Azacitidine + Venetoclax VS Azacitidineas Maintenance Therapy in AML

A Study of Azacitidine With or Without Venetoclax as Maintenance Therapy in Acute Myeloid Leukemia

This is a prospective, multicenter, randomized, open-label, phase III trial evaluating the efficacy and safety of azacitidine plus venetoclax versus azacitidine alone as maintenance therapy in patients with acute myeloid leukemia (AML) who have achieved first complete remission (CR) or CR with incomplete count recovery (CRi) after induction and consolidation. Eligible patients aged 14 to 74 years are randomized 1:1 to receive either azacitidine 50 mg/m²/day on days 1-5 every 6 weeks for up to 12 cycles, or the same azacitidine regimen combined with venetoclax 400 mg on days 1-7 per cycle. The primary endpoint is disease-free survival (DFS). Secondary endpoints include overall survival (OS), relapse-free survival (RFS), cumulative incidence of relapse (CIR), minimal residual disease (MRD) conversion rate, and safety. A total of 788 patients are planned with stratification by prior venetoclax exposure and MRD status.

Inclusion Criteria:

• Diagnosis of AML per WHO 2022 or ICC criteria, or MDS/AML (10-20% blasts) per ICC
• Age ≥14 and <75 years
• ECOG performance status 0-2
• First complete remission (CR) or CR with incomplete count recovery (CRi) after induction and consolidation
• Received at least 2 cycles of intermediate- or high-dose cytarabine (cumulative dose ≥6 g/m² per cycle)
• Time from first CR/CRi to enrollment ≤10 months, and time from last treatment to enrollment ≤3 months
• MRD-negative or low-level MRD detectable; MRD-positive patients are excluded
• Signed informed consent

Exclusion Criteria:

• Acute promyelocytic leukemia with PML-RARA
• AML with BCR-ABL
• Active central nervous system involvement
• Prior allogeneic hematopoietic stem cell transplantation
• Planned allogeneic HSCT within 6 months
• Deemed unsuitable by investigator