Hospices Civils de Lyon, Hôpital Louis Pradel, Service de pneumologie - Centre de référence national des maladies pulmonaires rares, Université Claude Bernard Lyon 1, Lyon, France.
Respir Res. 2013;14 Suppl 1(Suppl 1):S5. doi: 10.1186/1465-9921-14-S1-S5. Epub 2013 Apr 16.
Idiopathic pulmonary fibrosis (IPF) is a progressive disease, with a median survival time of 2-5 years. The search for effective treatment has involved numerous clinical trials of investigational agents without significant success. However, in 2011, pirfenidone was the first drug to be approved for the treatment of IPF in Europe. Four key clinical trials supported the efficacy and tolerability of pirfenidone. In the two recently published Phase III CAPACITY trials evaluating pirfenidone (studies 004 and 006), patients with mild-to-moderate IPF were treated with pirfenidone or placebo. Study 004 and pooled analysis of primary endpoint data from both studies showed that pirfenidone significantly reduced decline in percent-predicted forced vital capacity (FVC) compared with placebo (p<0.005). Evidence of beneficial effects of pirfenidone treatment was also observed with regard to several secondary endpoints. Pirfenidone was generally well tolerated, with the most common side effects being gastrointestinal and photosensitivity. Data from the RECAP extension phase of the CAPACITY studies, where patients were treated with pirfenidone for up to three years, further support the manageable tolerability profile of pirfenidone. The efficacy data, coupled with long-term safety data, provide further evidence of a clinically-meaningful treatment effect with pirfenidone in patients with IPF.
特发性肺纤维化（IPF）是一种进行性疾病，中位生存时间为 2-5 年。寻找有效的治疗方法涉及了许多针对研究药物的临床试验，但均未取得显著成功。然而，2011 年，吡非尼酮成为第一个在欧洲被批准用于治疗 IPF 的药物。四项关键临床试验支持了吡非尼酮的疗效和耐受性。在最近发表的两项评估吡非尼酮的 III 期 CAPACITY 试验（研究 004 和 006）中，轻度至中度 IPF 患者接受吡非尼酮或安慰剂治疗。研究 004 和两项研究主要终点数据的汇总分析显示，与安慰剂相比，吡非尼酮可显著降低预测用力肺活量（FVC）的百分比下降（p<0.005）。吡非尼酮治疗的有益效果也在一些次要终点中得到了观察。吡非尼酮总体上具有良好的耐受性，最常见的副作用是胃肠道和光敏性。来自 CAPACITY 研究的 RECAP 扩展阶段的数据进一步支持了吡非尼酮可管理的耐受性特征，这些患者接受了长达三年的吡非尼酮治疗。疗效数据加上长期安全性数据，为吡非尼酮在 IPF 患者中的临床有意义的治疗效果提供了进一步证据。
