Abstract
Two patients with Wiskott-Aldrich syndrome have had the establishment of both normal immune and hemopoietic function following allogeneic bone marrow transplantation from HLA-A, -B, and -D identical sibling donors. Previous attempts by others had achieved only partial engraftment. The patients were successfully transplanted only after preparation with rabbit anti-human thymocyte serum (ATS) and total body irradiation (TBI). One patient was initially transplanted after preparation with high dose cytosine arabinoside (150 mg/m2 × 7 days) and cyclophosphamide (50 mg/kg × 4 days). Only a T lymphocyte graft was achieved and the patient recovered his own hematopoietic function. After 6 months the recipient's T lymphocytes returned. The patient was successfully re-transplanted after ATS and TBI (880R) preparation. A second patient was successfully transplanted after initial ATS and TBI (850R) preparation. Both patients have normal platelet counts and function. The immune systems of both patients are of donor origin. Neither patient has had any acute nor chronic graft versus host disease. The successful treatment of the Wiskott-Aldrich syndrome may be a model for the treatment of other genetically determined bone marrow disorders. (Supported by NIH grants RR-128, AI-05877 and Ca-13472.)
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Parkman, R., Rappeport, J., Geha, R. et al. 725 SUCCESSFUL CORRECTION OF A BONE MARROW STEM CELL DEFECT (WISKOTT-ALDRICH SYNDROME) BY ALLOGENEIC MARROW TRANSPLANTATION. Pediatr Res 12 (Suppl 4), 484 (1978). https://doi.org/10.1203/00006450-197804001-00730
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DOI: https://doi.org/10.1203/00006450-197804001-00730