Inhaled Treprostinil in Pulmonary Hypertension Due to Interstitial Lung Disease
Inhaled Treprostinil in Pulmonary Hypertension Due to Interstitial Lung Disease
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DOI：
10.1056/nejmoa2008470
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发表时间：
2021-01-28
影响因子：
158.5
通讯作者：
Nathan, Steven D.
中科院分区：
文献类型：
--
作者：
Waxman, Aaron;Restrepo-Jaramillo, Ricardo;Nathan, Steven D.
背景：目前尚无治疗间质性肺病患者的肺动脉高压的治疗方法被批准。吸入曲普替尼治疗这种疾病的安全性和有效性尚不清楚。方法：在一项为期16周的多中心、随机、双盲、安慰剂对照试验中，我们招募了间质性肺疾病和肺动脉高压(右心导管术证实)的患者。患者按1：1的比例被分配接受吸入曲普替尼，通过超声脉冲雾化吸入，每天最多12次呼吸(总共72微克)，每天4次，或安慰剂。主要疗效终点是两组从基线到第16周的6分钟步行峰值距离变化的差异。次要终点包括第16周N末端B型利钠肽原(NT-proBNP)水平的变化和临床恶化的时间。结果共326例患者接受了随机分组，其中163人被分配给吸入曲普替尼，163人被分配给安慰剂。两组患者的基线特征相似。16周时，曲普替尼组与安慰剂组6min步行距离变化的最小二乘均值差为31.12m(95%可信区间，16.85~45.39；P
BACKGROUNDNo 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.METHODSWe 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 mu g) 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.RESULTSA 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