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吸入曲前列环素治疗间质性肺疾病所致肺动脉高压可有效改善运动能力
作者:小柯机器人 发布时间:2021/1/17 16:39:30

美国伊诺瓦费尔法克斯医院Steven D. Nathan团队研究了吸入曲前列环素治疗间质性肺疾病所致肺动脉高压的疗效。2021年1月13日,该研究发表在《新英格兰医学杂志》上。

当前尚无批准用于治疗间质性肺病患者肺动脉高压的方法。吸入曲前列环素对该状况患者的安全性和疗效尚不清楚。

研究组进行了一项多中心、随机、双盲、安慰剂对照、为期16周的研究,招募了326例间质性肺疾病和肺动脉高压(通过右心导管检查证明)的患者。将其按1:1随机分配,其中163例吸入曲前列环素,通过超声脉冲喷雾器给药,最多12次吸入(总计72μg),每天4次;163例按照相同频率吸入安慰剂。主要疗效终点是两组从基线检查到第16周6分钟步行距离峰值变化的差异。次要终点包括第16周时N端B型利钠肽前体(NT-proBNP)水平的变化和临床恶化的时间。

两组患者的基线特征相似。在第16周,曲前列环素组与安慰剂组间在6分钟步行距离内与基线相比的最小二乘均值差异为31.12 m,差异显著。曲前列环素组中NT-proBNP水平较基线降低了15%,而安慰剂组则升高了46%,差异显著。曲前列环素组中有37例(22.7%)发生临床恶化,而安慰剂组中有54例(33.1%),风险比为0.61。最常见的不良反应是咳嗽、头痛、呼吸困难、头晕、恶心、疲劳和腹泻。

研究结果表明,对于间质性肺疾病引起的肺动脉高压患者,与安慰剂相比,吸入曲前列环素相比基线水平显著提高了患者的运动能力。

附:英文原文

Title: Inhaled Treprostinil in Pulmonary Hypertension Due to Interstitial Lung Disease

Author: Aaron Waxman, M.D., Ph.D.,, Ricardo Restrepo-Jaramillo, M.D.,, Thenappan Thenappan, M.D.,, Ashwin Ravichandran, M.D.,, Peter Engel, M.D.,, Abubakr Bajwa, M.D.,, Roblee Allen, M.D.,, Jeremy Feldman, M.D.,, Rahul Argula, M.D.,, Peter Smith, Pharm.D.,, Kristan Rollins, Pharm.D.,, Chunqin Deng, M.D., Ph.D.,, Leigh Peterson, Ph.D.,, Heidi Bell, M.D.,, Victor Tapson, M.D.,, and Steven D. Nathan, M.D.

Issue&Volume: 2021-01-13

Abstract:

Background

No therapies are currently approved for the treatment of pulmonary hypertension in patients with interstitial lung disease. The safety and efficacy of inhaled treprostinil for patients with this condition are unclear.

Methods

We enrolled patients with interstitial lung disease and pulmonary hypertension (documented by right heart catheterization) in a multicenter, randomized, double-blind, placebo-controlled, 16-week trial. Patients were assigned in a 1:1 ratio to receive inhaled treprostinil, administered by means of an ultrasonic, pulsed-delivery nebulizer in up to 12 breaths (total, 72 μg) four times daily, or placebo. The primary efficacy end point was the difference between the two groups in the change in peak 6-minute walk distance from baseline to week 16. Secondary end points included the change in N-terminal pro–B-type natriuretic peptide (NT-proBNP) level at week 16 and the time to clinical worsening.

Results

A total of 326 patients underwent randomization, with 163 assigned to inhaled treprostinil and 163 to placebo. Baseline characteristics were similar in the two groups. At week 16, the least-squares mean difference between the treprostinil group and the placebo group in the change from baseline in the 6-minute walk distance was 31.12 m (95% confidence interval [CI], 16.85 to 45.39; P<0.001). There was a reduction of 15% in NT-proBNP levels from baseline with inhaled treprostinil as compared with an increase of 46% with placebo (treatment ratio, 0.58; 95% CI, 0.47 to 0.72; P<0.001). Clinical worsening occurred in 37 patients (22.7%) in the treprostinil group as compared with 54 patients (33.1%) in the placebo group (hazard ratio, 0.61; 95% CI, 0.40 to 0.92; P=0.04 by the log-rank test). The most frequently reported adverse events were cough, headache, dyspnea, dizziness, nausea, fatigue, and diarrhea.

Conclusions

In patients with pulmonary hypertension due to interstitial lung disease, inhaled treprostinil improved exercise capacity from baseline, assessed with the use of a 6-minute walk test, as compared with placebo.

DOI: 10.1056/NEJMoa2008470

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2008470

 

期刊信息

The New England Journal of Medicine:《新英格兰医学杂志》,创刊于1812年。隶属于美国麻省医学协会,最新IF:70.67
官方网址:http://www.nejm.org/
投稿链接:http://www.nejm.org/page/author-center/home