Safety, feasibility and efficacy of exercise as an airway clearance technique in cystic fibrosis: a randomised pilot feasibility trial.

Methods

Dual-site, two-arm randomised pilot trial. Fifty pwCF (≥10 years, forced expiratory volume in 1 s (FEV1) ≥40% predicted), stable on Elexacaftor/Tezacaftor/Ivacaftor, were recruited, of whom 48 were randomly assigned (1:1 with minimisation) to daily ExACT (stopping all other airway clearance techniques) or UC.

双站点、两臂随机试点试验。招募了50名囊性纤维化患者（≥10岁，第一秒用力呼气量（FEV1）≥40%预测值），在Elexacaftor/Tezacaftor/Ivacaftor治疗下病情稳定，其中48名患者被随机分配（1:1最小化）进行每日ExACT（停止所有其他气道清除技术）或常规护理（UC）。

Feasibility was measured by recruitment, retention and adherence against preset progression criteria.

可行性通过招募、留存和依从性按照预设的进展标准来衡量。

Key measures of safety and signals of efficacy included spirometry (FEV1), lung clearance index (LCI2.5), pulmonary exacerbations, physical activity, treatment burden and quality of life across 28 days.

安全性关键指标和疗效信号包括28天内的肺功能（FEV1）、肺清除指数（LCI2.5）、肺部急性加重、身体活动、治疗负担和生活质量。

Qualitative interview data and preliminary health economic data were also collected.

还收集了定性访谈数据和初步的健康经济数据。

Results

ExACT was safe over 28 days, measured by change in LCI2.5 (ExACT -0.1 (0.6) vs UC 0.2 (0.8), mean (SD)) and FEV1 (ExACT +2.1 (6.6) vs UC -0.8 (5.5), % predicted mean (SD)).

在28天的观察期内，ExACT的安全性通过LCI2.5的变化（ExACT -0.1 (0.6) vs UC 0.2 (0.8)，均值（标准差））和FEV1（ExACT +2.1 (6.6) vs UC -0.8 (5.5)，% 预测均值（标准差））来衡量。

Relative (ExACT/UC) differences of 0.97 (0.92, 1.02) for LCI2.5 and absolute differences (ExACT-UC) of 3.2 (-0.6, 6.9) % predicted for FEV1 suggest potential intervention efficacy.

LCI2.5的相对(ExACT/UC)差异为0.97(0.92, 1.02)，FEV1的绝对差异(ExACT-UC)为3.2(-0.6, 6.9)%预测值，这表明潜在的干预效果。

Few adverse events were reported; none serious.

报告的不良事件很少；没有严重的。

Recruitment and retention data suggest progression to a definitive trial, with 48/117 (41% of approached) randomised, 45/48 (92%) completing the study and a 60% overall adherence rate.

招募和保留数据表明，研究已进展到一个确定性试验阶段，共有117名被接触者中有48名（占41%）被随机分配，48名中有45名（占92%）完成了研究，总体依从率为60%。

Discussion

Testing of our primary hypothesis within a feasibility trial showed ExACT to be a safe, acceptable and feasible intervention for pwCF.

在可行性试验中对我们的主要假设进行测试表明，ExACT对于患有囊性纤维化（pwCF）的患者来说是一个安全、可接受且可行的干预措施。

These data support advancement to a definitive, longer-term, multisite trial evaluating the safety, efficacy and cost-effectiveness of ExACT, following minor refinement.

这些数据支持在对ExACT进行小幅改进后，推进到一个明确的、长期的、多地点的试验，以评估其安全性、有效性和成本效益。

trial_registration_number

NCT05482048.

NCT05482048。