Bone Marrow Transplantation Experience for Children With Aplastic Anemia.

Abstract. 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 bully 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 upsilon 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 upsilon 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 upsilon host disease were those patients who developed significant acute graft upsilon host disease (P < .01) and who received bully coat infusions (P < .025). All patients who were untransfused had a negative relative response and were not refractory to random donor platelets. [ABSTRACT FROM AUTHOR]