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Pablo AU - MacNee, William AU - Hopkinson, Nicholas S. AU - Piitulainen, Eeva AU - Seersholm, Niels AU - Vogelmeier, Claus F. AU - Bals, Robert AU - McElvaney, Gerry AU - Stockley，Robert A. Y1 - 2019/11/01 UR - //www.qdcxjkg.com/content/54/5/1900673.abstract N2 -遗传性α1-抗胰蛋白酶(AAT)缺乏症(ZZ-AATD)和严重慢性阻塞性肺病(COPD)患者经常出现病情加重。我们假设吸入雾化AAT是一种有效的治疗方法。我们随机分配168名患者接受每日两次80 mg AAT溶液或安慰剂的吸入治疗，持续50周。患者使用电子日记记录病情恶化情况。主要终点是从随机分组到第一次基于事件的病情恶化的时间。次要终点包括Anthonisen标准定义的病情恶化性质的改变。安全性也进行了评估。AAT组首次中度或重度发作的中位时间为112天(四分位数范围(IQR) 40-211天)，安慰剂组为140天(IQR 72-142天)(p=0.0952)。 The mean yearly rate of all exacerbations was 3.12 in the AAT-treated group and 2.67 in the placebo group (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 -