When leukemia returns or resists treatment, options are limited and the stakes are high. This review combined data from many studies on adults with relapsed or refractory acute myeloid leukemia. It looked at using two drugs together: hypomethylating agents and venetoclax. The main finding was that about half of patients saw their cancer respond. Specifically, the overall response rate was 50%, and 43% achieved a complete remission or a similar deep response. About 42% had no measurable residual disease left. The median overall survival was 8 months, and 40% were alive after one year. The analysis included 2,289 patients. Safety signals included serious infections, febrile neutropenia, and low blood cell counts. A key caveat is the high statistical heterogeneity, meaning the studies varied a lot, so the results should be interpreted with some caution. This combination may be a potential re-induction regimen for these patients, but it was not compared to more intensive salvage regimens.
HMA-venetoclax yields 50% overall response rate in relapsed/refractory AML meta-analysisCombination therapy shows response in relapsed leukemia
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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.
What this means for you:
For relapsed leukemia, combining two drugs helped about half of patients respond, but the evidence is mixed. More on Acute Myeloid Leukemia
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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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