Lower airway clinical outcome measures for use in primary ciliary dyskinesia research: a scoping review.
Journal
ERJ open research
ISSN: 2312-0541
Titre abrégé: ERJ Open Res
Pays: England
ID NLM: 101671641
Informations de publication
Date de publication:
Oct 2021
Oct 2021
Historique:
received:
07
05
2021
accepted:
31
07
2021
entrez:
2
12
2021
pubmed:
3
12
2021
medline:
3
12
2021
Statut:
epublish
Résumé
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). 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. 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 ( 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.
Identifiants
pubmed: 34853782
doi: 10.1183/23120541.00320-2021
pii: 00320-2021
pmc: PMC8628193
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Informations de copyright
Copyright ©The authors 2021.
Déclaration de conflit d'intérêts
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.
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