The efficacy of varying doses of venetoclax combined with azacitidine for newly diagnosed adult acute myeloid leukemia in real-world settings: a single-center retrospective analysis.

[INTRODUCTION] The combination of venetoclax and azacitidine shows significant efficacy in newly diagnosed acute myeloid leukemia (ndAML) patients. In specific contexts, low-dose venetoclax may be preferred to optimize patient tolerance.

[METHOD] The study included 64 adult ndAML patients treated at the Affiliated Hospital of Nantong University between April 1, 2021, and April 30, 2024, investigated the efficacy and outcomes of standard-dose versus reduced-dose venetoclax in combination with azacitidine for induction chemotherapy in adult ndAML patients.

[RESULTS] The analysis found no significant difference in composite complete remission (CRc) rates after the first cycle between groups; however, the median event-free survival (EFS) ( = 0.006) and overall survival (OS) ( = 0.014) were markedly shorter in the reduced-dose cohort. Additionally, validation analysis of the 2024 LeukemiaNet (ELN) risk classification revealed a noteworthy distinction in OS among patients with distinct risk classifications receiving reduced-dose venetoclax plus azacitidine ( = 0.048).

[CONCLUSION] These findings suggest that while both dosing strategies may yield comparable initial responses, long-term outcomes could be adversely influenced by dose reduction, and the 2024 ELN risk classification is more suitable for adult patients with ndAML receiving a reduced-dose regimen of venetoclax and azacitidine.