Unrelated partially matched peripheral blood stem cell transplantation with highly purified CD34+ cells in a child with Wiskott-Aldrich syndrome.
Stem cell transplantation is the only curative approach to the treatment of Wiskott-Aldrich syndrome. However, using grafts from partially matched unrelated donors is associated with increased risk of graft rejection and graft-versus-host disease. In an attempt to prevent these problems, a 6-year-old boy with Wiskott-Aldrich syndrome lacking a suitable family donor, was transplanted with large numbers of unrelated highly purified CD34+ peripheral blood stem cells mismatched at one C locus. Conditioning consisted of busulfan 16 mg/kg body weight, cyclophosphamide 200 mg/kg body weight and antithymocyte globulin 20 mg/kg body weight x 3 days. The boy had a rapid hematopoietic engraftment and showed immunologic reconstitution by day +92. Although he did not receive prophylactic immunosuppression he did not develop any graft-versus-host disease and is well and alive up to now, 25 months after transplantation.
Schwinger
W
W
Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Graz, Austria.
Urban
C
C
Lackner
H
H
Kerbl
R
R
Benesch
M
M
Dornbusch
H J
HJ
Sovinz
P
P
Schumm
M
M
Handgretinger
R
R
D018952:Antigens, CD34 / Q000032:analysis*; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006648:Histocompatibility / Q000276:immunology*; D006801:Humans; D007223:Infant; D007937:Leukapheresis; D008297:Male; D013234:Stem Cells / Q000276:immunology*; D014184:Transplantation, Homologous / Q000379:methods; D014923:Wiskott-Aldrich Syndrome / Q000276:immunology / Q000628:therapy*
D018952:Antigens, CD34