A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia.

One hundred ten patients with severe aplastic anemia were entered into a prospective study. Forty-seven patients who had HIA-identical siblings were treated with marrow transplantation. Sixty-three patients without marrow donors were randomized to supportive care with oral androgen (27 patients). intramuscular androgen (23 patients). or no androgen (1 3 patients). The distributions of the survival times for the patients in the three nontransplantation arms of the study were not different. Transplanted patients had significantly better survival than nontransplanted patients (p 0.0002). Evaluation of prognostic factors for nontransplanted patients showed a better prognosis for patients with unknown etiology than for those with a probable etiology (p = 0.01 ). Also, patients with larger mean red blood cell volumes tended to live longer (p = 0.03). For patients given marrow transplants. significant prognostic fators were age (p 0.01 5) and sex (p 0.027). with better survival for younger male patients. Twenty-seven transplant recipients (57 % ) are alive; 5 have severe chronic graft-versus-host disease, and 22 (47 %) are living and well. Sixteen nontransplanted patients (25 % ) are alive; 3 continue to have severe disease, and 13 (21 %) are clinically well and no longer require transfusion support. As used in this study, androgens were of no value in the treatment of patients with severe aplastic anemia. If a suitable donor is available, marrow transplantation is clearly superior to conventional management. at least in younger patients. A PLASTIC ANEMIA is the name commonly used to describe the syndrome of marrow hypoplasia with pancytopenia. This disorder is associated with a variety of etiologic agents and, most probably, with several different pathogenic mechanisms. A wide range of therapeutic modalities has been employed in uncontrolled studies of treatment of patients with aplastic anemia, most notably corticosteroids, androgens, and splenectomy. The effectiveness of therapy for aplastic anemia is difficult to evaluate because of the variability in the course of the disease and because a few patients with severe aplastic anemia may experience spontaneous complete recovery. In recent years marrow transplantation has been introduced as a therapeutic option for the patient with severe aplastic anemia who has a histocompatible donor.