Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone-marrow transplantation.
Two patients with the Wiskott-Aldrich syndrome had complete donor lymphoid and hematopoietic engraftment after successful allogeneic bone-marrow transplantation. One patient had had only a temporary donor T-lymphocyte graft after a previous transplantation, for which he had been prepared with cytarabine and cyclophosphamide; the patient's own T lymphocytes returned six months later. A repeat transplant, for which the patient was prepared with anti-human thymocyte serum, total-body irradiation and procarbazine, resulted in complete donor engraftment. The second patient underwent a successful transplantation after similar preparation, except that procarbazine was omitted. At 11 and five months after transplantation both had normal hematopoiesis and no evidence of graft-versus-host disease. This treatment of the Wiskott-Aldrich syndrome may be a model for the correction of other genetically determined immune and hematologic bone-marrow disorders.
D000961:Antilymphocyte Serum; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D003561:Cytarabine / Q000627:therapeutic use; D006648:Histocompatibility; D006801:Humans; D008297:Male; D011300:Preoperative Care; D011344:Procarbazine / Q000627:therapeutic use; D011830:Radiation Effects; D013601:T-Lymphocytes / Q000276:immunology; D013997:Time Factors; D014184:Transplantation, Homologous; D014923:Wiskott-Aldrich Syndrome / Q000276:immunology / Q000628:therapy*
D000961:Antilymphocyte Serum; D003561:Cytarabine; D011344:Procarbazine; D003520:Cyclophosphamide