Lechtzin Noah, Mayer-Hamblett Nicole, West Natalie E, Allgood Sarah, Wilhelm Ellen, Khan Umer, Aitken Moira L, Ramsey Bonnie W, Boyle Michael P, Mogayzel Peter J, Gibson Ronald L, Orenstein David, Milla Carlos, Clancy John P, Antony Veena, Goss Christopher H
1 Johns Hopkins University School of Medicine, Baltimore, Maryland.
2 University of Washington School of Medicine, Seattle, Washington.
Am J Respir Crit Care Med. 2017 Nov 1;196(9):1144-1151. doi: 10.1164/rccm.201610-2172OC.
Individuals with cystic fibrosis (CF) experience frequent acute pulmonary exacerbations, which lead to decreased lung function and reduced quality of life.
The goal of this study was to determine if an intervention directed toward early detection of pulmonary exacerbations using home spirometry and symptom monitoring would result in slower decline in lung function than in control subjects.
We conducted a multicenter, randomized trial at 14 CF centers with subjects at least 14 years old. The early intervention arm subjects measured home spirometry and symptoms electronically twice per week. Sites were notified if a participant met criteria for an exacerbation and contacted participants to determine if treatment for acute exacerbation was required. Participants in the usual care arm were seen every 3 months and were asked to contact the site if they were concerned about worsening pulmonary symptoms.
The primary outcome was the 52-week change in FEV. Secondary outcomes included time to first exacerbation and subsequent exacerbation, quality of life, and change in weight. A total of 267 patients were randomized, and the study arms were well matched at baseline. There was no significant difference between study arms in 52-week mean change in FEV slope (mean slope difference, 0.00 L, 95% confidence interval, -0.07 to 0.07; P = 0.99). The early intervention arm subjects detected exacerbations more frequently than usual care arm subjects (time to first exacerbation hazard ratio, 1.45; 95% confidence interval, 1.09 to 1.93; P = 0.01). Adverse events were not significantly different between treatment arms.
An intervention of home monitoring among patients with CF was able to detect more exacerbations than usual care, but this did not result in slower decline in lung function. Clinical trial registered with www.clinicaltrials.gov (NCT01104402).
囊性纤维化（CF）患者经常经历急性肺部加重，这会导致肺功能下降和生活质量降低。
本研究的目的是确定使用家庭肺活量测定法和症状监测进行的旨在早期发现肺部加重的干预措施是否会比对照组导致肺功能下降更缓慢。
我们在14个CF中心对至少14岁的受试者进行了一项多中心随机试验。早期干预组的受试者每周通过电子方式测量两次家庭肺活量和症状。如果参与者符合加重标准，会通知研究地点，并联系参与者以确定是否需要对急性加重进行治疗。常规护理组的参与者每3个月就诊一次，并被要求如果担心肺部症状恶化就联系研究地点。
主要结局是第52周时第一秒用力呼气容积（FEV）的变化。次要结局包括首次加重和随后加重的时间、生活质量以及体重变化。共有267名患者被随机分组，研究组在基线时匹配良好。两组在第52周FEV斜率的平均变化方面无显著差异（平均斜率差异为0.00L，95%置信区间为-0.07至0.07；P = 0.99）。早期干预组的受试者比常规护理组的受试者更频繁地检测到加重（首次加重的风险比为1.45；95%置信区间为1.09至1.93；P = 0.01）。治疗组之间的不良事件无显著差异。
CF患者的家庭监测干预措施比常规护理能检测到更多的加重情况，但这并未导致肺功能下降更缓慢。该临床试验已在www.clinicaltrials.gov注册（NCT01104402）。
