Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
College of Applied Medical Sciences, King Faisal University, Al-Ahsa, Saudi Arabia.
BMJ Open Respir Res. 2024 Aug 15;11(1):e002456. doi: 10.1136/bmjresp-2024-002456.
Pulmonary exacerbations (PExs) are clinically important in people with cystic fibrosis (CF). Multiple definitions have been used for PEx, and this scoping review aimed to identify the different definitions reported in the literature and to ascertain which signs and symptoms are commonly used to define them.
A search was performed using Embase, MEDLINE, Cochrane Library, Scopus and CINAHL. All publications reporting clinical trials or prospective observational studies involving definitions of PEx in people with CF published in English from January 1990 to December 2022 were included. Data were then extracted for qualitative thematic analysis.
A total of 14 039 records were identified, with 7647 titles and abstracts screened once duplicates were removed, 898 reviewed as full text and 377 meeting the inclusion criteria. Pre-existing definitions were used in 148 publications. In 75% of papers, an objective definition was used, while 25% used a subjective definition, which subcategorised into treatment-based definitions (76%) and those involving clinician judgement (24%). Objective definitions were subcategorised into three groups: those based on a combination of signs and symptoms (50%), those based on a predefined combination of signs and symptoms plus the initiation of acute treatment (47%) and scores involving different clinical features each with a specific weighting (3%). The most common signs and symptoms reported in the definitions were, in order, sputum production, cough, lung function, weight/appetite, dyspnoea, chest X-ray changes, chest sounds, fever, fatigue or lethargy and haemoptysis.
We have identified substantial variation in the definitions of PEx in people with CF reported in the literature. There is a requirement for the development of internationally agreed-upon, standardised and validated age-specific definitions. Such definitions would allow comparison between studies and effective meta-analysis to be performed and are especially important in the highly effective modulator therapy era in CF care.
在囊性纤维化（CF）患者中，肺部加重（PEx）具有重要的临床意义。已经使用了多种定义，本范围界定综述旨在确定文献中报告的不同定义，并确定用于定义它们的常见症状和体征。
使用 Embase、MEDLINE、Cochrane 图书馆、Scopus 和 CINAHL 进行了搜索。纳入了 1990 年 1 月至 2022 年 12 月期间以英文发表的报告 CF 患者 PEx 定义的临床试验或前瞻性观察研究的所有出版物。然后提取数据进行定性主题分析。
共确定了 14039 条记录，去除重复后筛选出 14039 个标题和摘要，898 个作为全文进行了审查，377 个符合纳入标准。148 篇论文使用了预先存在的定义。在 75%的论文中，使用了客观定义，而 25%使用了主观定义，主观定义分为基于治疗的定义（76%）和基于临床医生判断的定义（24%）。客观定义分为三组：基于症状和体征组合的定义（50%）、基于预先定义的症状和体征组合加上急性治疗开始的定义（47%）和涉及不同临床特征的评分，每个特征都有特定的权重（3%）。定义中报告的最常见的症状和体征依次为痰液产生、咳嗽、肺功能、体重/食欲、呼吸困难、胸部 X 射线变化、胸部声音、发热、疲劳或嗜睡和咯血。
我们已经确定了文献中 CF 患者 PEx 定义存在很大差异。需要制定国际公认的、标准化的和经过验证的、针对特定年龄的定义。这种定义将允许对研究进行比较，并进行有效的荟萃分析，在 CF 治疗的高度有效的调节剂治疗时代尤其重要。
