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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

Phase 1 trial evaluates venetoclax safety and impacts chemotherapy efficacy in pediatric AML patients, suggesting improved outcomes.

Key Points

  • To investigate the safety, tolerability, and preliminary efficacy of venetoclax in pediatric patients with relapsed/refractory AML.
  • Single-arm, multicenter, phase 1 dose-escalation study of venetoclax with azacitidine and vorinostat.
  • Eligible patients aged 1-25 with relapsed/refractory AML or therapy-related AML were enrolled.
  • Venetoclax is administered alongside 5-azacitidine and FLAG chemotherapy regimen.
  • Primary endpoint is the occurrence of dose-limiting toxicity (DLT) during cycle 1.
  • Secondary endpoints include response rates and survival analyses.
  • The regimen was well tolerated with a 54% complete response rate in previous studies.
  • Eight patients have been enrolled to date, with ongoing recruitment expected until late 2026.
  • Exploratory analysis includes patients with Down syndrome and correlative studies.

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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