Practical aspects in the diagnosis and management of aplastic anemia.

Aplastic anemia may result from several pathogenic mechanisms, the most common is idiopathic. The current definitive treatments for aplastic anemia are bone marrow transplantation (BMT) or immunosuppressive (IS) therapy. The benefits of each are comparable. However, certain subsets of patients derive superior benefit from one or the other. Bone marrow transplantation is the initial treatment of choice for young patients (< 20 years old). It results in the complete reconstitution of hematopoiesis, whereas autologous hematopoietic remissions after IS therapy are more susceptible to relapse. Survival rates after BMT, in patients between the ages of 20 and 40, are comparable to those reported for IS therapy. Better survival rates after BMT have been achieved with improved conditioning regimens and graft-versus-host disease prophylaxis. For patients older than 40, the treatment of choice is IS. Long-term complications of IS therapy include recurrence and development of clonal myeloid disorders. Long-term complications after BMT include graft-versus-host disease and secondary neoplasms. The IS regimen includes the combination of antithymocyte globulin and cyclosporin A. The addition of growth factor to the IS regimen seems promising; however, their use on their own is not recommended. Androgens have been shown to be inferior in the treatment of aplastic anemia. The role of BMT from an unrelated donor is being investigated.