HMA-venetoclax yields 50% overall response rate in relapsed/refractory AML meta-analysis
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Key Takeaway
Consider HMA-venetoclax as a re-induction option in R/R AML, but interpret pooled estimates cautiously due to high heterogeneity.
This systematic review and meta-analysis pooled data from 2289 adult patients with relapsed/refractory acute myeloid leukemia (AML) treated with a combination of hypomethylating agents (HMA) and venetoclax (VEN). The analysis aimed to characterize treatment response and survival outcomes in this difficult-to-treat population.
Key findings include a complete remission rate of 26% (95% CI: 24-48%), a composite CR/CRi rate of 43% (95% CI: 41-46%), and an overall response rate of 50% (95% CI: 48-53%). Measurable residual disease negativity was achieved in 42% of patients (95% CI: 38-46%). Median overall survival was 8 months (IQR: 3-25), with a one-year overall survival rate of 40% (IQR: 23-55).
Safety data indicate that grade ≥3 toxicities included febrile neutropenia, infection, and cytopenia. The authors note high statistical heterogeneity as a key limitation, which tempers the certainty of pooled estimates. The comparator was not reported, and the analysis does not directly compare HMA-VEN to more intensive salvage regimens.
Despite these limitations, the findings suggest HMA-VEN may serve as a potential re-induction regimen for patients with relapsed/refractory AML, though clinicians should interpret the results cautiously given the heterogeneity and lack of direct comparator data.
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View Original Abstract ↓
BACKGROUND: Despite recent advances in the treatment of acute myeloid leukemia (AML), primary refractory disease and relapse (R/R) remain frequent, and there is no clearly established standard of care in this setting. While the combination of hypomethylating agents (HMA) with venetoclax (VEN) is a standard front-line therapy for patient's ineligible for intensive chemotherapy, its efficacy in the R/R setting remains poorly defined. The purpose of this systematic review and meta-analysis was to review the efficacy and safety of HMA/VEN for management of R/R AML.
METHODS: A systematic review was conducted using Embase and MEDLINE, identifying studies published between 2017 and 2025. Inclusion required adult R/R AML cohorts with at least 20 participants. The primary focus included treatment response and adverse events.
RESULTS: A total of 32 studies (N = 2289) were included. Patients were heavily pretreated, with 34% of patients having prior HMA exposure. Pooled efficacy estimates showed: complete remission (CR) 26% (95% CI: 24-48%), composite CR (CR/CRi) 43% (95% CI: 41-46%), overall response rate (ORR) 50% (95% CI: 48-53%), and measurable residual disease (MRD) negativity 42% (95% CI: 38-46%). The median overall survival (OS) was 8 months (IQR: 3-25) with one-year OS 40% (IQR: 23-55). Safety concerns were notable, with significant grade ≥ 3 toxicities, including febrile neutropenia, infection, and cytopenia. No significant publication bias was observed.
CONCLUSION: This study demonstrates that HMA/VEN is a potential re-induction regimen for patients with R/R AML. However, the high statistical heterogeneity identified underscores the need for further randomized studies to establish efficacy compared to more intensive salvage regimens.
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