Pirfenidone for the treatment of Hermansky-Pudlak syndrome pulmonary fibrosis.
Pirfenidone for the treatment of Hermansky-Pudlak syndrome pulmonary fibrosis.
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DOI：
10.1016/j.ymgme.2011.02.003
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发表时间：
2011-06
影响因子：
3.8
通讯作者：
Gahl, William A.
中科院分区：
文献类型：
--
作者：
O'Brien, Kevin;Troendle, James;Gochuico, Bernadette R.;Markello, Thomas C.;Salas, Jose;Cardona, Hilda;Yao, Jianhua;Bernardini, Isa;Hess, Richard;Gahl, William A.
关键词：
Hermansky-Pudlak综合征(HPS)是一种罕见的眼皮肤白化病、血小板功能障碍和某些亚型的致死性肺纤维化的疾病。除肺移植外，肺纤维化尚无有效的治疗方法，但使用抗纤维化药物吡非尼酮的初步试验显示，结果令人振奋。目前的随机、安慰剂对照、前瞻性、双盲试验调查了吡非尼酮治疗轻中度HPS-1和4肺纤维化的安全性和有效性。每4个月在美国国立卫生研究院临床中心对受试者进行评估，主要结果参数是用力肺活量的变化，使用随机系数的重复测量分析。35名患有HPS-1肺纤维化的受试者在4年的时间间隔内被纳入研究；23名受试者接受吡非尼酮治疗，12名接受安慰剂治疗。4名受试者退出试验，3名受试者死亡，报告了10例严重不良事件。两组都有相似的副作用，尤其是胃食道反流。在30名患者入选12个月后，对主要结果参数进行的中期分析显示，安慰剂组和吡非尼酮组之间没有统计学差异，研究因无效而停止。没有重大的安全问题。其他临床试验表明，确定单一或多个药物方案可能对治疗进行性HPS-1肺纤维化有效。
Hermansky-Pudlak syndrome (HPS) type is a rare disorder of oculocutaneous albinism, platelet dysfunction, and in some subtypes, fatal pulmonary fibrosis. There is no effective treatment for the pulmonary fibrosis except lung transplantation, but an initial trial using pirfenidone, an anti-fibrotic agent, showed promising results. The current, randomized, placebo-controlled, prospective, double-blind trial investigated the safety and efficacy of pirfenidone for mild to moderate HPS-1 and 4 pulmonary fibrosis. Subjects were evaluated every 4 months at the National Institutes of Health Clinical Center, and the primary outcome parameter was change in forced vital capacity using repeated measures analysis with random coefficients. Thirty-five subjects with HPS-1 pulmonary fibrosis were enrolled during a 4-year interval; 23 subjects received pirfenidone and 12 received placebo. Four subjects withdrew from the trial, 3 subjects died, and 10 serious adverse events were reported. Both groups experienced similar side effects, especially gastroesophageal reflux. Interim analysis of the primary outcome parameter, performed 12 months after 30 patients were enrolled, showed no statistical difference between the placebo and pirfenidone groups, and the study was stopped due to futility. There were no significant safety concerns. Other clinical trials are indicated to identify single or multiple drug regimens that may be effective in treatment for progressive HPS-1 pulmonary fibrosis.
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