{"title":"Transforming paediatric AML trials: from failing one-size-fits-all methods to precision oncology","authors":"Branko Cuglievan, Vivek Subbiah","doi":"10.1038/s41571-025-00989-0","DOIUrl":null,"url":null,"abstract":"Paediatric acute myeloid leukaemia (AML) highlights the challenges of drug development for rare diseases, in which limited patient numbers and substantial heterogeneity hinder progress. Traditional one-size-fits-all randomized trials are ineffective. Nonetheless, tailored therapies and biomarker-driven studies can improve outcomes and transform the treatment of paediatric patients with AML and potentially other rare cancers.","PeriodicalId":19079,"journal":{"name":"Nature Reviews Clinical Oncology","volume":"35 1","pages":""},"PeriodicalIF":81.1000,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nature Reviews Clinical Oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1038/s41571-025-00989-0","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Paediatric acute myeloid leukaemia (AML) highlights the challenges of drug development for rare diseases, in which limited patient numbers and substantial heterogeneity hinder progress. Traditional one-size-fits-all randomized trials are ineffective. Nonetheless, tailored therapies and biomarker-driven studies can improve outcomes and transform the treatment of paediatric patients with AML and potentially other rare cancers.
期刊介绍：
Nature Reviews publishes clinical content authored by internationally renowned clinical academics and researchers, catering to readers in the medical sciences at postgraduate levels and beyond. Although targeted at practicing doctors, researchers, and academics within specific specialties, the aim is to ensure accessibility for readers across various medical disciplines. The journal features in-depth Reviews offering authoritative and current information, contextualizing topics within the history and development of a field. Perspectives, News & Views articles, and the Research Highlights section provide topical discussions, opinions, and filtered primary research from diverse medical journals.
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