Novel therapies for children with acute myeloid leukaemia

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

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Author Moore, A. S.
Kearns, P. R.
Knapper, S.
Pearson, A. D. J.
Zwaan, C. M.
Title Novel therapies for children with acute myeloid leukaemia
Journal name Leukemia   Check publisher's open access policy
ISSN 0887-6924
1476-5551
Publication date 2013-07-01
Year available 2013
Sub-type Critical review of research, literature review, critical commentary
DOI 10.1038/leu.2013.106
Open Access Status File (Author Post-print)
Volume 27
Issue 7
Start page 1451
End page 1460
Total pages 10
Place of publication United Kingdom
Publisher Nature Publishing Group
Language eng
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.
Formatted 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.
Keyword Acute myeloid leukaemia
AML
Children
Novel therapeutics
Inhibitors
Chemotherapy
Acute myelogenous leukemia
Internal tandem duplication
Preclinical testing program
Childhood-cancer survivor
Phase-II trial
Gemtuzumab ozogamicin
Valproic acid
Oncology-group
Pediatric-patients
Kinase inhibitors
Q-Index Code C1
Q-Index Status Confirmed Code
Grant ID C1178/A10294
Institutional Status UQ

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collections: Faculty of Health and Behavioural Sciences -- Publications
Official 2014 Collection
Queensland Children's Medical Research Institute Publications
 
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Citation counts: TR Web of Science Citation Count  Cited 25 times in Thomson Reuters Web of Science Article | Citations
Scopus Citation Count Cited 27 times in Scopus Article | Citations
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