运动作为囊性纤维化气道清除技术的安全性、可行性和有效性：一项随机试点可行性试验

IF 7.7 1区医学 Q1 RESPIRATORY SYSTEM

Thorax Pub Date : 2025-10-01 DOI:10.1136/thorax-2025-223080

Don S Urquhart, Emily Taylor, Steve Cunningham, Steff Lewis, Aileen Rae Neilson, Dia Soilemezi, Hannah Ensor, Ioannis Vogiatzis, Lorna J Allen, Zoe Louise Saynor

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引用次数: 0

摘要

目的：对比常规治疗（UC），验证运动作为一种气道清除技术（ExACT）对囊性纤维化（pwCF）患者的可行性和安全性。方法双中心、双臂随机对照试验。招募50名pwCF患者（≥10岁，1秒内用力呼气量(FEV1)≥预测的40%），使用Elexacaftor/Tezacaftor/Ivacaftor稳定，其中48名随机分配（1:1）至每日ExACT（停止所有其他气道清除技术）或UC。可行性是通过招募、保留和遵守预设的进展标准来衡量的。安全性和有效性的关键指标包括肺活量测定（FEV1）、肺清除率指数（LCI2.5）、肺恶化、身体活动、治疗负担和28天的生活质量。定性访谈数据和初步卫生经济数据也被收集。通过LCI2.5 (ExACT + 0.1 (0.6) vs UC 0.2（0.8)，平均值（SD））和FEV1 (ExACT +2.1 (6.6) vs UC−0.8（5.5)，%预测平均值（SD））的变化来测量，ExACT在28天内是安全的。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)%，表明干预可能有效。报告的不良事件很少；没有认真的。招募和保留数据表明进展到最终试验，48/117（41%）随机分组，45/48（92%）完成研究，总体依从率为60%。在可行性试验中对我们的主要假设进行了测试，结果表明ExACT是一种安全、可接受和可行的pwCF干预措施。这些数据支持推进一项明确的、长期的、多地点的试验，评估ExACT的安全性、有效性和成本效益。试验注册号[NCT05482048][1]。如有合理要求，可提供资料。在当前研究期间产生或分析的所有数据均可根据合理要求从研究的主要研究者（ZLS和DSU）处获得。[1]: /查找/ external-ref ? link_type = CLINTRIALGOV&access_num = NCT05482048&atom = % 2 fthoraxjnl % 2恐惧% 2 f2025 % 2 f09 % 2 + 30 % 2 fthorax - 2025 - 223080. -原子

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

Objectives To test the feasibility and safety of exercise as an airway clearance technique (ExACT) for people with cystic fibrosis (pwCF) versus usual care (UC). 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. Feasibility was measured by recruitment, retention and adherence against preset progression criteria. 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. Qualitative interview data and preliminary health economic data were also collected. Findings 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)). 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. Few adverse events were reported; none serious. 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. Discussion Testing of our primary hypothesis within a feasibility trial showed ExACT to be a safe, acceptable and feasible intervention for pwCF. These data support advancement to a definitive, longer-term, multisite trial evaluating the safety, efficacy and cost-effectiveness of ExACT, following minor refinement. Trial registration number [NCT05482048][1]. Data are available upon reasonable request. All data generated or analysed during the current study are available from the Principal Investigators of the study (ZLS and DSU) on reasonable request. [1]: /lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT05482048&atom=%2Fthoraxjnl%2Fearly%2F2025%2F09%2F30%2Fthorax-2025-223080.atom

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来源期刊

Thorax 医学-呼吸系统

CiteScore

16.10

自引率

2.00%

发文量

197

审稿时长

1 months

期刊介绍： Thorax stands as one of the premier respiratory medicine journals globally, featuring clinical and experimental research articles spanning respiratory medicine, pediatrics, immunology, pharmacology, pathology, and surgery. The journal's mission is to publish noteworthy advancements in scientific understanding that are poised to influence clinical practice significantly. This encompasses articles delving into basic and translational mechanisms applicable to clinical material, covering areas such as cell and molecular biology, genetics, epidemiology, and immunology.