A BEAT-PCD consensus statement: a core outcome set for pulmonary disease interventions in primary ciliary dyskinesia
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Methods 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.
Results 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.
Conclusion This expert consensus resulted in a COS for clinical trials on pulmonary health among people with PCD.
| Originalsprog | Engelsk |
|---|---|
| Artikelnummer | 00115-2023 |
| Tidsskrift | ERJ Open Research |
| Vol/bind | 10 |
| Udgave nummer | 1 |
| Antal sider | 11 |
| ISSN | 2312-0541 |
| DOI | |
| Status | Udgivet - 2024 |
Bibliografisk note
Funding Information:
This study was developed in the framework of Better Experimental Approaches to Treat Primary Ciliary Dyskinesia (BEAT-PCD), a clinical research collaboration (CRC) of researchers and clinicians, supported by the European Respiratory Society. The primary goal of the network is to improve diagnosis and treatment of people with PCD through the coordination of research from basic science to clinical care. As part of the project, a working group was established on the topic of clinical trials, of which one objective was to define reliable clinical outcome measures and biomarkers [21]. During an open BEAT-PCD online meeting, participants were invited to join the core group of the consensus project. Additional experts were invited due to their expertise in research on pulmonary outcome measures. The core group also contributed to the consensus, with the exclusion of one facilitator (R. Kos), who did not participate in the e-survey voting. After the establishment of the core group, they selected experts for this consensus statement based on their experience in PCD research, particularly on respiratory disease. The core group aimed to ensure that experts from different countries and continents were included, with expertise on both children and adults with PCD. This resulted in a list of 25 experts from 17 countries who were invited to form the expert panel. Since the consensus focuses on outcomes related to pulmonary disease interventions, the panel did not include any members with expertise in manifestations of PCD affecting other organs. Furthermore, as the focus was on experts in design and execution of studies, no patient representatives were included in the set-up of the COS. However, patient support groups are an integral part of the BEAT-PCD CRC and will be involved in later phases of this process.
Publisher Copyright:
© The authors 2024.
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