Australian Centre for Health Services Innovation, Queensland University of Technology, Brisbane, Australia
Dept of Respiratory and Sleep Medicine, Queensland Children's Hospital, Brisbane, Australia.
Eur Respir J. 2022 Nov 3;60(5). doi: 10.1183/13993003.00300-2022. Print 2022 Nov.
Bronchiectasis is being diagnosed increasingly in children and adolescents. Recurrent respiratory exacerbations are common in children and adolescents with this chronic pulmonary disorder. Respiratory exacerbations are associated with an impaired quality of life, poorer long-term clinical outcomes, and substantial costs to the family and health systems. The 2021 European Respiratory Society (ERS) clinical practice guideline for the management of children and adolescents with bronchiectasis provided a definition of acute respiratory exacerbations for clinical use but to date there is no comparable universal definition for clinical research. Given the importance of exacerbations in the field, this ERS Task Force sought to obtain robust definitions of respiratory exacerbations for clinical research. The panel was a multidisciplinary team of specialists in paediatric and adult respiratory medicine, infectious disease, physiotherapy, primary care, nursing, radiology, methodology, patient advocacy, and parents of children and adolescents with bronchiectasis. We used a standardised process that included a systematic literature review, parent survey, and a Delphi approach involving 299 physicians (54 countries) caring for children and adolescents with bronchiectasis. Consensus was obtained for all four statements drafted by the panel as the disagreement rate was very low (range 3.6-7.2%). The panel unanimously endorsed the four consensus definitions for 1a) non-severe exacerbation and 1b) severe exacerbation as an outcome measure, 2) non-severe exacerbation for studies initiating treatment, and 3) resolution of a non-severe exacerbation for clinical trials involving children and adolescents with bronchiectasis. This ERS Task Force proposes using these internationally derived, consensus-based definitions of respiratory exacerbations for future clinical paediatric bronchiectasis research.
支气管扩张症在儿童和青少年中的诊断率越来越高。患有这种慢性肺部疾病的儿童和青少年经常会出现反复的呼吸道恶化。呼吸道恶化会导致生活质量下降、长期临床结局较差，并给家庭和医疗系统带来巨大的负担。2021 年欧洲呼吸学会（ERS）临床实践指南为支气管扩张症患儿和青少年的管理提供了急性呼吸道恶化的临床定义，但迄今为止，尚无用于临床研究的可比通用定义。鉴于恶化在该领域的重要性，该 ERS 工作组旨在为临床研究获得稳健的呼吸道恶化定义。该小组由儿科和成人呼吸医学、传染病、物理治疗、初级保健、护理、放射学、方法学、患者权益、患有支气管扩张症的儿童和青少年的父母等方面的专家组成。我们使用了一种标准化的流程，包括系统文献回顾、家长调查以及涉及 299 名（来自 54 个国家）治疗支气管扩张症儿童和青少年的医生的 Delphi 方法。专家组起草的所有四项声明都达成了共识，因为意见分歧率非常低（范围为 3.6-7.2%）。专家组一致认可四项共识定义，即 1a）非严重恶化和 1b）严重恶化作为结局指标、2）非严重恶化用于启动治疗的研究，以及 3）涉及支气管扩张症儿童和青少年的临床试验中解决非严重恶化。该 ERS 工作组建议在未来的儿科支气管扩张症临床研究中使用这些国际上制定的、基于共识的呼吸道恶化定义。
