Oxymetholone treatment in myelofibrosis

Abstract

In order to study the effect of oxymetholone therapy in advanced myelofibrosis, 11 patients (4 females, 7 males) were given, 3--5 mg per kg body weight, long-term oxymetholone treatment in a prospective multicenter study. Five cases had previously had a diagnosis of polycythemia vera. All patients had anemia initially, 4 leukocytopenia and 10 thrombocytopenia in addition. Hepato-splenomegaly was present in all cases but in varying degree. Five patients required regular blood transfusions before treatment. In 9 of the 15 courses given, there was normalization of the peripheral blood or substantial improvement (better than 3 g hemoglobin/dl or 50 X 10(9) platelets/1) after androgens. Significant effects were noted both on hemoglobin values and platelet counts. The need for blood transfusions ceased completely in all 5 cases. When oxymetholone treatment was reduced or interrupted 4 patients relapsed; 2 of them responded to a renewed course. The red cell counts returned to previous polycythemic values in one patient and another died from acute leukemia. The results of this study suggest that androgens might be of value in advanced cases of myelofibrosis with transfusion-requiring anemia or severe thrombocytopenia.

Danazol therapy for the anemia of myelofibrosis: assessment of efficacy with current criteria of response and long-term results

“…Androgens have long been used to treat the anemia of MF [22,23], with responses ranging from 30 to 60 % of patients. While conventional androgens are associated with important side effects, similar therapeutic results, with less toxicity, have been observed with danazol, a semisynthetic attenuated androgen.…”

Modern management of myelofibrosis

“…The efficacy of testosterone in the anaemia of IM was shown in two reports from the sixties (Kennedy, 1962;Silver et al, 1964) and its main mechanism of action seems to be the stimulation of the bone marrow function (Reilly, 1997). According to the few available studies, androgens, such as nandrolone, fluoxymesterolone, methandrostenolone and oxymetholone, improve the anaemia in 30-60% of patients (Hast et al, 1978;Besa et al, 1982;Brubaker et al, 1982), with an abnormal karyotype and severely compromised haemopoiesis being the factors associated with a low chance of achieving a response (Besa et al, 1982). Side effects include fluid retention, hirsutism, virilization, abnormal liver function tests and, more rarely, liver and prostate tumours.…”

Guideline for the diagnosis and management of myelofibrosis

“…A number of androgens have been trialled including nandrolone (Gardner & Besa, 1977;Besa et al, 1982), testosterone (Silver et al, 1964;Gardner & Besa, 1977;Besa Guideline et al, 1982), fluoxymesterone (Gardner & Besa, 1977;Brubaker et al, 1982), oxymethalone (Alexanian et al, 1972;Gardner & Besa, 1977;Hast et al, 1978), etiocholanolone (Gardner & Besa, 1977) and danazol (Levy et al, 1996;Cervantes et al, 2000Cervantes et al, , 2005. Response rates to androgen therapy are reported as varying, most frequently between 30% and 60%.…”