title: Outcome of unrelated donor stem cell transplantation for children with severe aplastic anemia.
abstract: 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.
authors.affiliation: Division of Stem Cell Transplantation and Immunology, Children's National Medical Center, Washington, DC, USA.
authors.forename: Evelio D
authors.initials: ED
authors.lastname: Perez-Albuerne
authors.forename: Mary
authors.initials: M
authors.lastname: Eapen
authors.forename: John
authors.initials: J
authors.lastname: Klein
authors.forename: Thomas J
authors.initials: TJ
authors.lastname: Gross
authors.forename: Jeffery M
authors.initials: JM
authors.lastname: Lipton
authors.forename: K Scott
authors.initials: KS
authors.lastname: Baker
authors.forename: Anne
authors.initials: A
authors.lastname: Woolfrey
authors.forename: Naynesh
authors.initials: N
authors.lastname: Kamani
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000097:blood / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D006086:Graft vs Host Disease / Q000209:etiology; D006680:HLA Antigens / Q000032:analysis; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006650:Histocompatibility Testing; D006801:Humans; D007958:Leukocyte Count; D009504:Neutrophils / Q000473:pathology; D016019:Survival Analysis; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens