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December 8, 2025BloodOpen Access

Venetoclax and azacytidine in childhood primary advanced myelodysplastic syndromes, refractory/relapsed Acute Myeloid Leukemia and therapy-related myeloid diseases

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Authors

GPGennaro PaganoEMEdoardo MuratoreFVFrancesca Vendemini

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Overview

Retrospective analysis shows venetoclax and azacytidine achieve complete response in childhood acute myeloid leukemia and myelodysplastic syndromes, suggesting a new treatment avenue.

Key Points

  • Forty-six patients with r/r-AML and MDS-EB were treated with venetoclax and azacytidine, achieving a complete response in 29 patients (58%).
  • Adverse events were noted in 17 patients, highlighting the importance of monitoring therapy tolerance including febrile neutropenia and fungal infection.
  • Bone marrow response assessments revealed non-response in 12 patients, indicating resistance to treatment in certain cases.
  • Overall, this analysis supports the potential for venetoclax and azacytidine to improve pediatric outcomes in refractory acute myeloid leukemia and associated syndromes.

Cite This Study

Pagano et al. (2025) studied this question.

synapsesocial.com/papers/69362f484fa91c937236d700https://doi.org/10.1182/blood-2025-289
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Also Consider

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

  1. 1Venetoclax in combination with cytarabine with or without idarubicin in children with relapsed or refractory acute myeloid leukemia: A multi-center phase II expansion study2025
  2. 2Phase II study of clinical efficacy of venetoclax in combination with azacitidine in patients with therapy related myelodysplastic syndrome (t-MDS)2025
  3. 3A phase I/II trial of azacitidine and venetoclax in previously untreated Myelodysplastic Syndromes2025 · 1 citations
  4. 4Azacitidine combined with Venetoclax for pre-emptive treatment of AML/MDS after allogeneic stem cell transplantation: A prospective phase II study2025
  5. 5Trial in progress: A pilot study of venetoclax to AUGMENT epigenetic modification and chemotherapy in pediatric and young adult patients with relapsed and refractory Acute Myeloid Leukemia2025 · 1 citations