Reconstitution of normal megakaryocytopoiesis and immunologic functions in Wiskott-Aldrich syndrome by marrow transplantation following myeloablation and immunosuppression with busulfan and cyclophosphamide

Blood. 1981 Apr;57(4):692-6.

Abstract

Three patients with Wiskott-Aldrich syndrome received transplants of marrow from their HLA-A, B, C, D identical siblings after myeloablation with busulfan, 2 mg/kg/day x 4 days, followed by immunosuppression with cyclophosphamide, 50 mg/kg/day x 4. Sustained engraftment of lymphoid and hematopoietic elements was documented in each case. Platelet counts in excess of 100,000/cu mm were restored 20--50 days posttransplant and remain in the normal range 6--12 mo later. Platelets exhibit normal size and in vitro aggregation. The patients produce isoagglutinins and antibodies to other polysaccharides. The use of busulfan in moderate dosages as a myeloablative agent, coupled with cyclophosphamide, may offer an improved alternative to the use of lethal total body irradiation as a preparative regimen for complete correction of Wiskott-Aldrich syndrome by marrow transplantation.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Bone Marrow Transplantation*
  • Busulfan / therapeutic use
  • Child
  • Child, Preschool
  • Chromosomes, Human, 13-15
  • Cyclophosphamide / therapeutic use
  • Dysgammaglobulinemia / immunology
  • Hematopoiesis*
  • Humans
  • Immunoglobulin M / deficiency
  • Immunosuppressive Agents
  • Male
  • Megakaryocytes / cytology*
  • Thrombocytopenia / complications
  • Wiskott-Aldrich Syndrome / drug therapy
  • Wiskott-Aldrich Syndrome / immunology*

Substances

  • Immunoglobulin M
  • Immunosuppressive Agents
  • Cyclophosphamide
  • Busulfan