Treatment of acquired severe aplastic anemia

The management of patients with acquired severe aplastic anemia has been directed at intensive supportive care and restoration of normal marrow function. Bone marrow transplantation remains the treatment of choice with approximately 70% long-term survivors, after related histocompatible transplants. More intensive conditioning regimens, using cyclophosphamide with some form of radiation, have decreased the incidence of graft rejection to less than 5%. Immunosuppression with antilymphocyte (ALG) or antithymocyte (ATG) globulin may induce complete or partial hematologic recovery in 35-70% of patients, with 1- and 2-year survival rates in the range of 50-70% and 45-55%, respectively. The role of high-dose corticosteroids, cyclosporine A, and colony-stimulating factors in the management of patients with acquired severe aplastic anemia is less clear and requires further investigation. Finally, androgens, either alone or in combination with other agents, do not seem to have a place in the treatment of severe bone marrow aplasia.