	10.3% 4/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Nervous system disorders Headache	30.8% 12/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Nervous system disorders Hypoaesthesia	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Nervous system disorders Tremor	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Psychiatric disorders Anxiety	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Psychiatric disorders Insomnia	15.4% 6/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Renal and urinary disorders Dysuria	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Renal and urinary disorders Haematuria	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Renal and urinary disorders Pollakiuria	12.8% 5/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Respiratory, thoracic and mediastinal disorders Cough	15.4% 6/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Respiratory, thoracic and mediastinal disorders Dyspnoea	17.9% 7/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Respiratory, thoracic and mediastinal disorders Epistaxis	12.8% 5/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Respiratory, thoracic and mediastinal disorders Nasal congestion	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Skin and subcutaneous tissue disorders Dry skin	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Skin and subcutaneous tissue disorders Hyperhidrosis	12.8% 5/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Skin and subcutaneous tissue disorders Pruritus	17.9% 7/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Skin and subcutaneous tissue disorders Rash	7.7% 3/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Vascular disorders Hot flush	5.1% 2/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.
Vascular disorders Hypertension	17.9% 7/39 • Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months. This was a single arm study. The study drugs of the hypomethylating agents decitabine, azacitidine, and oral decitabine (cedazuridine/decitabine) were considered to be clinically equivalent towards the standard of care for the treatment of higher risk Myelodysplastic syndrome at the doses administered; therefore, these were reported in the same arm. The study had a small sample size and was not powered to separate into different arms by design.

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: Novartis.email@Novartis.com

Results disclosure agreements

• Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
• Publication restrictions are in place

Restriction type: OTHER