Complete Correction of the Wiskott-Aldrich Syndrome by Allogeneic Bone-Marrow Transplantation

Authors:
  • RPRobertson Parkman
    Robertson Parkman
  • JRJoel Rappeport
    Joel Rappeport
  • RGRaif Geha
    Raif Geha
  • JBJames Belli
    James Belli
  • RCRobert Cassady
    Robert Cassady
  • RLRaphael Levey
    Raphael Levey
  • DNDavid G. Nathan
    David G. Nathan
  • FRFred S. Rosen
    Fred S. Rosen
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Abstract
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. (N Engl J Med 298:921–927, 1978)

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