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) (P = 0.006) and overall survival (OS) (P = 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 (P = 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.