The Effect of Danazol in Primary Immune Thrombocytopenia: An Analysis of a Large Cohort From a Single Center in China.

Aim: This study assessed the long-term benefits and side effects of low-to-medium dose of danazol therapy in primary immune thrombocytopenia (ITP). Methods: The retrospective analysis included 319 patients with ITP who accepted danazol therapy. Patients accepted danazol alone or in combination with glucocorticoids. Clinical outcome and tolerance were assessed in all patients. Results: Among patients with persistent or chronic ITP, the overall response rate of danazol therapy was 65.0%. Sixty-five (63.1%) of the 103 patients reached remission with danazol alone, and 93 (48.7%) of the 191 patients who accepted combination therapies acquired remission and discontinued glucocorticoids successfully. Age and previous treatments were 2 risk factors for response rate. In newly diagnosed patients with ITP, the response rate and median response time did not differ significantly with or without danazol. However, the relapse rate was significantly lower in patients administered danazol combined with glucocorticoids than those accepted glucocorticoids alone. Totally, 21.1% of the patients experienced mild or moderate side effects, and 1.2% of the patients discontinued treatment due to intolerable side effects. Conclusion: Low-to-medium dosage of danazol is better tolerated and effective in patients with ITP, even in those refractory to other treatments. Combination of danazol and glucocorticoids for initial treatment may decrease relapse rates to achieve well-tolerated long-term remission.