VA Consolidation in Intermediate-Risk AML

The goal of this prospective, randomized, open-label, multi-center study is to demonstrate that consolidation therapy with venetoclax + azacitidine (VA) is non-inferior to conventional intensive chemotherapy (cytarabine-based regimens) in this specific patient population, while offering a more favorable safety profile.Eligible patients will be randomized 1:1 to receive either 1-2 cycles of VA (Venetoclax 400mg d1-28 + Azacitidine 75mg/m² d1-7) or 1-2 cycles of intermediate/high-dose cytarabine (AraC) ± anthracycline. The primary endpoint is 2-year Leukemia-Free Survival (LFS). Secondary endpoints include pre-transplant MRD-negative rate, Overall Survival (OS), Cumulative Incidence of Relapse (CIR), Non-Relapse Mortality (NRM), and safety profile (CTCAE v5.0). This study will provide high-level evidence for consolidation therapy in intermediate-risk AML in the venetoclax era.

Inclusion criteria

• Confirmed diagnosis of acute myeloid leukemia (AML) according to WHO 2022 classification criteria;
• Age ≥ 18 years;
• Classified as intermediate-risk based on European LeukemiaNet (ELN) 2022 prognostic risk stratification;
• Achieved first complete remission (CR) or CR with incomplete hematologic recovery (CRi) after ≤ 2 cycles of Venetoclax + Azacitidine (VA) induction therapy;
• Has a suitable donor and is planned to undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT);
• ECOG performance status score 0 to 2
• Adequate organ function: creatinine clearance ≥ 50 mL/min; AST and ALT ≤ 3 × ULN; total bilirubin ≤ 2 × ULN; LVEF ≥ 50%; life expectancy > 8 weeks
• Voluntarily signed informed consent form and able to comply with study requirements

Exclusion criteria

• Clinically active cardiovascular disease (uncontrolled arrhythmia/hypertension, NYHA Class 3/4 heart failure, myocardial infarction within 3 months)
• Active central nervous system (CNS) leukemia or extramedullary infiltration
• Other serious diseases limiting participation (e.g., severe infection, renal failure)
• Known HIV infection or uncontrolled severe viral hepatitis
• Pregnant or breastfeeding women
• Inability to understand, comply with protocol, or sign informed consent
• Any other conditions deemed unsuitable by the investigator