TY - JOUR T1 - Lower airways clinical outcome measures for use in primary ciliary dyskinesia research, a scoping review JF - ERJ Open Research JO - erjor DO - 10.1183/23120541.00320-2021 SP - 00320-2021 AU - Florian Gahleitner AU - James Thompson AU - Claire L Jackson AU - Jana F Hueppe AU - Laura Behan AU - Eleonora Dehlink AU - Myrofora Goutaki AU - Florian Halbeisen AU - Ana Paula L Queiroz AU - Guillaume Thouvenin AU - Claudia Kuehni AU - Philipp Latzin AU - Jane S Lucas AU - Bruna Rubbo Y1 - 2021/01/01 UR - http://openres.ersjournals.com/content/early/2021/08/12/23120541.00320-2021.abstract N2 - Objectives Disease-specific, well-defined, and validated clinical outcome measures are essential in designing research studies. Poorly defined outcome measures hamper pooling of data and comparisons between studies. We aimed to identify and describe pulmonary outcome measures that could be used for follow-up of patients with primary ciliary dyskinesia (PCD).Methods We conducted a scoping review by systematically searching Medline, Embase and Cochrane Systematic Review online databases for studies published from 1996 to 2020 that included ≥10 PCD adult and/or paediatric patients.Results We included 102 studies (7289 patients). Eighty-three studies reported on spirometry, 11 on body plethysmography, 15 on multiple breath washout, 36 on high-resolution computed tomography (HRCT), 57 on microbiology, and 17 on health-related quality of life. Measurement and reporting of outcomes varied considerably between studies (e.g. different scoring systems for chest HRCT scans). Additionally, definitions of outcome measures varied (e.g. definition of chronic colonisation by respiratory pathogen), impeding direct comparisons of results.Conclusions This review highlights the need for standardisation of measurements and reporting of outcome measures to enable comparisons between studies. Defining a core set of clinical outcome measures is necessary to ensure reproducibility of results and for use in future trials and prospective cohorts.FootnotesThis manuscript has recently been accepted for publication in the ERJ Open Research. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJOR online. Please open or download the PDF to view this article.Conflict of interest: Dr. Latzin reports personal fees from Vertex, personal fees from Novartis, personal fees from Roche, personal fees from Polyphor, personal fees from Vifor, personal fees from Gilead, personal fees from Schwabe, personal fees from Zambon, personal fees from Santhera, grants from Vertex, outside the submitted work. ER -