PEDIATRICS Vol. 77 No. 2 February 1986, pp. 179-186
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Bone Marrow Transplantation Experience for Children With Aplastic Anemia

Jean E. Sanders MD1, John Whitehead PhD1, Rainer Storb MD1, C. Dean Buckner MD1, Reginald A. Clift FIMLS1, Eric Mickelson 1, Frederick R. Appelbaum MD1, William I. Bensinger MD1, Patricia S. Stewart MD1, Kristine Doney MD1, Keith Sullivan MD1, H. Joachim Deeg MD1, Robert P. Witherspoon MD1, and E. Donnall Thomas MD1

1 From the Departments of Medicine and Pediatrics, University of Washington School of Medicine and the Division of Oncology, The Fred Hutchinson Cancer Research Center, Seattle

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.

Key Words: bone marrow transplantation • aplastic anemia

Submitted on March 21, 1985
Accepted on June 10, 1985




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