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July 1, 2026Blood Cancer JournalOpen Access

Outcomes of patients with higher-risk myelodysplastic syndromes/neoplasms treated with hypomethylating agents + venetoclax—an analysis from the International Consortium for MDS (icMDS) VALIDATE database

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Authors

JBJan Philipp BewersdorfMemorial Sloan Kettering Cancer CenterTKTariq KewanMayo ClinicLLLuca LaninoYale University

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Overview

Analysis reveals improved composite response rates with HMA plus venetoclax in higher-risk MDS patients, suggesting potential benefits in specific subsets.

Key Points

  • To evaluate whether venetoclax enhances outcomes for patients with higher-risk myelodysplastic syndromes when added to hypomethylating agents.
  • Analyzed data from 1907 patients across 31 centers in 9 countries.
  • Compared outcomes between HMA monotherapy and HMA plus venetoclax.
  • Responses assessed using IWG 2023 criteria.
  • Addition of venetoclax improved composite complete remission rates (48.8% vs. 27.7%, p < 0.001).
  • No significant difference in overall survival between HMA/venetoclax and HMA monotherapy (HR: 0.83; 95% CI: 0.64-1.07; p = 0.15).
  • Patients with TP53 wild-type disease showed improved OS (HR: 0.47; 95% CI: 0.29-0.74; p = 0.002).

Cite This Study

Bewersdorf et al. (2026) studied this question.

synapsesocial.com/papers/6a44add75cd2549c8bc43424https://doi.org/10.1038/s41408-026-01543-6
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Outcomes of patients (pts) with higher-risk myelodysplastic syndromes (HR-MDS) treated with hypomethylating agents (HMA) + venetoclax (VEN) – a large analysis from the international consortium for MDS (icMDS) validate database2025
  2. 2Hypomethylating agent with venetoclax versus hypomethylating agent alone in frontline management of myelodysplastic syndrome - impact of subgroups on clinical outcomes2025
  3. 3A real-world experience of venetoclax combined with hypomethylating agents vs. monotherapy hypomethylating agents in patients with myelodysplastic syndromes and chronic myelomonocytic leukemia patients2024 · 2 citations
  4. 4The role of hypomethylating agents and venetoclax combination in higher risk myelodysplastic syndromes2025
  5. 5Real-World Outcomes of Hypomethylating Agents and Venetoclax Combination Therapy in AML and Myelodysplastic Syndrome in Pakistan2026 · 1 citations