From May 1971 through December 1981, 81 children (22 months to 17 years of age) received allogeneic bone marrow grafts for severe aplastic anemia. All donors were HLA-identical family members. Fifty-seven of the 81 (70%) are still alive. Twenty-three untransfused patients were conditioned with cyclophosphamide, 50 mg/kg/d, for four days, and 19 (83%) have survived from 5 to 12 years. All 58 transfused patients were conditioned with cyclophosphamide, 50 mg/kg/d, for four days, 11 received additional immunosuppression, and 19 received posttransplantation donor buffy coat cells. Thirty-eight (65%) have survived from 3 to 13 years ( P = .1). In a multivariate analysis, the only factor significantly associated with increased survival among patients with sustained grafts was the absence of significant graft v host disease ( P .0001). The factors significantly related to increased rejection were low bone marrow cell dose ( P .05) and positive relative response in mixed leukocyte culture ( P .0001), but the addition of buffy coat cells did not significantly influence graft rejection. The development of grades II to IV acute graft v host disease was associated with random donor platelet refractoriness ( P .05) and donor/recipient sex differences ( P .05). Patients at highest risk for chronic graft v host disease were those patients who developed significant acute graft v host disease ( P .01) and who received buffy coat infusions ( P .025). All patients who were untransfused had a negative relative response and were not refractory to random donor platelets.
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