1 The transplant was carried out based on principles established during years of preclinical experimentation, including the cyclophosphamide (CY)-conditioning regimen, the importance of HLA matching between marrow donor and recipient, and the need for postgrafting immunosuppressive drugs to control graft-versus-host disease (GVHD). That year saw the first successful marrow transplant from an HLA-identical sibling donor. The 2 major current competing treatment strategies for severe aplastic anemia, allogeneic bone marrow transplantation (BMT) and immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG), date back to 1970. Implementation of this first-line treatment strategy will provide patients with severe aplastic anemia the best chance of long-term disease-free survival. In contrast, marrow transplantation is associated with high cure rates of aplastic anemia and a relatively low risk for graft-versus-host disease, with many patients now living for decades without the risk for disease recurrence or the development of clonal disorders. We make this recommendation because of the long-term persistent risk for disease relapse and secondary myelodysplastic syndrome or acute myeloid leukemia with the use of nontransplant IST for patients with aplastic anemia. Each of these donor marrow sources may be preferable to nontransplant IST. The priority order of donor source for bone marrow transplantation is: (1) HLA-identical sibling, (2) HLA-matched unrelated donor, and (3) HLA-haploidentical donor if an HLA-matched unrelated donor is not rapidly available. At the time of diagnosis, before beginning treatment, HLA typing should be done to identify a marrow donor among family members or in the unrelated donor registries, and a marrow transplant should be considered first-line therapy. Based on recent data, we argue that guidelines for the initial treatment of patients with newly diagnosed severe aplastic anemia require revision. This review will summarize the key areas of progress in the use of allogeneic hematopoietic cell transplantation and nontransplant immunosuppressive therapy (IST) for the treatment of aplastic anemia and then summarize the recommendations for first-line treatment. Treatment of severe aplastic anemia has improved significantly over the past 4 decades.
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