Venetoclax Combined With Intensive Chemotherapy Regimens for Patients With Relapsed/Refractory Acute Myeloid Leukemia: A Systematic Review and Single-Arm Meta-Analysis.

OBJECTIVES

To evaluate the efficacy and safety of venetoclax combined with intensive chemotherapy regimens in patients with relapsed or refractory acute myeloid leukemia (R/R AML) through a systematic review and single-arm meta-analysis.

METHODS

A systematic search of PubMed, Embase, and Cochrane CENTRAL was conducted from inception to January 2025. Non-randomized studies enrolling R/R AML patients treated with venetoclax plus intensive chemotherapy were included. Pooled rates of complete remission (CR), composite complete remission (CRc), overall response rate (ORR), and adverse events were estimated using random-effects models. Heterogeneity was assessed with I2 statistics, and sensitivity and subgroup analyses were performed according to chemotherapy regimen.

RESULTS

Six retrospective studies comprising 235 patients were included. The pooled CR rate was 27.0% (95% CI, 9.18-57.50; I2 = 84.6%), CRc rate was 51.45% (95% CI, 36.15-66.76; I2 = 83.5%), and ORR was 63.79% (95% CI, 49.67-77.92; I2 = 82.0%). Febrile neutropenia occurred in 71.38% and pneumonia in 23.6% of patients.

CONCLUSIONS

Venetoclax combined with intensive chemotherapy demonstrates meaningful antileukemic activity in R/R AML, with an expected toxicity profile. Prospective trials are warranted to define its role relative to standard salvage therapies.