Efficacy and prognostic analysis of venetoclax combined with hypomethylating agents for induction therapy in acute myeloid leukemia: a multi-center real-world study on indication-specific stratification, molecular markers, and hematologic toxicities.

ABSTRACT: The combination of venetoclax and hypomethylating agents (VEN + HMAs) has shown significant progress in treating acute myeloid leukemia (AML), especially for patients not suitable for intensive chemotherapy. However, outcomes for relapsed/refractory AML remain uncertain, and factors influencing VEN + HMAs efficacy are not yet conclusively determined. A retrospective analysis of 181 AML patients treated with VEN + HMAs from October 2020 to January 2024 revealed a CR rate of 39.2%, CR/CRi of 52.5%, ORR of 63.5%, and MRD negativity of 47.3% in patients receiving at least 7 days of treatment. Newly diagnosed patients had better outcomes than the relapsed/refractory group, with CEBPA or IDH1 mutations associated with better CR/CRi rates. Optimization of the treatment regimen with azacitidine ma

SUBMITTER: Xia L 

PROVIDER: S-EPMC12220736 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature