Novel therapies for children with acute myeloid leukaemia

Research output: Contribution to journalArticlepeer-review


External organisations

  • Cardiff University
  • Department of Haematology, University Medical Centre, Utrecht, The Netherlands.
  • Queensland Children's Medical Research Institute
  • Royal Children's Hospital Brisbane
  • Children's Health Queensland Hospital and Health Service
  • Divisions of Clinical Studies and Therapeutics
  • Institute of Cancer Research and Royal Marsden
  • School of Medicine
  • Innovative Therapies for Children with Cancer (ITCC)
  • European Consortium
  • Pediatric Oncology/Hematology
  • Erasmus MC/Sophia Children's Hospital


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. © 2013 Macmillan Publishers Limited.


Original languageEnglish
Pages (from-to)1451-1460
Number of pages10
Issue number7
Publication statusPublished - 1 Jul 2013


  • Acute myeloid leukaemia, AML, Chemotherapy, Children, Inhibitors, Novel therapeutics