A randomised trial of lung sealant versus medical therapy for advanced emphysema.
A randomised trial of lung sealant versus medical therapy for advanced emphysema.
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DOI：
10.1183/09031936.00205614
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发表时间：
2015-09
期刊：
影响因子：
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通讯作者：
Washko GR
中科院分区：
文献类型：
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作者：
Come CE;Kramer MR;Dransfield MT;Abu-Hijleh M;Berkowitz D;Bezzi M;Bhatt SP;Boyd MB;Cases E;Chen AC;Cooper CB;Flandes J;Gildea T;Gotfried M;Hogarth DK;Kolandaivelu K;Leeds W;Liesching T;Marchetti N;Marquette C;Mularski RA;Pinto-Plata VM;Pritchett MA;Rafeq S;Rubio ER;Slebos DJ;Stratakos G;Sy A;Tsai LW;Wahidi M;Walsh J;Wells JM;Whitten PE;Yusen R;Zulueta JJ;Criner GJ;Washko GR
非对照的先导性研究显示，在晚期、以上叶为主的肺气肿患者中，使用肺气肿密封剂(ELS)进行内窥镜肺减容治疗效果良好。我们的目标是在随机对照环境中评估ELS的安全性和有效性。患者被随机分为ELS加内科治疗或单独内科治疗。尽管由于业务原因和无法评估最初的12个月终点而提前终止，300名患者中有95名被成功随机分组，为3个月和6个月的分析提供了足够的数据。57例患者(34例治疗组和23例对照组)在3个月时有疗效，34例(21例治疗组和13例对照组)在6个月时有疗效。与对照组相比，治疗组3个月后的肺功能、呼吸困难和生活质量显著改善。改善持续6个月，50%的接受治疗的患者经历了临床上重要的改善，包括一些肺功能改善了100%。44%的接受治疗的患者经历了需要住院的不良事件(是对照组的2.5倍，p=0.01)，在接受治疗的队列中有两人死亡。治疗应答者往往是那些经历呼吸道不良事件的人。尽管提前终止，但结果显示微创ELS可能是有效的，但重大风险(可能是炎症)限制了其目前的应用。
Uncontrolled pilot studies demonstrated promising results of endoscopic lung volume reduction using emphysematous lung sealant (ELS) in patients with advanced, upper lobe predominant emphysema. We aimed to evaluate the safety and efficacy of ELS in a randomised controlled setting. Patients were randomised to ELS plus medical treatment or medical treatment alone. Despite early termination for business reasons and inability to assess the primary 12-month end-point, 95 out of 300 patients were successfully randomised, providing sufficient data for 3- and 6-month analysis. 57 patients (34 treatment and 23 control) had efficacy results at 3 months; 34 (21 treatment and 13 control) at 6 months. In the treatment group, 3-month lung function, dyspnoea, and quality of life improved significantly from baseline when compared to control. Improvements persisted at 6 months with >50% of treated patients experiencing clinically important improvements, including some whose lung function improved by >100%. 44% of treated patients experienced adverse events requiring hospitalization (2.5-fold more than control, p=0.01), with two deaths in the treated cohort. Treatment responders tended to be those experiencing respiratory adverse events. Despite early termination, results show that minimally invasive ELS may be efficacious, yet significant risks (probably inflammatory) limit its current utility.