ArticleBlood neoplasia2026
Hypomethylating agents-venetoclax as salvage therapy for relapse of AML and MDS after allogeneic HCT.
Article in Blood neoplasia, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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25 authors.
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Abstract
Relapse after allogeneic hematopoietic cell transplantation (alloHCT) remains a challenge for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), and salvage therapy in this setting is not standardized. Given the efficacy of hypomethylating agents and venetoclax (HMA-VEN) in the frontline and relapsed settings, we evaluated the use of this therapy for post-alloHCT relapse. Eighty-three patients with AML (n = 69) or MDS (n = 14) who relapsed after alloHCT were included in this retrospective analysis. The median time to relapse after alloHCT was 7.0 months. Most patients (55%) had prior VEN exposure, and 28% of these patients had previously experienced treatment failure with a VEN-based regimen. HMA-VEN treatment-emergent toxicities were predominantly neutropenia and thrombocytopenia; however, the 30-day mortality was only 3.6%. There were 3 deaths within 30 days because of infectious complications. HMA-VEN led to a complete response (CR) or CR with incomplete hematological recovery in 48% of the patients, with 58% of these patients achieving minimal residual disease negativity. In multivariable analysis, European LeukemiaNet 2024 genetic risk stratification was a predictor of survival outcome. Additionally, prior VEN failure was not a predictor of overall survival. In conclusion, HMA-VEN provides an efficacious and well-tolerated option for post-alloHCT AML or MDS relapse regardless of their prior therapy and may allow responders to undergo a second alloHCT with curative intent.
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