title: AML patients with Down syndrome have a high cure rate with AML-BFM therapy with reduced dose intensity.
abstract: Despite improved prognosis in acute myelogenous leukaemia (AML) children with Down syndrome (DS), therapy-related toxicity remained a problem. We compared 67 DS patients from study AML-BFM 98 with 51 DS patients of the previous study AML-BFM 93, and the non-DS groups of both studies. Compared to non-DS patients, DS patients were treated with reduced anthracycline doses, without high-dose cytarabine/mitoxantrone and without cranial irradiation. AML-DS patients were in median 1.8 years old, and 102/118 (86%) showed the typical morphology of acute megakaryoblastic leukaemia. In study 93, seven DS patients did not receive AML-specific chemotherapy, and treatment modifications were more common. Results improved significantly for patients treated in study 98 with a 3-year survival of 91+/-4 vs 70+/-7% in study 93 (P=0.001). There were no differences in outcome concerning the age groups 0-<or=2 and 2-<or=4 years (event-free survival for treated patients 0-<or=2 years 83+/-4%, 2-<or=4 years 81+/-7%). The cumulative incidence of relapses was significantly lower in DS (7+/-3%) than in non-DS patients (28+/-7%). Therapy-related toxicity was generally lower in DS patients treated according to study 98. We conclude that a standardised and dose-reduced treatment schedule including the main components of AML treatment is advisable for AML children with DS.
authors.affiliation: Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany. ursula@creutzig.de
authors.forename: U
authors.initials: U
authors.lastname: Creutzig
authors.forename: D
authors.initials: D
authors.lastname: Reinhardt
authors.forename: S
authors.initials: S
authors.lastname: Diekamp
authors.forename: M
authors.initials: M
authors.lastname: Dworzak
authors.forename: J
authors.initials: J
authors.lastname: Stary
authors.forename: M
authors.initials: M
authors.lastname: Zimmermann
mesh_terms: D000293:Adolescent; D000367:Age Factors; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000627:therapeutic use* / Q000633:toxicity; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage; D018572:Disease-Free Survival; D004314:Down Syndrome / Q000150:complications* / Q000188:drug therapy / Q000401:mortality; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D015255:Idarubicin / Q000008:administration & dosage; D007223:Infant; D007947:Leukemia, Megakaryoblastic, Acute / Q000473:pathology; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000209:etiology / Q000401:mortality; D008297:Male; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D003561:Cytarabine; D005047:Etoposide; D015255:Idarubicin