Immunosuppressive treatment of acquired aplastic anemia and immune-mediated bone marrow failure syndromes.

Modern therapeutic strategies for the treatment of acquired aplastic anemia are based on the current understanding of its pathophysiology as well as empiric observations. Most cases of aplastic anemia appear to be the result of immune-mediated destruction of hematopoietic cells, which can be approached by stem cell transplantation in younger patients with appropriate histocompatible donors or by immunosuppression to reduce T-cell activity. Popular treatment regimens combine antithymocyte globulin with cyclosporine. Although a majority of patients respond with improved blood counts and achieve transfusion-independence, late clonal complications of myelodysplasia and cytogenetic abnormalities occur in a substantial minority of cases. Additionally, there is no clear algorithm for the treatment of refractory disease. Newer methods of treatment, including high-dose cyclophosphamide and the development of potentially tolerizing combinations of drugs. are under study. Effective therapies for aplastic anemia might also be applied to other T-cell mediated, organ-specific human diseases.