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Transforming paediatric AML trials: from failing one-size-fits-all methods to precision oncology

Nature Reviews Clinical Oncology volume 22, pages 155–156 (2025)Cite this article

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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.

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Fig. 1: The limitations of a one-size-fits-all approach to paediatric AML.

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Acknowledgements

University of Texas MD Anderson is supported by MD Anderson Cancer Center Support Grant (P30 CA016672). The content is solely the responsibility of the authors and does not necessarily represent the official views of the institution.

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Authors and Affiliations

  1. The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Branko Cuglievan

  2. Sarah Cannon Research Institute, Nashville, TN, USA

    Vivek Subbiah

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  1. Branko Cuglievan
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  2. Vivek Subbiah
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Correspondence to Vivek Subbiah.

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Competing interests

B.C. has received research funding from Kura Oncology and the Leukemia Lymphoma Society, Octapharma and Syndax Pharmaceuticals and travel support from Octapharma and Syndax Pharmaceuticals. V.S. has acted (directly) as a consultant and/or adviser of Bayer, Daiichi Sankyo, Illumina, Incyte, Jazz Pharmaceuticals, Loxo Oncology/Lilly, Novartis and Relay Therapeutics, has acted as a consultant or adviser (with reimbursement to SCRI) of Abbvie, Agenus, Astex Pharmaceuticals, AstraZeneca, Bayer, Endeavor BioMedicine, Enlaza Therapeutics, Genmab, Hyku Biosciences, Incyte, Jazz Pharmaceuticals, LabGenius Limited, Lilly/Loxo Oncology, Merck, Nimbus Discovery, Novartis, Obsidian Therapeutics, PERS, Pfizer, Pheon Therapeutics, Regeneron, Relay Therapeutics, Revolution Medicine, Roche, has received institutional research funding from Abbvie, Amgen, Arvinas, Bayer, Blueprint Medicines, Deciphera, Dragonfly Therapeutics, Exelixis, Fujifilm, GlaxoSmithKline, Incyte, Inhibrix, Loxo Oncology/Eli Lilly, Novartis, PharmaMar, Pfizer, OnCusp, Relay Therapeutics, Roche/Genentech, Synnovation Therapeutics, Takeda, Turning Point Therapeutics, Xencor, CME education funds from Clinical care communications, Medscape and OncLive, and personal travel expenses from AACR, ASCO and ESMO.

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Cuglievan, B., Subbiah, V. Transforming paediatric AML trials: from failing one-size-fits-all methods to precision oncology. Nat Rev Clin Oncol 22, 155–156 (2025). https://doi.org/10.1038/s41571-025-00989-0

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