Maloisel Frédéric, Andrès Emmanuel, Zimmer Jacques, Noel Esther, Zamfir Alina, Koumarianou Argyro, Dufour Patrick
Departments of Hematology and Oncology, Strasbourg, France.
Am J Med. 2004 May 1;116(9):590-4. doi: 10.1016/j.amjmed.2003.12.024.
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.
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.
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%).
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).
对于标准剂量皮质类固醇和脾切除术治疗失败或存在这些治疗禁忌证的慢性特发性血小板减少性紫癜（ITP）成人患者，常需要针对危及生命的血小板减少或出血进行进一步治疗。我们研究了弱雄激素达那唑在此情况下是否有用。
为评估临床结局和耐受性问题，对57例难治性慢性ITP患者（n = 27）、存在脾切除术或皮质类固醇禁忌证的患者（n = 30）或拒绝这些治疗选择的患者进行了研究。
38例患者对治疗有部分或完全反应（67%），其中27例（46%）在中位（±标准差）119 ± 45个月时仍处于缓解状态。治疗耐受性尚可，尽管有9例患者（16%）报告了严重不良事件。
我们的研究结果表明，达那唑治疗可能有助于难治性慢性ITP的管理，或在存在脾切除术或皮质类固醇（或两者）禁忌证时使用。
