title: Allogenic stem cell transplantation for nonmalignant disorders using matched unrelated donors.
abstract: We here report 25 patients with nonmalignant disorders, ie, severe aplastic anemia (SAA, n = 12) or inborn errors of metabolism (IEM, n = 13), who underwent allogeneic hematopoietic stem cell transplantation (HSCT) from unrelated high-resolution typed HLA-A, -B, and -DRbeta1 identical donors. One patient had an HLA-B subtype-mismatched donor. Conditioning for SAA mainly consisted of cyclophosphamide and total body irradiation, and that for IEM consisted of busulfan and cyclophosphamide. All patients received antithymocyte globulin during conditioning. After HSCT, they were given cyclosporine combined with methotrexate for immunosuppression. Two patients rejected their grafts: 1 died of pneumonia, and the other was successfully regrafted. The cumulative incidence of acute graft-versus-host disease grades II to IV was 24%, whereas chronic graft-versus-host disease occurred in 21%. The 5-year survival rates were 83% in the SAA group and 85% in those with IEM. We conclude that HSCT with HLA-A, -B, and -DRbeta1 genomically matched unrelated donors in combination with antithymocyte globulin in the conditioning regimen gives encouraging results in patients with SAA or IEM.
authors.affiliation: Centre for Allogenic Stem Cell Transplantation, Department of Paediatrics, Karolinska University Hospital, Huddinge, SE-14186 Stockholm, Sweden. petter.svenberg@kus.se
authors.forename: Petter
authors.initials: P
authors.lastname: Svenberg
authors.forename: Mats
authors.initials: M
authors.lastname: Remberger
authors.forename: Johan
authors.initials: J
authors.lastname: Svennilson
authors.forename: Jonas
authors.initials: J
authors.lastname: Mattsson
authors.forename: Katarina
authors.initials: K
authors.lastname: Leblanc
authors.forename: Britt
authors.initials: B
authors.lastname: Gustafsson
authors.forename: Johan
authors.initials: J
authors.lastname: Aschan
authors.forename: Lisbeth
authors.initials: L
authors.lastname: Barkholt
authors.forename: Jacek
authors.initials: J
authors.lastname: Winiarski
authors.forename: Per
authors.initials: P
authors.lastname: Ljungman
authors.forename: Olle
authors.initials: O
authors.lastname: Ringdén
mesh_terms: D016470:Bacteremia / Q000628:therapy*; D003586:Cytomegalovirus Infections / Q000628:therapy; D004927:Escherichia coli Infections / Q000628:therapy; D006650:Histocompatibility Testing / Q000379:methods; D006801:Humans; D019520:Living Donors; D012189:Retrospective Studies; D013203:Staphylococcal Infections / Q000628:therapy; D033581:Stem Cell Transplantation / Q000401:mortality; D016019:Survival Analysis; D014184:Transplantation, Homologous / Q000401:mortality / Q000502:physiology*