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December 8, 2025BloodOpen Access

Phase II study of clinical efficacy of venetoclax in combination with azacitidine in patients with therapy related myelodysplastic syndrome (t-MDS)

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Authors

UBUma BorateSHShivani HandaCLCurtis A. Lachowiez

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Overview

Multi-center trial shows effective response rates in patients with t-MDS, highlighting the role of venetoclax and azacitidine.

Key Points

  • A combination therapy using venetoclax and azacitidine achieved a response rate exceeding 50% in therapy related myelodysplastic syndrome patients.
  • The overall response rate was documented at 56.5% including complete remission in 30% of cases, with a median follow-up of 16 months.
  • Multi-center trial design allowed for broad patient enrollment with specific focus on TP53 mutations and bone marrow status.
  • The findings support continued investigation into venetoclax and azacitidine for patients with limited treatment options due to complex karyotype.

Cite This Study

Borate et al. (2025) studied this question.

synapsesocial.com/papers/69362f5d4fa91c937236dc6dhttps://doi.org/10.1182/blood-2025-238
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Also Consider

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

  1. 1A phase I/II trial of azacitidine and venetoclax in previously untreated Myelodysplastic Syndromes2025 · 1 citations
  2. 2Venetoclax and azacytidine in childhood primary advanced myelodysplastic syndromes, refractory/relapsed Acute Myeloid Leukemia and therapy-related myeloid diseases2025
  3. 3Azacitidine combined with Venetoclax for pre-emptive treatment of AML/MDS after allogeneic stem cell transplantation: A prospective phase II study2025
  4. 4A pre-emptive bridge-to-transplant therapy for measurable residual disease with venetoclax and azacitidine in NPM1-mutated Acute Myeloid Leukemia: Updates from the ongoing GIMEMA AML2521 phase 2 trial2025
  5. 5Subgroup analyses from the randomized, Phase 3 VERONA study of venetoclax with azacitidine (Ven+Aza) versus placebo with azacitidine (Pbo+Aza) in patients with treatment-naïve, intermediate and higher-risk Myelodysplastic Syndromes (HR MDS)2025 · 6 citations