Androgen therapy of aplastic anaemia

The current published reports have indicated that the young patient with aplastic anaemia who has a compatible marrow donor can obtain a successful marrow graft, especially if sex matched and with a past record of little or no transfusion therapy. Despite these encouraging results, only a small number of patients will have such donors available. Immunosuppressive therapy has been considered as an alternative, but this treatment has high risk in older patients. Past studies with androgen therapy have reported a response rate at one year similar to the current recovery rates with bone marrow transplantation. However, contemporary reports have indicated a marked decrease in the recovery rate following androgens, and some of these comparisons may be related to differences in supportive transfusion therapy. In part, the decreased rates may be related to an inadequate evaluation of residual marrow function in the aplastic patient. Patients to be treated with androgens always should have a physiological evaluation of residual erythropoietic committed bone marrow cells. In the absence of such an erythropoietic nidus, one may anticipate a poor response to any steroid therapy. Supportive blood component transfusions should be provided, especially in the initial three months of androgen treatment. In past studies the majority of patients have received only oral androgens, predominantly oxymetholone. Other androgens may be more effective in a particular patient, and there is an urgent need to develop procedures that define stem cell receptors for specific testosterone preparations. Current investigations have indicated that the 5 beta steroid metabolites of testosterone are haematopoietic without the complication of virilization. It is anticipated that a variety of these metabolites can be prepared for evaluation in the patient with aplasia. While there is continuing evaluation of the immune responses and suppressive therapies the clinician should continue to treat the aplastic patient with vigorous supportive transfusion therapy and different androgens for comparative evaluation.