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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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