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  • Title: Bone marrow transplantation in pediatric patients with severe aplastic anemia: cyclophosphamide and anti-thymocyte globulin conditioning followed by recombinant human granulocyte-macrophage colony stimulating factor.
    Author: Bunin N, Leahey A, Kamani N, August C.
    Journal: J Pediatr Hematol Oncol; 1996 Feb; 18(1):68-71. PubMed ID: 8556374.
    Abstract:
    PURPOSE: Graft rejection remains a serious problem in patients transplanted for severe aplastic anemia. Although additional immunosuppression with irradiation may decrease graft failure, significant sequelae may ensue. We evaluated a nonirradiation containing conditioning regimen for children with severe aplastic anemia with matched sibling donors utilizing cyclophosphamide and anti-thymocyte globulin (ATG). To accelerate myeloid recovery, GM-CSF was used posttransplant. PATIENTS AND METHODS: Twelve patients, with a median age of 3 years underwent BMT from HLA identical sibling (n = 11) or syngeneic (n = 1) donors. Conditioning was cyclophosphamide 50 mg/kg x 4 days and anti-thymocyte globulin 30 mg/kg x 3 days. GM-CSF was administered at 10 micrograms/kg until a neutrophil count of 1,000 was achieved. Cyclosporine alone was used for graft-versus-host disease prophylaxis. RESULTS: All patients achieved durable engraftment at follow-up of 5-51 + months, with the exception of the identical twin. Median time to neutrophil counts > 200/microliters, 500/microliters, and 1,000/microliters were 12, 13, and 15 days, respectively. Acute GVHD of less than or equal to grade II occurred in four patients; one patient had grade III. This has resolved in all but one. CONCLUSION: The nonradiation conditioning regimen of cyclophosphamide/ATG appears to achieve durable engraftment in transfused children with matched sibling donors. GM-CSF may accelerate myeloid recovery without exacerbating GVHD, but its contribution to allogeneic transplant required further study.
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