Cyclosporine Therapy for Acquired Aplastic Anemia: Predictive Factors for the Response and Long-term Prognosis
Cyclosporine Therapy for Acquired Aplastic Anemia: Predictive Factors for the Response and Long-term Prognosis
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DOI：
10.1532/ijh97.06156
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发表时间：
2007-04
影响因子：
2.1
通讯作者：
H. Yamazaki;C. Sugimori;T. Chuhjo;S. Nakao
中科院分区：
文献类型：
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作者：
H. Yamazaki;C. Sugimori;T. Chuhjo;S. Nakao
虽然环孢素(CsA)是治疗获得性再生障碍性贫血(AA)的关键药物，但CsA与CsA单药治疗的作用尚不清楚。为确定环孢素A(CsA)治疗再生障碍性贫血(AA)的疗效，对38例AA患者单用CsA治疗，并进行6个月~16年的随访。20名患者(53%)在开始接受CsA治疗后1年内获得部分或完全缓解。网织红细胞数在2个月内增加的16例患者中有13例(81%)获得缓解，而网织红细胞数未增加的患者有效率仅为32%。5年总生存率和无失败生存率分别为91%和37%。这些数据表明，在大约40%的再生障碍性贫血患者中，单用环孢素A可以实现持续缓解，而引发继发性克隆性疾病的可能性很低。鉴于CsA的毒性低，而且CsA的疗效可以在治疗后2个月内判断，CsA可能是不需要输血的AA患者的门诊首选药物。
Although cyclosporine (CsA) is a key drug in the treatment of acquired aplastic anemia (AA), the role of single-agent therapy with CsA remains unclear. To determine the efficacy of CsA in the treatment of AA, we treated 38 AA patients with CsA alone and followed up the patients for 6 months to 16 years. Twenty patients (53%) achieved either a partial or complete remission within 1 year of starting CsA therapy. Thirteen (81%) of 16 patients who showed an increase in the reticulocyte count of >20 x 109/L within 2 months achieved remission, whereas the response rate was only 32% in patients who failed to show such an increase in the reticulocyte count. The actuarial overall survival and failure-free survival rates at 5 years were 91% and 37%, respectively. These data indicate that CsA alone can achieve a sustained remission in approximately 40% of AA patients, with a low probability of inducing secondary clonal diseases. Given its low toxicity and because the effectiveness of CsA can be judged within 2 months of therapy, CsA may be the first drug of choice at outpatient clinics for AA patients not requiring transfusions.