TY - T1的疗效和安全性的吸入α1-antitrypsin患者严重α1-antitrypsin不足和频繁的慢性阻塞性肺病加重病人的JF -欧洲呼吸杂志》乔和J - 10.1183/13993003.00673 -2019欧元六世- 54 - 5 SP - 1900673 AU Stolk Jan AU - Tov Naveh AU -查普曼,肯尼斯·r . AU -费尔南德斯,巴勃罗盟,马克尼威廉盟,霍普金森尼古拉斯·s . AU - Piitulainen Eeva AU - Seersholm,尼尔斯盟——Vogelmeier老人f . AU -巴尔斯,罗伯特•AU - McElvaney摘要N2期-遗传型1-抗胰蛋白酶(AAT)缺乏症(zzl - aatd)和严重慢性阻塞性肺疾病(COPD)患者经常病情加重。我们假设吸入雾化的AAT是一种有效的治疗方法。我们随机分配了168名患者，在50周内每天吸入两次80毫克AAT溶液或安慰剂。患者使用电子日记记录病情恶化情况。主要终点是从随机化到第一次基于事件的恶化的时间。二次端点包括变化的性质恶化Anthonisen定义的标准。安全性也进行了评估。AAT中值为112天(四分位范围(IQR) 40-211天)，安慰剂为140天(IQR 72-142天)(p=0.0952)。所有恶化的平均年发生率在aat治疗组为3.12，安慰剂组为2.67 (p=0.31)。 More patients receiving AAT reported treatment-related treatment-emergent adverse events compared to placebo (57.5% versus 46.9%, respectively) and they were more likely to withdraw from the study. After the first year of the study, when modifications to the handling of the nebuliser were introduced, the rate of safety events in the AAT-treated group dropped to that of the placebo group.We conclude that in AATD patients with severe COPD and frequent exacerbations, AAT inhalation for 50 weeks showed no effect on time to first exacerbation but may have changed the pattern of the episodes.Inhaled α1-antitrypsin did not significantly reduce the time to first exacerbation in patients with severe α1-antitrypsin deficiency who experience frequent exacerbations of COPD in a randomised placebo-controlled clinical trial of 1 year http://bit.ly/2P5zXdK ER -