title: Outcome of transplantation for acute myelogenous leukemia in children with Down syndrome.
abstract: Data on outcomes of allogeneic transplantation in children with Down syndrome and acute myelogenous leukemia (DS-AML) are scarce and conflicting. Early reports stress treatment-related mortality as the main barrier; a recent case series points to posttransplantation relapse. We reviewed outcome data for 28 patients with DS-AML reported to the Center for International Blood and Marrow Transplant Research between 2000 and 2009 and performed a first matched-pair analysis of 21 patients with DS-AML and 80 non-DS AML controls. The median age at transplantation for DS-AML was 3 years, and almost half of the cohort was in second remission. The 3-year probability of overall survival was only 19%. In multivariate analysis, adjusting for interval from diagnosis to transplantation, risks of relapse (hazard ratio [HR], 2.84; P < .001; 62% versus 37%) and transplant-related mortality (HR, 2.52; P = .04; 24% versus 15%) were significantly higher for DS-AML compared to non-DS AML. Overall mortality risk (HR, 2.86; P < .001; 21% versus 52%) was significantly higher for DS-AML. Both transplant-related mortality and relapse contribute to higher mortality. Excess mortality in DS-AML patients can only effectively be addressed through an international multicenter effort to pilot strategies aimed at lowering both transplant-related mortality and relapse risks.
authors: The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
authors: Johann K
authors: JK
authors: Hitzler
authors: Wensheng
authors: W
authors: He
authors: John
authors: J
authors: Doyle
authors: Mitchell
authors: M
authors: Cairo
authors: Bruce M
authors: BM
authors: Camitta
authors: Ka Wah
authors: KW
authors: Chan
authors: Miguel A
authors: MA
authors: Diaz Perez
authors: Christopher
authors: C
authors: Fraser
authors: Thomas G
authors: TG
authors: Gross
authors: John T
authors: JT
authors: Horan
authors: Alana A
authors: AA
authors: Kennedy-Nasser
authors: Carrie
authors: C
authors: Kitko
authors: Joanne
authors: J
authors: Kurtzberg
authors: Leslie
authors: L
authors: Lehmann
authors: Tracey
authors: T
authors: O'Brien
authors: Michael A
authors: MA
authors: Pulsipher
authors: Franklin O
authors: FO
authors: Smith
authors: Mei-Jie
authors: MJ
authors: Zhang
authors: Mary
authors: M
authors: Eapen
authors: Paul A
authors: PA
authors: Carpenter
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D004314:Down Syndrome / Q000150:complications / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000150:complications / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D008297:Male; D012008:Recurrence; D012074:Remission Induction; D012307:Risk Factors; D016019:Survival Analysis; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents