Double-blind, placebo-controlled trial of pirfenidone in patients with idiopathic pulmonary fibrosis
Double-blind, placebo-controlled trial of pirfenidone in patients with idiopathic pulmonary fibrosis
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DOI：
10.1164/rccm.200404-571oc
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发表时间：
2005-05-01
影响因子：
24.7
通讯作者：
Raghu, G
中科院分区：
文献类型：
--
作者：
Azuma, A;Nukiwa, T;Raghu, G
特发性肺纤维化（IPF）是一种致命性疾病，迄今为止尚无有效的治疗方法。在一项双盲、随机、安慰剂对照试验中，前瞻性评估了 107 名患者新型化合物吡非尼酮的疗效。在 6 分钟运动测试（主要终点）期间，脉搏血氧测定法测得的最低氧饱和度 (Sp(o2)) 从基线到 6 个月的变化在两组之间不显着 (p = 0.0722)。在基线时 6 分钟运动测试期间 Spot 保持大于 80% 的预先指定的患者子集中，吡非尼酮组在 6 个月和 9 个月时的 6 分钟运动测试期间最低 Sp(o2) 有所改善（分别为 p = 0.0069 和 0.0305）。次要终点显示了积极的治疗效果：(1) 9 个月时 VC 测量值的变化 (p = 0.0366) 和 (2) 9 个月期间仅在安慰剂组中发生 IPF 急性加重发作 (p = 0.0031)。显着的不良事件与吡非尼酮有关；然而，吡非尼酮组和安慰剂组对治疗方案的依从性相似。总之，吡非尼酮治疗在 9 个月的随访期间改善了 VC 并预防了 IPF 急性加重。未来需要进行长期研究来阐明吡非尼酮治疗 IPF 的整体安全性和有效性。
Idiopathic pulmonary fibrosis (IPF) is a fatal disorder without an effective therapy to date. In a double-blind, randomized, placebo-controlled trial, 107 patients were prospectively evaluated for efficacy of a novel compound, pirfenidone. The difference in the change in the lowest oxygen saturation by pulse oximetry (Sp(o2)) during a 6-minute exercise test, the primary endpoint, from baseline to 6 months was not significant between the two groups (p = 0.0722). In a prespecified subset of patients who maintained a Spot greater than 80% during a 6-minute exercise test at baseline, the lowest Sp(o2) improved during a 6-minute exercise test in the pirfenidone group at 6 and 9 months (p = 0.0069 and 0.0305, respectively). Positive treatment effect was demonstrated in secondary endpoints: (1) change in VC measurements at 9 months (p = 0.0366) and (2) episodes of acute exacerbation of IPF occurring exclusively in the placebo group during the 9 months (p = 0.0031). Significant adverse events were associated with pirfenidone; however, adherence to treatment regimen was similar between pirfenidone and placebo groups. In conclusion, treatment with pirfenidone improved VC and prevented acute exacerbation of IPF during the 9 months of follow-up. Future long-term studies are needed to clarify the overall safety and efficacy of pirfenidone in IPF.