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Novel therapies for children with acute myeloid leukaemia

Moore, A. S., Kearns, P. R., Knapper, Steven ORCID:, Pearson, A. D. J. and Zwaan, C. M. 2013. Novel therapies for children with acute myeloid leukaemia. Leukemia 27 (7) , pp. 1451-1460. 10.1038/leu.2013.106

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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.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Subjects: R Medicine > R Medicine (General)
R Medicine > RC Internal medicine > RC0254 Neoplasms. Tumors. Oncology (including Cancer)
R Medicine > RJ Pediatrics
Uncontrolled Keywords: acute myeloid leukaemia; AML; children; novel therapeutics; inhibitors; chemotherapy
Publisher: Nature Publishing Group
ISSN: 0887-6924
Date of Acceptance: 4 April 2013
Last Modified: 28 Oct 2022 09:40

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