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Unrelated partially matched peripheral blood stem cell transplantation with highly purified CD34+ cells in a child with Wiskott–Aldrich syndrome

Abstract

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 × 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. Bone Marrow Transplantation (2000) 26, 235–237.

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Schwinger, W., Urban, C., Lackner, H. et al. Unrelated partially matched peripheral blood stem cell transplantation with highly purified CD34+ cells in a child with Wiskott–Aldrich syndrome. Bone Marrow Transplant 26, 235–237 (2000). https://doi.org/10.1038/sj.bmt.1702473

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