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 unr elated donors is associated with increased risk of graft rejection and graf t-versus-host disease. In an attempt to prevent these problems, a 6-year-ol d boy with Wiskott-Aldrich syndrome lacking a suitable family donor, was tr ansplanted with large numbers of unrelated highly purified CD34(+) peripher al blood stem cells mismatched at one C locus. Conditioning consisted of bu sulfan 16 mg/kg body weight, cyclophosphamide 200 mg/kg body weight and ant ithymocyte globulin 20 mg/kg body weight x 3 days. The boy had a rapid hema topoietic engraftment and showed immunologic reconstitution by day +92, Alt hough 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 af ter transplantation.