Introduction Recurrent pulmonary exacerbations lead to progressive lung damage in cystic fibrosis (CF). Inhaled medications (mucoactive agents and antibiotics) help prevent exacerbations, but objectively measured adherence is low. We investigated whether a multi-component (complex) self-management intervention to support adherence would reduce exacerbation rates over 12 months. Methods Between October 2017 and May 2018, adults with CF (aged ≥16 years; 19 UK centres) were randomised to the intervention (data-logging nebulisers, a digital platform and behavioural change sessions with trained clinical interventionists) or usual care (data-logging nebulisers). Outcomes included pulmonary exacerbations (primary outcome), objectively measured adherence, body mass index (BMI), lung function (FEV1) and Cystic Fibrosis Questionnaire-Revised (CFQ-R). Analyses were by intent to treat over 12 months. Results Among intervention (n=304) and usual care (n=303) participants (51% female, median age 31 years), 88% completed 12-month follow-up. Mean exacerbation rate was 1.63/year with intervention and 1.77/year with usual care (adjusted ratio 0.96; 95% CI 0.83 to 1.12; p=0.64). Adjusted mean differences (95% CI) were in favour of the intervention versus usual care for objectively measured adherence (9.5% (8.6% to 10.4%)) and BMI (0.3 (0.1 to 0.6) kg/m2), with no difference for %FEV1 (1.4 (−0.2 to 3.0)). Seven CFQ-R subscales showed no between-group difference, but treatment burden reduced for the intervention (3.9 (1.2 to 6.7) points). No intervention-related serious adverse events occurred. Conclusions While pulmonary exacerbations and FEV1 did not show statistically significant differences, the intervention achieved higher objectively measured adherence versus usual care. The adherence difference might be inadequate to influence exacerbations, though higher BMI and lower perceived CF treatment burden were observed. Data are available on reasonable request. Requests for patient level data and statistical code should be made to the corresponding author and will be considered by members of the original trial management group, including the chief investigators and members of clinical trials research unit, who will release data on a case-by-case basis. Data will be shared following the principles for sharing patient level data as described by Tudur Smith C, et al BMC Medicine 2015;13:298 (). The data will not contain any direct identifiers, and we will minimise indirect identifiers and remove free-text data to minimise the risk of identification.

中文翻译：

---

通过支持患有囊性纤维化的成人的治疗依从性来减少肺部恶化的自我管理干预：一项随机对照试验

简介 反复肺部恶化导致囊性纤维化 (CF) 中进行性肺损伤。吸入药物（粘液活性剂和抗生素）有助于预防恶化，但客观测量的依从性较低。我们调查了支持依从性的多组分（复杂）自我管理干预是否会降低 12 个月内的恶化率。方法 2017 年 10 月至 2018 年 5 月，患有 CF 的成年人（年龄≥16 岁；19 个英国中心）随机接受干预（数据记录雾化器、数字平台和训练有素的临床干预师的行为改变课程）或常规护理（数据-记录雾化器）。结果包括肺部恶化（主要结果）、客观测量的依从性、体重指数（BMI）、肺功能（FEV1）和修订的囊性纤维化问卷（CFQ-R）。分析的目的是治疗超过 12 个月。结果 在干预（n=304）和常规护理（n=303）参与者（51% 女性，中位年龄 31 岁）中，88% 完成了 12 个月的随访。干预组平均恶化率为 1.63/年，常规治疗组为 1.77/年（调整比率 0.96；95% CI 0.83 至 1.12；p=0.64）。对于客观测量的依从性（9.5%（8.6% 至 10.4%））和 BMI（0.3（0.1 至 0.6）kg/m2），调整后的平均差异（95% CI）有利于干预与常规护理，在%FEV1（1.4（-0.2 到 3.0））。七个 CFQ-R 分量表显示没有组间差异，但干预减少了治疗负担（3.9（1.2 至 6.7）分）。未发生与干预相关的严重不良事件。结论 虽然肺部恶化和 FEV1 没有显示出统计学上的显着差异，与常规护理相比，干预实现了更高的客观测量依从性。尽管观察到较高的 BMI 和较低的 CF 治疗负担，但依从性差异可能不足以影响恶化。可根据合理要求提供数据。患者水平数据和统计代码的请求应向通讯作者提出，并由原试验管理组成员考虑，包括首席研究员和临床试验研究单位的成员，他们将逐案发布数据案例基础。将按照 Tudur Smith C 等人 BMC Medicine 2015;13:298 ( 尽管观察到较高的 BMI 和较低的感知 CF 治疗负担。可根据合理要求提供数据。患者水平数据和统计代码的请求应向通讯作者提出，并由原试验管理组成员考虑，包括首席研究员和临床试验研究单位的成员，他们将逐案发布数据案例基础。将按照 Tudur Smith C 等人 BMC Medicine 2015;13:298 ( 尽管观察到较高的 BMI 和较低的感知 CF 治疗负担。可根据合理要求提供数据。患者水平数据和统计代码的请求应向通讯作者提出，并由原试验管理组成员考虑，包括首席研究员和临床试验研究单位的成员，他们将逐案发布数据案例基础。将按照 Tudur Smith C 等人 BMC Medicine 2015;13:298 ( 包括首席研究员和临床试验研究组成员，他们将根据具体情况发布数据。将按照 Tudur Smith C 等人 BMC Medicine 2015;13:298 ( 包括首席研究员和临床试验研究组成员，他们将根据具体情况发布数据。将按照 Tudur Smith C 等人 BMC Medicine 2015;13:298 (）。数据将不包含任何直接标识符，我们将尽量减少间接标识符并删除自由文本数据，以最大限度地降低识别风险。