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