title: Novel therapies for children with acute myeloid leukaemia.
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.
authors.affiliation: Queensland Children's Medical Research Institute, The University of Queensland, Brisbane, Australia. andrew.moore@uq.edu.au
authors.forename: A S
authors.initials: AS
authors.lastname: Moore
authors.forename: P R
authors.initials: PR
authors.lastname: Kearns
authors.forename: S
authors.initials: S
authors.lastname: Knapper
authors.forename: A D J
authors.initials: AD
authors.lastname: Pearson
authors.forename: C M
authors.initials: CM
authors.lastname: Zwaan
mesh_terms: D000227:Adenine Nucleotides / Q000627:therapeutic use*; D000617:Aminoglycosides / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000627:therapeutic use; D001087:Arabinonucleosides / Q000627:therapeutic use*; D002648:Child; D000077866:Clofarabine; D000079982:Gemtuzumab; D006801:Humans; D000922:Immunotoxins / Q000627:therapeutic use; D007948:Leukemia, Monocytic, Acute / Q000188:drug therapy*; D009536:Niacinamide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000627:therapeutic use*; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D000077157:Sorafenib
chemical_list: D000227:Adenine Nucleotides; D000617:Aminoglycosides; D061067:Antibodies, Monoclonal, Humanized; D000970:Antineoplastic Agents; D001087:Arabinonucleosides; D000922:Immunotoxins; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D009536:Niacinamide; D000077866:Clofarabine; D000079982:Gemtuzumab; D000077157:Sorafenib