title: Optimal treatment intensity in children with Down syndrome and myeloid leukaemia: data from 56 children treated on NOPHO-AML protocols and a review of the literature.
abstract: Children with Down syndrome (DS) and myeloid leukaemia have a significantly higher survival rate than other children, but they also experience considerable treatment-related toxicity. We analysed data on 56 children with DS who were treated on the Nordic Society for Paediatric Haematology and Oncology-acute myeloid leukaemia (NOPHO-AML)88 and NOPHO-AML93 protocols and reviewed the literature. In the dose-intensive NOPHO-AML88 protocol, 8 out of 15 patients (53%) experienced an event. In the less dose-intensive NOPHO-AML93 protocol, 7 out of 41 patients (17%) had an event. Therapy was reduced in 29 patients (52%) with in average 75% and 67% of the scheduled dose of anthracycline and cytarabine, respectively. Treatment-related death occurred in seven who all received full treatment. Relapse and resistant disease occurred at a similar rate in those receiving full and reduced treatment. Review of major series of myeloid leukaemia of DS showed no clear relationship between dose and survival; however, it appears that both a reduction in treatment dose and a less intensively timed treatment regimen improved the outcome. Further studies are needed to define the optimal regimen for treating myeloid leukaemia of DS.
authors.affiliation: Department of Paediatrics, Skejby Hospital, 8200, Aarhus N, Denmark.
authors.forename: Lotte
authors.initials: L
authors.lastname: Abildgaard
authors.forename: Eva
authors.initials: E
authors.lastname: Ellebaek
authors.forename: Göran
authors.initials: G
authors.lastname: Gustafsson
authors.forename: Jonas
authors.initials: J
authors.lastname: Abrahamsson
authors.forename: Liisa
authors.initials: L
authors.lastname: Hovi
authors.forename: Gudmundur
authors.initials: G
authors.lastname: Jonmundsson
authors.forename: Bernward
authors.initials: B
authors.lastname: Zeller
authors.forename: Henrik
authors.initials: H
authors.lastname: Hasle
mesh_terms: D018943:Anthracyclines / Q000008:administration & dosage / Q000009:adverse effects; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000009:adverse effects; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage / Q000009:adverse effects; D018572:Disease-Free Survival; D004314:Down Syndrome / Q000150:complications / Q000401:mortality; D006801:Humans; D007951:Leukemia, Myeloid / Q000150:complications / Q000188:drug therapy* / Q000401:mortality; D008297:Male; D012074:Remission Induction; D012189:Retrospective Studies; D015996:Survival Rate
chemical_list: D018943:Anthracyclines; D003561:Cytarabine