Treatment of Aplastic Anemia

There is general agreement that in children and adolescents with an HLA-identical or syngeneic sibling, bone-marrow transplantation (BMT) should be performed without delay. More controversial are young-to-middle-aged adults with an HLA-identical sibling. Because of comparable survival rates, some centers advocate BMT; others advocate immunosuppression as primary treatment. BMT cures severe aplastic anemia (SAA), but has a higher early mortality. Immunosuppression usually induces hemopoietic improvement and has hardly any early mortality. However, there are late problems. After immunosuppression, there may be relapse and clonal hematopoietic disease. After BMT, there may be late graft failure, severe or even fatal chronic graft-versus-host-disease (GVHD). Both procedures are followed by a slight increase in second malignancies. Retrospective studies show no significant differences in long-term survival between the two treatment modalities. In patients over the age of 40, immunosuppression is favored by most centers because in this age group there is a very high transplant-related mortality with BMT. Androgens and hematopoietic growth factors have hardly any role in therapy of SAA. Splenectomy helps some patients with severe thrombocytopenia and problems with supportive care, but does not improve the overall prognosis. BMT with unrelated donors has usually been performed in late stages of the disease with disappointing results.