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Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic review

Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic review Thumbnail


Authors

Charlie McLeod

Jamie Wood

Schultz

Richard Norman

SHERIE SMITH sherie.smith@nottingham.ac.uk
Cochrane Systematic Reviewer

Christopher C. Blyth

Steve Webb

Alan R. Smyth

Thomas L. Snelling



Abstract

Background: There is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations in people with cystic fibrosis (CF). Outcomes used for evaluation should be meaningful; that is, they should capture how people feel, function or survive and be acknowledged as important to people with CF, or should be reliable surrogates of those outcomes. We aimed to summarise the outcomes and corresponding endpoints which have been reported in studies of pulmonary exacerbations, and to identify those which are most likely to be meaningful. Methods: A PROSPERO registered systematic review (CRD42020151785) was conducted in Medline, Embase and Cochrane from inception until July 2020. Registered trials were also included. Results: 144 studies met the inclusion criteria. A wide range of outcomes and corresponding endpoints were reported. Death, QoL and many patient-reported outcomes are likely to be meaningful as they directly capture how people feel, function or survive. Forced expiratory volume in 1-second [FEV1] is a validated surrogate of risk of death and reduced QoL. The extent of structural lung disease has also been correlated with lung function, pulmonary exacerbations and risk of death. Since no evidence of a correlation between airway microbiology or biomarkers with clinically meaningful outcomes was found, the value of these as surrogates was unclear. Conclusions: Death, QoL, patient-reported outcomes, FEV1, and structural lung changes were identified as outcomes that are most likely to be meaningful. Development of a core outcome set in collaboration with stakeholders including people with CF is recommended.

Citation

McLeod, C., Wood, J., Schultz, A., Norman, R., Smith, S., Blyth, C. C., …Snelling, T. L. (2020). Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic review. Journal of Cystic Fibrosis, 19(6), 858-867. https://doi.org/10.1016/j.jcf.2020.08.015

Journal Article Type Review
Acceptance Date Aug 25, 2020
Online Publication Date Nov 13, 2020
Publication Date 2020-11
Deposit Date Nov 14, 2020
Publicly Available Date Nov 14, 2021
Journal Journal of Cystic Fibrosis
Print ISSN 1569-1993
Electronic ISSN 1873-5010
Publisher Elsevier
Peer Reviewed Peer Reviewed
Volume 19
Issue 6
Pages 858-867
DOI https://doi.org/10.1016/j.jcf.2020.08.015
Keywords Pediatrics, Perinatology, and Child Health; Pulmonary and Respiratory Medicine
Public URL https://nottingham-repository.worktribe.com/output/5040298
Publisher URL https://www.cysticfibrosisjournal.com/article/S1569-1993(20)30829-8/fulltext
Related Public URLs https://www.sciencedirect.com/science/article/abs/pii/S1569199320308298

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