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Novel therapies for children with acute myeloid leukaemia

Leukemia volume 27, pages 1451–1460 (2013)Cite this article

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Abstract

Significant improvements in survival for children with acute myeloid leukaemia (AML) have been made over the past three decades, with overall survival rates now approximately 60–70%. However, these gains can be largely attributed to more intensive use of conventional cytotoxics made possible by advances in supportive care, and although over 90% of children achieve remission with frontline therapy, approximately one third in current protocols relapse. Furthermore, late effects of therapy cause significant morbidity for many survivors. Novel therapies are therefore desperately needed. Early-phase paediatric trials of several new agents such as clofarabine, sorafenib and gemtuzumab ozogamicin have shown encouraging results in recent years. Due to the relatively low incidence of AML in childhood, the success of paediatric early-phase clinical trials is largely dependent upon collaborative clinical trial design by international cooperative study groups. Successfully incorporating novel therapies into frontline therapy remains a challenge, but the potential for significant improvement in the duration and quality of survival for children with AML is high.

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Acknowledgements

ASM is supported by the National Health and Medical Research Council (Australia) and the Children’s Health Foundation Queensland. ADJP is supported by Cancer Research UK (programme grant C1178/A10294). ASM and ADJP acknowledge NHS funding to the NIHR Biomedical Research Centre at The Institute of Cancer Research and the Royal Marsden NHS Foundation Trust.

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Authors and Affiliations

  1. Queensland Children’s Medical Research Institute, The University of Queensland, Brisbane, Australia

    A S Moore

  2. Children’s Health Queensland Hospital and Health Service, Brisbane, Australia

    A S Moore

  3. Divisions of Clinical Studies and Therapeutics, The Institute of Cancer Research and Royal Marsden, Sutton, UK

    A S Moore & A D J Pearson

  4. School of Cancer Sciences, University of Birmingham, Birmingham, UK

    P R Kearns

  5. Innovative Therapies for Children with Cancer (ITCC) European Consortium,

    P R Kearns, A D J Pearson & C M Zwaan

  6. Department of Haematology, School of Medicine, Cardiff University, Cardiff, UK

    S Knapper

  7. Pediatric Oncology/Hematology, Erasmus MC/Sophia Children’s Hospital, Rotterdam, The Netherlands

    C M Zwaan

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  1. A S Moore
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  2. P R Kearns
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Correspondence to A S Moore.

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Competing interests

ASM and ADJP are past and present employees respectively of The Institute of Cancer Research, which has a commercial interest in drug development programmes (see www.icr.ac.uk), and are subject to a ‘Rewards to Inventors Scheme’ which may reward contributors to a programme that is subsequently licensed. ASM has received competitive research funding from Pfizer Inc., by way of a Pfizer Australia Cancer Research Grant.

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Moore, A., Kearns, P., Knapper, S. et al. Novel therapies for children with acute myeloid leukaemia. Leukemia 27, 1451–1460 (2013). https://doi.org/10.1038/leu.2013.106

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