Androgen therapy of aplastic anaemia--a prospective study of 352 cases

A prospective study of 352 patients with aplastic anaemia on androgen therapy has been performed. The following main observations have been obtained: The actuarial mortality rate at the 20th month is 52%, half the deaths being observed during the first 3 months; these figures are similar to those previously published, from smaller series of androgen-treated patients, and lower than those of non-androgen-treated cases. Differences in survival and improvement were observed between groups of patients treated for more than 3 months with either alkylated or non-alkylated drugs. Signs of liver damage were observed no matter which was the drug used. Continous improvement can be observed even in the 2nd year of treatment indicating that full-dose androgen therapy should be continued up to 20 months in not fully improved patients. The degree of initial disease activity is a clear prognostic parameter for the mortality in the first quarter of the course. In case of survival of severe cases, improvement can be obtained to the same extent as in milder cases. This stress the need for adequate maintenance therapy in all types of patients. Addition of glucocorticoids harms the prognosis, mainly in most granulocytopenic patients. Glucocorticoids have no effect upon the liver damage induced by androgens.