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December 8, 2025BloodOpen Access

Trial 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 Leukemia

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Authors

APAmy PanNGNathan GossaiSRSridhar Rao

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Overview

Pilot trial demonstrates safety of venetoclax combined with azacitidine and vorinostat in pediatric acute myeloid leukemia, suggesting enhanced response rates.

Key Points

  • Response rates improved significantly with venetoclax in conjunction with epigenetic agents.
  • Preliminary response data shows a 54% complete response rate in pediatric AML patients receiving the regimen.
  • This phase 1 trial investigates the safety and tolerability of venetoclax with azacitidine and vorinostat.
  • May identify novel strategies to improve chemotherapy resistance in acute myeloid leukemia.

Cite This Study

Pan et al. (2025) studied this question.

synapsesocial.com/papers/69362f6c4fa91c937236e02fhttps://doi.org/10.1182/blood-2025-5244
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Also Consider

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

  1. 1ITCC-101/APAL2020D: A randomized Phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamycin with or without venetoclax in children with relapsed Acute Myeloid Leukemia2025 · 1 citations
  2. 2Venetoclax in combination with cytarabine with or without idarubicin in children with relapsed or refractory acute myeloid leukemia: A multi-center phase II expansion study2025
  3. 3Venetoclax and azacytidine in childhood primary advanced myelodysplastic syndromes, refractory/relapsed Acute Myeloid Leukemia and therapy-related myeloid diseases2025
  4. 4Trial in progress: Phase II Study of (early) combination salvage therapy with venetoclax and intensified decitabine in relapsed/refractory AML (VenSwitch)2025
  5. 5Venetoclax combined with three-day multi-frequency Decitabine (DEC3-VEN) in the treatment of adult patients with de novo Acute Myeloid Leukemia：updated results of a phase II trial2025