Objective To evaluate the efficacy and safety of venetoclax in the treatment of relapsed/refractory acute myeloid leukemia (AML) by meta-analysis. Method We searched PubMed, Embase and the Cochrane Library for relevant studies. Randomized controlled trials (RCTs) or clinical trials that included venetoclax in relapsed/refractory acute myeloid leukemia published with the primary outcome of overall survival (OS). According to the heterogeneity among different studies, Revman5.4 was used to

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... fixed effect or random effect model analysis. Egger test were used to detect publication bias. The GRADE system was used to evaluate the quality of the evidence for the primary outcome measure. Results A total of 5 RCTs were included, including 487 cases in the experimental group and 251 cases in the control group. Compared with the control group, the overall survival (HR=1.56, 95%CI: 1.38-1.77, P < 0.00001) of relapsed/refractory acute myeloid leukemia was longer and better with venetoclax. However, the risk of hematological adverse events (HR=1.25, 95% CI: 0.99-1.58, P=0.05), non-hematological adverse events (HR=1.22, 95% CI: 1.09-1.36, P=0.29) and serious adverse events (HR=1.33, 95% CI: 1.07-1.65, P=0.002) in the treatment of relapsed/refractory acute myeloid leukemia was relatively low. Conclusions Compared with the control group, Venetoclax can improve the clinical efficacy of patients with relapsed/refractory acute myeloid leukemia, but its adverse reaction rate is relatively high. This therapy may be a promising treatment for patients with relapsed/refractory acute myeloid leukemia, but it is necessary to monitor the related adverse events of patients for symptomatic treatment during use.