Danazol therapy in patients with chronic idiopathic thrombocytopenic purpura: long-term results.

Background: Adults with chronic idiopathic thrombocytopenic purpura (ITP) in whom standard-dose corticosteroids and splenectomy have failed or who have contraindications to these therapies often require further treatment for life-threatening thrombocytopenia or bleeding. We studied whether danazol, an attenuated androgen, is useful in this setting.

Methods: To assess both clinical outcome and tolerance issues, 57 patients who had refractory chronic ITP (n = 27) or who had contraindications to splenectomy or corticosteroids or who refused these therapeutic options (n = 30) were studied.

Results: Thirty-eight patients experienced a partial or complete response to therapy (67%), among whom 27 (46%) remained in remission at a median (+/- SD) of 119 +/- 45 months. Treatment tolerance was acceptable, although severe adverse events were reported in 9 patients (16%).

Conclusion: Our findings suggest that danazol therapy may be beneficial in the management of refractory chronic ITP or when there are contraindications to splenectomy or corticosteroids (or both).