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

Am J Med. 2004 May 1;116(9):590-4. doi: 10.1016/j.amjmed.2003.12.024.


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).

Publication types

  • Clinical Trial

MeSH terms

  • Adrenal Cortex Hormones
  • Adult
  • Aged
  • Aged, 80 and over
  • Chronic Disease
  • Contraindications
  • Danazol / adverse effects
  • Danazol / therapeutic use*
  • Disease-Free Survival
  • Estrogen Antagonists / adverse effects
  • Estrogen Antagonists / therapeutic use*
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Prospective Studies
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Purpura, Thrombocytopenic, Idiopathic / mortality
  • Splenectomy
  • Time Factors


  • Adrenal Cortex Hormones
  • Estrogen Antagonists
  • Danazol