Abstract
Although the mortality and morbidity of CDG has been reduced by prophylactic antibiotics, certain CDG patients still have a severe course leading to death. A 15 year old white male with CDG had an acute reduction in pulmonary function (70→45% of expected) and it was decided to transplant him with 2.4 × 108 bone marrow cells/kg from his HLA identical sister after preparation with rabbit anti-human thymocyte serum, procarbazine, and total body irradiation (800 R). Prior to transplantation all the patient's peripheral blood granulocytes and bone marrow CFU-C were NBT negative while greater than 98% of the donor cells were NBT positive;after BMT all hematopoietic and lymphoid elements were of donor origin; 100% of the recipients peripheral granulocytes, monocytes and bone marrow CFU-C were NBT positive. The patient developed chronic graft versus host disease (GVHD) and died six months following transplantation of renal failure. CDG, like other genetic disorders of bone marrow function, may be corrected by BMT in selective cases. The transplant related problem of chronic GVHD is the major limitation to the increased use of BMT.
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Rappeport, J., Newburger, P., Goldman, A. et al. 856 CORRECTION OF CHRONIC GRANULOMATOUS DISEASE (CDG) BY BONE MARROW TRANSPLANTATION (BMT). Pediatr Res 15 (Suppl 4), 585 (1981). https://doi.org/10.1203/00006450-198104001-00881
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DOI: https://doi.org/10.1203/00006450-198104001-00881