[Long-term survival of patients with bone marrow aplasia treated with androgen therapy. 166 cases]

166 aplastic anaemia patients involved in a cooperative study including androgens were followed up for more than 2 years from the beginning of treatment. The mortality rate from aplasia was low (20% of all cases) after 2 years and nil beyond 5 years. The patients who died were either those who failed to improve but survived 2 years or those who late untreated relapse. Five deaths were due to acute leukaemia, but in 3 of these the diagnosis of aplastic anaemia was retrospectively doubtful. Long-term adverse reactions were rare: 2 cases each of benign hepatoma and peliosis; jaundice was frequent, but no cirrhosis was observed. Survivors who had been treated before puberty reached normal weight and height. Six normal pregnancies occurred. Paroxysmal nocturnal haemoglobinuria was noted in 8 patients with uncontrolled disease. After androgen therapy was discontinued 50% of the patients relapsed with equally or less severe symptoms. Relapses were more frequent when androgens were abruptly withdrawn or in cases with incomplete initial improvement. Most relapses responded to androgen treatment. More than 25% of the patients are still androgen-dependent. With or without maintenance androgen therapy, long-term improvement is incomplete in most cases, which suggests residual bone marrow deficiency, as seen in rodents with experimentally induced aplastic anaemia.