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

作者：Evelio D. Perez‐Albuerne, Mary Eapen, John P. Klein, Thomas J Gross, Jeffery M. Lipton, Kevin Scott Baker, Anne Woolfrey, Naynesh R. Kamani · 发表于：British Journal of Haematology · 年份：2008 · DOI：10.1111/j.1365-2141.2008.07030.x · 被引用次数：58 · 研究领域：Hematopoietic Stem Cell Transplantation、T-cell and B-cell Immunology、Immunotherapy and Immune Responses

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 < or =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.