Danazol treatment of idiopathic myelofibrosis with severe anemia

Background and Objectives. Severe anemia is an important problem in patient s with idiopathic myelofibrosis (IM), When other therapeutic measures are u nsuccessful or not applicable, 40-50% favorable responses are obtained with androgen therapy. Oxymetholone Is the drug usually employed, but good resu lts have also been reported with danazol, although the experience is limite d to a few patients. The aim of the present study was to evaluate the effec t of danazol on the anemia of IM. Design and Methods, Seven out of 22 consecutive IM patients were eligible f or danazol treatment because of severe anemia not treatable with (four case s) or refractory to (three cases) other therapies. Danazol (600-800 mg/day) was given orally for six months and thereafter progressively tapered to th e minimum effective dose In responding patients or discontinued in non-resp onders. Complete response was considered cessation of trans- fusion require ments with normalization of hemoglobin (Hb) values; partial response was de fined as a > 30% reduction in transfusional needs or an increase > 10 g/L I n the Hb. The effect on platelet counts was also analyzed. Results. One patient splenectomized three years earlier achieved a complete response and three a partial response, giving an overall response rate of 57 %, A significant increase in platelet counts was also observed In three responders. The responses were first seen between three and six months afte r;the start of treatment, which was usually well tolerated. Interpretation and Conclusions. Danazol, given at an appropriate dosage for a sufficient time, is an effective treatment for a substantial proportion of IM patients with severe anemia without marked splenomegaly or who have b een previously splenectomized. (C) 2000, Ferrata Storti Foundation.