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<p>Paediatric acute myeloid leukaemia (AML) is a&#xa0;biologically distinct disease from adult AML, characterised by a&#xa0;predominance of gene fusions and age-specific genomic patterns. Advances in molecular profiling have refined risk stratification, particularly through the integration of measurable residual disease (MRD), and have identified novel therapeutic targets. While intensive chemotherapy remains the backbone of treatment, outcomes differ substantially from adult AML, and the efficacy of targeted agents varies across age groups. Emerging therapies, including FLT3 and menin inhibitors or venetoclax, offer promising strategies, particularly for high-risk subtypes. Continued international collaboration and paediatric-specific clinical trials are essential to translate biological insights into improved outcomes.</p>

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Paediatric AML in the context of adult AML

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年7月7日

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  • Anna Cvrtak,
  • Michael N. Dworzak

摘要

Paediatric acute myeloid leukaemia (AML) is a biologically distinct disease from adult AML, characterised by a predominance of gene fusions and age-specific genomic patterns. Advances in molecular profiling have refined risk stratification, particularly through the integration of measurable residual disease (MRD), and have identified novel therapeutic targets. While intensive chemotherapy remains the backbone of treatment, outcomes differ substantially from adult AML, and the efficacy of targeted agents varies across age groups. Emerging therapies, including FLT3 and menin inhibitors or venetoclax, offer promising strategies, particularly for high-risk subtypes. Continued international collaboration and paediatric-specific clinical trials are essential to translate biological insights into improved outcomes.