717 MARROW TRANSPLANTATION (MTP) IN WISKOTT-ALDRICH SYNDROME (WAS): T CELL ENGRAFTMENT WITH CYCLOPHOSPHAMIDE (CY), COMPLETE ENGRAFTMENT WITH TOTAL BODY IRRADIATION

Abstract

We studied two patients with WAS who had MTP with marrow from HLA-identical siblings. The first patient received 200 mg/kg CY over 4 days (Bach et al., Lancet 1:1364, 1968). We have followed this patient for 7 years. He has petechiae, bleeding in joints, and severe thrombocytopenia. IgM has remained low; antibody production to some antigens, lymphocyte response to mitogens and in vitro B cell functions are defective. Growth and development have been normal. The second patient received cytosine arabinoside 5 mg/kg/day ×7, 6-thioquanine 4 mg/kg/day ×7, and CY 50 mg/kg/day ×4 prior to MTP. In both patients after MTP, megakaryocytes, red cells, neutrophils, macrophages, and most B cells remained of recipient type while most T cells were of donor type. The second patient was retransplanted after preparation with anti-lymphocyte globulin, 2 ml IV daily x4, Procarbazine, 12.5 mg/kg/day × 3, and 800 R total body irradiation. Ninety days after the 2nd transplant, all nucleated marrow and blood cells, including B and T cells were of donor type; WBC 12,500, platelets 132,000/mm³, hemoglobin 9.8 gms % and reticulocytes 3.5%. No graft-versus-host disease was observed. We conclude that in marrow transplantation for WAS, 200 mg/kg CY may be inadequate preparation. In WAS, T cells may more readily be engrafted than other blood cells. Supported by USPHS GCRC #MO-1-R00749-05.

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