Efficacy and safety of venetoclax plus azacitidine based regimens in the treatment of relapsed or refractory acute myeloid leukemia: a systematic review and meta-analysis.

This meta-analysis aimed to evaluate the efficacy and safety of venetoclax plus azacitidine (VEN + AZA) regimens in patients with relapsed or refractory acute myeloid leukemia (R/R AML) and to explore the effects of different combination strategies, including chemotherapy and targeted agents, on clinical outcomes. A systematic search of PubMed, Web of Science, Embase, and Cochrane Library databases was performed up to February 2025. Studies that reported complete remission or complete remission with incomplete hematologic recovery (CR/CRi) were included. Study quality was assessed using the Newcastle-Ottawa Scale (NOS) for Non-Randomized Controlled Trials (NRCTs). Pooled estimates were calculated using random-effects models, and subgroup analyses were performed. The CR/CRi rate for VEN + AZA-based regimens was 43% (95% CI: 33-53%), with substantial heterogeneity (I²=89.20%). Subgroup analysis indicated higher CR/CRi rates for VEN + AZA combined with chemotherapy (68%, 95% CI: 62-73%) compared to VEN + AZA alone (38%, 95% CI: 28-47%) or VEN + AZA with targeted agents (28%, 95% CI: 18-40%). The most common grade ≥ 3 adverse events were neutropenia (89%) and thrombocytopenia (82%). VEN + AZA combined with chemotherapy significantly improved CR/CRi rates in R/R AML compared to VEN + AZA alone or with targeted agents.