Androstane therapy to treat aplastic anaemia in adults: an uncontrolled pilot study

During the past 9 years, 43 adult patients with severe aplastic anaemia have been treated with a combination of 3 alpha etiocholanolone and nandrolone decanoate in an uncontrolled pilot study. Eleven patients were considered acute and the remainder as chronic severe aplasia. 50% (22 patients) had a haematopoietic recovery and 40% have had a sustained remission varying from 1.5 to 8 years. Three patients who did not respond to 3 alpha etiocholanolone had a haematologic response when treated with the isomer 3 beta etiocholanolone. The recovery did not appear to be associated with age or duration of marrow aplasia. In view of the reproducible chemical structure of these androstanes, we believe this group of steroids should be evaluated further in the treatment of severe aplastic anaemia.