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December 8, 2025BloodOpen Access

MRD-guided azacitidine maintenance improves outcomes in molecularly persistent pediatric core-binding factor AML: A retrospective cohort study

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YCYiqiao Chen

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Overview

Retrospective cohort study shows azacitidine maintenance improves DFS in pediatric AML, suggesting safety and efficacy of MRD-guided treatment.

Key Points

  • This study aims to assess the efficacy and safety of azacitidine maintenance therapy in pediatric patients with AML.
  • Retrospective cohort study involving 78 pediatric patients with de novo AML in remission.
  • Patients were stratified into azacitidine maintenance (n=27) or observation (n=51) groups.
  • Measurable residual disease was monitored using multiparameter flow cytometry and PCR for fusion transcripts.
  • Azacitidine maintenance therapy showed comparable OS (89.7% vs. 85.0%) and DFS (77.7% vs. 79.0%) to observation overall.
  • Intermediate-risk patients exhibited improved DFS (85.1% vs. 73.1%) and OS (92.9% vs. 81.6%).
  • In core-binding factor-AML with baseline fusion transcripts ≥0.1%, azacitidine significantly prolonged DFS (100% vs. 62.5%).
  • MRD negativity persisted in 92.6% of azacitidine-treated patients, with one molecular relapse (0.02% blasts).

Cite This Study

Yiqiao Chen (2025) studied this question.

synapsesocial.com/papers/69362f3d4fa91c937236d54dhttps://doi.org/10.1182/blood-2025-7050
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