Treatment of idiopathic pulmonary fibrosis with a new antifibrotic agent, pirfenidone - Results of a prospective, open-label phase II study
Treatment of idiopathic pulmonary fibrosis with a new antifibrotic agent, pirfenidone - Results of a prospective, open-label phase II study
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DOI：
10.1164/ajrccm.159.4.9805017
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发表时间：
1999-04-01
影响因子：
24.7
通讯作者：
Mageto, Y
中科院分区：
文献类型：
--
作者：
Raghu, G;Johnson, WC;Mageto, Y
特发性肺纤维化（IPF）是一种病因不明且具有致命性结果的进行性临床综合征。目前可用的疗法无效并且伴有显着的副作用。吡非尼酮是一种新的研究性抗纤维化药物，评估了其对晚期 IPF 末期患者的耐受性和有效性。连续出现 IPF 且尽管接受常规治疗但病情恶化的患者或无法耐受或不愿尝试常规治疗的患者接受口服吡非尼酮治疗。治疗是在富有同情心的基础上进行的（开放标签）。对 54 名患者的死亡率、肺功能变化和不良反应进行了随访。他们的平均年龄为 62 岁，平均症状持续时间为 4.6 年，自肺活检以来的时间为 3.2 年。 46 名患者中有 38 名停止了常规治疗；其他八人能够减少泼尼松剂量，八人之前没有接受过常规治疗。一年和两年生存率分别为 78%（95% CI 66%、89%）和 63%（95% CI 50%、76%）。入组前肺功能恶化的患者在开始治疗后似乎稳定下来。不良反应相对较小。这项研究的结果令人鼓舞。吡非尼酮是一种有前景的 IPF 新疗法，具有良好的耐受性。
Idiopathic pulmonary fibrosis (IPF) is a progressive clinical syndrome of unknown etiology and fatal outcome. Currently available therapies are ineffective and associated with significant adverse effects. Pirfenidone, a new, investigational antifibrotic agent, was evaluated for its tolerability and usefulness in terminally ill patients with advanced IPF. Consecutive patients with IPF and deterioration despite conventional therapy or who were unable to tolerate or unwilling to try conventional therapy were treated with oral pirfenidone. Treatment was administered on a compassionate basis (open-label). Fifty-four patients were followed for mortality, change in lung function, and adverse effects. Their mean age was 62, mean duration of symptoms 4.6 yr, and time since lung biopsy was 3.2 yr. Conventional therapy was discontinued in 38 of 46 patients; the other eight were able to decrease their prednisone dosage and eight had no previous conventional treatment. One- and 2-yr survival was 78% (95% CI 66%, 89%) and 63% (95% CI 50%, 76%), respectively. Patients whose lung functions had deteriorated prior to enrollment appeared to stabilize after beginning treatment. Adverse effects were relatively minor. The results of this study are encouraging. Pirfenidone is a promising new treatment for IPF that is well tolerated.