Clinical course in 28 unselected patients with aplastic anaemia treated with anabolic steroids

Twenty-eight unselected patients with histologically proven aplastic anaemia were electively treated with anabolic steroids (75-150 mg orally q.d.) Additional supportive treatment with blood cell components and antibiotics was given if indicated. Response to therapy was defined as favourable if after 3 months of anabolic therapy overt bleeding tendency had disappeared, there was no need for transfusion therapy, a spontaneous increase of haemoglobin had occurred of greater than 3 g/dl above the initial level, and a platelet rise of twofold the initial count (up to at least greater than 30 x 10(9) /L) had occurred. Of 22 patients evaluable for the results of long-term (greater than 3 months) anabolic treatment, six showed a partial response and eleven responded favourably. These 11 are all alive at the end of the study. Five of these patients proved to be anabolic steroid-dependent. The 50% actuarial survival is approximately 4 years after diagnosis, which compares favourably with the best published results from bone marrow transplantation for aplastic anaemia. It is concluded that anabolic therapy in aplastic anaemia should be tried for 2-3 months before the bone marrow transplantation or immunosuppressive therapy is taken into consideration.