Outcome of unrelated donor stem cell transplantation for children with severe aplastic anemia

Abstract
Summary: For children with severe aplastic anemia (SAA) who fail immunosuppressive therapy and lack a human leucocyte antigen (HLA)‐matched sibling donor, unrelated donors provide a source of hematopoietic stem cells. Data from 195 children with acquired SAA who underwent unrelated donor transplantation between 1989 and 2003 were analyzed. Neutrophil recovery (86% at day‐28) was higher with total body irradiation‐containing conditioning regimen and in younger recipients (aged ≤16 years) receiving grafts from older donors (aged >40 years). Recovery was lower after mismatched transplants and transplantations prior to 1997. Mortality rates were higher after mismatched transplants, in recipients with a poor performance score, and when the interval between diagnosis and transplantation was longer than 4 years. When restricted to donor‐recipient pairs with allele‐level HLA typing (8‐loci;n = 118), mortality rates were also higher after mismatched transplants and older recipients receiving grafts from older donors; 5‐year probabilities of overall survival after HLA‐A, ‐B, ‐C, ‐DRB1 matched and mismatched transplants adjusted for donor and recipient age were 57% and 39%, respectively (P = 0·008). The data suggest that unrelated donor transplantation is an acceptable alternative for children; early referral for transplantation and identification of an HLA‐matched (allele‐level) donor offers the best outcome.

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