Androgen therapy in myelodysplastic syndromes with thrombocytopenia: a report on 20 cases

Twenty patients with myelodysplastic syndromes (MDS) and (i) platelets < 50 x 10(9)/l and (ii) bone marrow blasts < or = 10% were treated with androgen therapy (fluoxymesterone at 1 mg/kg/d: seven patients; danazol at 600 mg/d: 13 patients) for at least 3 months. 11 of them (55%) had an increase in platelet counts by at least 30 x 10(9)/l and a disappearance of bleeding symptoms was seen in 6/6 patients with initial bleeding. A response with neutrophil counts (six cases) or haemoglobin levels (five cases) was less often seen. Treatment was continued for 3+ to 27 months in responders (the dose being reduced by 50% after 6 months). Seven patients on maintenance treatment were still responding. Another patient died while he was still responding, and the remaining three patients relapsed after discontinuation (two cases) and dose reduction to 50% (one case) of the androgen used. Side-effects of treatment were moderate. In our experience, androgen therapy can be useful in patients with 'low risks' MDS (i.e. with marrow blasts < or = 10%) and severe thrombocytopenia, especially because no growth factor regularly active on platelets is currently available.