Abstract
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 the Cochrane Database of Systematic Reviews 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). 83 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.
Abstract
Measurement and reporting of lower airway outcome measures in primary ciliary dyskinesia research are not standardised. Validated, disease-specific clinical outcomes are needed to monitor disease progression in future trials and prospective cohorts. https://bit.ly/3yHERQm
Footnotes
Provenance: Submitted article, peer reviewed.
This article has supplementary material available from openres.ersjournals.com
Author contributions: B. Rubbo, P. Latzin and J.S. Lucas conceived the review; B. Rubbo, J. Thompson and C.L. Jackson performed the literature search; B. Rubbo, C.L. Jackson, E. Dehlink, P. Latzin and J.S. Lucas discussed and agreed on the inclusion and exclusion criteria; B. Rubbo, F. Gahleitner, J. Thompson and C.L. Jackson screened publications for eligibility; B. Rubbo, F. Gahleitner, J. Thompson, C.L. Jackson and A.P.L. Queiroz extracted the data; B. Rubbo, F. Gahleitner and J.F. Hueppe analysed the data; B. Rubbo and F. Halbeisen plotted the figures; B. Rubbo drafted the manuscript; F. Gahleitner, J. Thompson, C.L. Jackson, L. Behan, E. Dehlink, M. Goutaki, F. Halbeisen, G. Thouvenin, C. Kuehni, P. Latzin and J.S. Lucas critically reviewed the manuscript. All authors reviewed and approved the final manuscript.
Conflict of interest: M. Goutaki and C.E. Kuehni report that the BEAT-PCD COST Action supported their travel to and attendance of meetings during the period 2015–2019. P. Latzin reports personal fees from Vertex, Novartis, Roche, Polyphor, Vifor, Gilead, Schwabe, Zambon and Santhera, and grants from Vertex, outside the submitted work. J.S. Lucas reports that on 2 December 2019 she attended the Rare Lung and Airway Diseases on Childhood meeting at the Primary Ciliary Dyskinesia and Neonatal Interstitial Lung Diseases Conference in Jerusalem, Israel, to deliver an invited lecture on monitoring disease progression in PCD; her local travel and accommodation were arranged by the conference organisers, and her international travel was supported by BEAT-PCD. All other authors have nothing to disclose.
Support statement: B. Rubbo, J. Thompson, C.L. Jackson, J.F. Hueppe, L. Behan, E. Dehlink, M. Goutaki, F. Halbeisen, G. Thouvenin, C. Kuehni, P. Latzin and J.S. Lucas participate in the Better Evidence to Advance Therapeutic Options for PCD (BEAT-PCD) network (COST action BM 1407 and ERS Clinical Research Collaboration). B. Rubbo received a short-term scientific mission, provided through BEAT-PCD funding, to travel to Switzerland to discuss the review with M. Goutaki, F. Halbeisen and C. Kuehni.
- Received May 7, 2021.
- Accepted July 31, 2021.
- Copyright ©The authors 2021
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