Kos Renate, Goutaki Myrofora, Kobbernagel Helene E, Rubbo Bruna, Shoemark Amelia, Aliberti Stefano, Altenburg Josje, Anagnostopoulou Pinelopi, Athanazio Rodrigo A, Beydon Nicole, Dell Sharon D, Emiralioglu Nagehan, Ferkol Thomas W, Loebinger Michael R, Lorent Natalie, Maître Bernard, Marthin June, Morgan Lucy C, Nielsen Kim G, Ringshausen Felix C, Shteinberg Michal, Tiddens Harm A W M, Maitland-Van der Zee Anke H, Chalmers James D, Lucas Jane S A, Haarman Eric G
Dept of Pulmonary Medicine, Amsterdam University Medical Centres - loc. AMC, University of Amsterdam, Amsterdam, The Netherlands.
Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
ERJ Open Res. 2024 Jan 8;10(1). doi: 10.1183/23120541.00115-2023. eCollection 2024 Jan.
Consistent use of reliable and clinically appropriate outcome measures is a priority for clinical trials, with clear definitions to allow comparability. We aimed to develop a core outcome set (COS) for pulmonary disease interventions in primary ciliary dyskinesia (PCD).
A multidisciplinary international PCD expert panel was set up. A list of outcomes was created based on published literature. Using a modified three-round e-Delphi technique, the panel was asked to decide on relevant end-points related to pulmonary disease interventions and how they should be reported. First, inclusion of an outcome in the COS was determined. Second, the minimum information that should be reported per outcome. The third round finalised statements. Consensus was defined as ≥80% agreement among experts.
During the first round, experts reached consensus on four out of 24 outcomes to be included in the COS. Five additional outcomes were discussed in subsequent rounds for their use in different subsettings. Consensus on standardised methods of reporting for the COS was reached. Spirometry, health-related quality-of-life scores, microbiology and exacerbations were included in the final COS.
This expert consensus resulted in a COS for clinical trials on pulmonary health among people with PCD.
在临床试验中，持续使用可靠且临床适用的结局指标是一项优先事项，要有清晰的定义以确保可比性。我们旨在为原发性纤毛运动障碍（PCD）的肺部疾病干预制定一个核心结局集（COS）。
成立了一个多学科的国际PCD专家小组。根据已发表的文献创建了一份结局清单。使用改良的三轮电子德尔菲技术，要求专家小组确定与肺部疾病干预相关的相关终点以及应如何报告这些终点。首先，确定某个结局是否纳入COS。其次，确定每个结局应报告的最低信息。第三轮确定最终声明。共识定义为专家之间≥80%的一致意见。
在第一轮中，专家们就COS中应纳入的24个结局中的4个达成了共识。在随后的轮次中又讨论了另外5个结局在不同亚组中的应用。就COS的标准化报告方法达成了共识。最终的COS包括肺活量测定、健康相关生活质量评分、微生物学和病情加重情况。
这一专家共识产生了一个用于PCD患者肺部健康临床试验的COS。
