Bone marrow transplantation. II. Use for aplastic anemia, hereditary diseases, and hemoglobinopathies

Severe aplastic anemia has had a uniformly poor prognosis and is another area in which bone marrow transplantation has been performed in large numbers of patients. Graft rejection has proven to be a significant problem which has been successfully reduced by a variety of modalities which will be discussed. Over the past decade, clinical results have continued to improve, mostly as a result of the reduction in the rate of graft rejection. Immunotherapy with antithymocyte globulin, however, has recently been shown to be at least partially effective in this same group of patients. The clinical results and its role in the treatment of this disorder will also be discussed. A large number of congenital and hereditary disorders have been also successfully treated with bone marrow transplantation and, recently, severe beta thalassemia has been approached with this modality in a single patient. This is obviously an extremely large and heterogeneous group of disorders and few patients in any single category have been transplanted.