This study aimed to investigate the efficacy and the clinical and molecular predictors of response and survival following venetoclax plus hypomethylating agents (VEN+HMAs) in relapsed/refractory acute myeloid leukemia (R/R AML) patients. We retrospectively analyzed 197 R/R AML patients with next-generation sequencing (NGS) treated with VEN+HMAs regimen. The median treatment cycle was 1 (1-4) cycle. Composite complete remission (CRc) including the complete remission (CR) plus CR with incomplete hematologic recovery (CRi) rate was 44.7%, and the overall response rate (ORR) was 59.9%. The median follow-up was 14.0 (0.7- 54.0) months, 1- year and 2- year overall survival (OS) was 55.4% and 40.2%, respectively. Mutations in NPM1 and SRSF2 were favorable factors for response (Odds Ratio (OR) 2.3, P = 0.008; OR 2.5, P = 0.036, respectively). Prior HMA, early relapse and GATA2 mutation were unfavorable factors for response [OR 0.4, P = 0.006; OR 0.4, P = 0.021; OR 0.1, P = 0.005, respectively]. For survival, mutations in NPM1 and IDH1/2 were the protective factors for OS; mutations in FLT3-ITD, TP53, DNMT3A and GATA2 were the risk factors for OS. In conclusion, VEN+HMAs regimen was effective for R/R AML patients, and the treatment response and OS were associated with genetic characteristics.

Disclosures

No relevant conflicts of interest to declare.

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2024
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