title: Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan.
abstract: A total of 57 patients with Wiskott-Aldrich syndrome (WAS) were studied after undergoing stem cell transplantation (SCT) in Japan between January 1985 and December 2004. Eleven patients received transplants from human leucocyte antigen (HLA)-matched related donors, 10 from HLA-mismatched related donors, 21 from unrelated bone marrow donors, and 15 from unrelated cord blood donors. Nine of the 57 patients rejected the initial graft. The overall 5-year survival rate was 73.7% and the 5-year failure-free survival rate was 65.7% (failure was defined as rejection or death). The overall 5-year survival rates for patients receiving bone marrow and cord blood from unrelated donors were both 80.0%. Based on univariate analysis, the factors associated with poor survival were: transplantation from an HLA-mismatched related donor, patient age of more than 5 years at the time of transplantation, and a conditioning regimen other than busulfan and cyclophosphamide (BU-CY) or busulfan, cyclophosphamide and antithymocyte globulin (BU-CY-ATG). In a multivariate analysis, a conditioning regimen other than BU-CY and BU-CY-ATG was the only independent factor associated with transplantation failure. Given the improved outcome for WAS patients following transplantation from an unrelated donor, we conclude that patients with WAS should receive SCT as soon as possible after diagnosis.
authors.affiliation: Department of Paediatrics, Hokkaido University Graduate School of Medicine, Sapporo, Japan. r-koba@med.hokudai.ac.jp
authors.forename: Ryoji
authors.initials: R
authors.lastname: Kobayashi
authors.forename: Tadashi
authors.initials: T
authors.lastname: Ariga
authors.forename: Shigeaki
authors.initials: S
authors.lastname: Nonoyama
authors.forename: Hirokazu
authors.initials: H
authors.lastname: Kanegane
authors.forename: Shigeru
authors.initials: S
authors.lastname: Tsuchiya
authors.forename: Tomohiro
authors.initials: T
authors.lastname: Morio
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Yoshihisa
authors.initials: Y
authors.lastname: Nagatoshi
authors.forename: Keisei
authors.initials: K
authors.lastname: Kawa
authors.forename: Ken
authors.initials: K
authors.lastname: Tabuchi
authors.forename: Masahiro
authors.initials: M
authors.lastname: Tsuchida
authors.forename: Toshio
authors.initials: T
authors.lastname: Miyawaki
authors.forename: Shunichi
authors.initials: S
authors.lastname: Kato
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000379:methods; D002066:Busulfan / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D046528:Chimerism; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods; D003520:Cyclophosphamide / Q000627:therapeutic use; D006084:Graft Rejection / Q000276:immunology; D006085:Graft Survival / Q000276:immunology; D006680:HLA Antigens / Q000276:immunology; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D011183:Postoperative Complications; D033581:Stem Cell Transplantation / Q000379:methods*; D016896:Treatment Outcome; D014923:Wiskott-Aldrich Syndrome / Q000276:immunology / Q000401:mortality / Q000601:surgery*
chemical_list: D000961:Antilymphocyte Serum; D006680:HLA Antigens; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D002066:Busulfan