Outcome of transplantation for acute myelogenous leukemia in children with Down syndrome.

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Hitzler, JK; He, W; Doyle, J; Cairo, M; Camitta, BM; Chan, KW; Diaz Perez, MA; Fraser, C; Gross, TG; Horan, JT; Kennedy-Nasser, AA; Kitko, C; Kurtzberg, J; Lehmann, L; O'Brien, T; Pulsipher, MA; Smith, FO; Zhang, M-J; Eapen, M; Carpenter, PA; CIBMTR Pediatric Cancer Working Committee,

Published Date

  • June 2013

Published In

Volume / Issue

  • 19 / 6

Start / End Page

  • 893 - 897

PubMed ID

  • 23467128

Pubmed Central ID

  • PMC3707801

Electronic International Standard Serial Number (EISSN)

  • 1523-6536

Digital Object Identifier (DOI)

  • 10.1016/j.bbmt.2013.02.017


  • eng

Conference Location

  • United States