Danazol therapy for aplastic anemia refractory to immunosuppressive therapy
Danazol therapy for aplastic anemia refractory to immunosuppressive therapy
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DOI：
10.1002/ajh.21118
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发表时间：
2008-05-01
影响因子：
12.8
通讯作者：
Nakao, Shinji
中科院分区：
文献类型：
--
作者：
Chuhjo, Tatsuya;Yamazaki, Hirohito;Nakao, Shinji
尽管有达那唑治疗再生障碍性贫血（AA）疗效的轶事报道，但尚未有系统研究阐明其疗效和毒性。因此，我们在一项前瞻性临床试验中评估了达那唑治疗免疫抑制治疗 (IST) 难治性 AA 患者以及 IST 后复发患者的疗效。 16 名患者（12 名男性和 4 名女性；6 名重度病例和 10 名中度病例）每天接受 300 mg 达那唑治疗，持续 12 周。所有患者均完成治疗期，未发生严重毒性反应。 3 名女性患者获得部分缓解，而 12 名男性患者中只有 2 人实现部分缓解。没有一个反应者对之前的 IST 表现出反应，也没有表现出阵发性睡眠性血红蛋白尿 (PNH) 型细胞百分比的增加，而众所周知，PNH 型细胞是对 IST 良好反应的标志。这些研究结果表明，达那唑对部分 AA 患者有效，尤其是对 IST 难治性 AA 女性患者。
Although there are anecdotal reports of the efficacy of danazol in the treatment of aplastic anemia (AA), there has been no systematic study to clarify its efficacy and toxicity. Therefore, we assessed the efficacy of danazol for treatment of patients with AA refractory to immunosuppressive therapy (IST) and those who relapsed after IST, in a prospective clinical trial. Sixteen patients (12 males and four females; six severe cases and 10 moderate cases) were treated with 300 mg of danazol daily for 12 weeks. All patients completed the treatment period without occurrence of severe toxicity. Three female patients achieved partial remission, whereas only two of the 12 male patients did so. None of the responders had shown a response to previous IST or an increase in the percentage of paroxysmal nocturnal hemoglobinuria (PNH)-type cells which are known to be a marker for a good response to IST. These findings indicate that danazol is effective for a subset of AA patients, and particularly for female patients with AA refractory to IST.