运动作为囊性纤维化患者的气道清除技术（ExACT-CF）：一项随机试点试验的原理与研究方案
Exercise as an Airway Clearance Technique in people with Cystic Fibrosis (ExACT-CF): rationale and study protocol for a randomised pilot trial.
作者信息
Urquhart Don S, Cunningham Steve, Taylor Emily, Vogiatzis Ioannis, Allen Lorna, Lewis Steff, Neilson Aileen R, Soilemezi Dia, Akooji Naseerah, Saynor Zoe L
机构信息
Department of Child Life and Health, University of Edinburgh, Edinburgh, UK.
Department of Paediatric Respiratory and Sleep Medicine, Royal Hospital for Children and Young People, Edinburgh, UK.
出版信息
NIHR Open Res. 2022 Dec 19;2:64. doi: 10.3310/nihropenres.13347.1. eCollection 2022.
BACKGROUND
Chest physiotherapy is an established cornerstone of care for people with cystic fibrosis (pwCF), but is often burdensome. Guidelines recommend at least one chest physiotherapy session daily, using various airway clearance techniques (ACTs). Exercise (with huffs and coughs) may offer an alternative ACT, however the willingness of pwCF to be randomised into a trial needs testing. The 'ExACT-CF: Exercise as an Airway Clearance Technique in people with Cystic Fibrosis' trial will test the feasibility of recruiting pwCF to be randomised to continue usual care (chest physiotherapy) or replace it with exercise ACT (ExACT) for 28-days. Secondary aims include determining the short-term clinical impact (and safety) of stopping routine chest physiotherapy and replacing it with ExACT, and effects on physical activity, sleep, mood, quality of life and treatment burden, alongside preliminary health economic measures and acceptability.
METHODS
Multi-centre, two-arm, randomised (1:1 allocation using minimisation), pilot trial at two sites. Fifty pwCF (≥10 years, FEV >40% predicted, stable on Elexacaftor/Tezacaftor/Ivacaftor (ETI)) will be randomised to an individually-customised ExACT programme (≥once daily aerobic exercise of ≥20-minutes duration at an intensity that elicits deep breathing, with huffs and coughs), or usual care. After baseline assessments, secondary outcomes will be assessed after 28-days, with additional home lung function and exacerbation questionnaires at 7, 14 and 21-days, physical activity and sleep monitoring throughout, and embedded qualitative and health-economic components. Feasibility measures include recruitment, retention, measurement completion, adverse events, interviews exploring the acceptability of trial procedures, and a trial satisfaction questionnaire.
DISCUSSION
Co-designed with the UK CF community, the ExACT-CF pilot trial is the first multi-centre RCT to test the feasibility of recruiting pwCF stable on ETI into a trial investigating ExACT. This pilot trial will inform the feasibility, design, management, likely external validity for progression to a main phase randomised controlled trial.
REGISTRATION
Clinicaltrials.gov ( NCT05482048).
背景
胸部物理治疗是囊性纤维化患者（pwCF）护理的既定基石，但通常较为繁重。指南建议每天至少进行一次胸部物理治疗，采用各种气道清除技术（ACTs）。运动（配合呵气和咳嗽）可能是一种替代的ACT，但需要测试pwCF参与试验随机分组的意愿。“ExACT-CF：运动作为囊性纤维化患者的气道清除技术”试验将测试招募pwCF随机分组以继续常规护理（胸部物理治疗）或用运动ACT（ExACT）替代28天的可行性。次要目标包括确定停止常规胸部物理治疗并用ExACT替代的短期临床影响（和安全性），以及对身体活动、睡眠、情绪、生活质量和治疗负担的影响，同时进行初步的健康经济评估和可接受性评估。
方法
在两个地点进行多中心、双臂、随机（使用最小化法1:1分配）试点试验。五十名pwCF（年龄≥10岁，FEV>预测值的40%，在依列卡福/替扎卡福/依伐卡福（ETI）治疗下病情稳定）将被随机分配到个性化定制的ExACT方案（每天至少进行一次持续时间≥20分钟的有氧运动，强度要能引起深呼吸，配合呵气和咳嗽）或常规护理组。在基线评估后，28天后评估次要结局，在第7、14和21天进行额外的家庭肺功能和病情加重情况问卷调查，全程监测身体活动和睡眠，并纳入定性和健康经济部分。可行性指标包括招募、留存、测量完成情况、不良事件、探索试验程序可接受性的访谈以及试验满意度问卷。
讨论
ExACT-CF试点试验是与英国囊性纤维化群体共同设计的，是首个测试将在ETI治疗下病情稳定的pwCF招募到研究ExACT的试验中的可行性的多中心随机对照试验。该试点试验将为推进到主要阶段随机对照试验的可行性、设计、管理以及可能的外部有效性提供信息。
注册
Clinicaltrials.gov（NCT05482048）
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