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Lower airways clinical outcome measures for use in primary ciliary dyskinesia research, a scoping review

Lower airways clinical outcome measures for use in primary ciliary dyskinesia research, a scoping review
Lower airways clinical outcome measures for use in primary ciliary dyskinesia research, a scoping review
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.
2312-0541
Lucas, Jane
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Gahleitner, Florian
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Thompson, James
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Jackson, Claire
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Hueppe, Jana, Franziska
e5062ce1-72de-4a12-9025-6676ed7470a4
Behan, Laura
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Dehlink, Eleonora
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Goutaki, Myrofora
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Halbeisen, Florian S.
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de Queiroz, Ana Paula Lima
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Thouvenin, Guillaume
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Kuehni, Claudia E.
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Latzin, Philipp
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Rubbo, Bruna
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Lucas, Jane
5cb3546c-87b2-4e59-af48-402076e25313
Gahleitner, Florian
4be83303-fd24-45b4-a99e-fdd360a29d6a
Thompson, James
a0a1e940-d720-47de-81d7-ebcd48738239
Jackson, Claire
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Hueppe, Jana, Franziska
e5062ce1-72de-4a12-9025-6676ed7470a4
Behan, Laura
cf1a7b5e-64c5-4b02-8db2-7ad96781d40d
Dehlink, Eleonora
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Goutaki, Myrofora
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Halbeisen, Florian S.
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de Queiroz, Ana Paula Lima
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Thouvenin, Guillaume
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Kuehni, Claudia E.
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Latzin, Philipp
3b0bef77-dcdd-4360-8422-8c877dea657e
Rubbo, Bruna
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Lucas, Jane, Gahleitner, Florian, Thompson, James, Jackson, Claire, Hueppe, Jana, Franziska, Behan, Laura, Dehlink, Eleonora, Goutaki, Myrofora, Halbeisen, Florian S., de Queiroz, Ana Paula Lima, Thouvenin, Guillaume, Kuehni, Claudia E., Latzin, Philipp and Rubbo, Bruna (2021) Lower airways clinical outcome measures for use in primary ciliary dyskinesia research, a scoping review. ERJ Open Research. (In Press)

Record type: Article

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 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.

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Clinical outcome measures for use in PCD research_R2_submit - Accepted Manuscript
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Fig 1. Outcome measures figure updated
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Fig 2. FEV1 data
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Fig 3. HRCT_updated
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Fig 4. Correlations_updated
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Supplementary files_outcome measures_R2_submit
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More information

Accepted/In Press date: 31 July 2021

Identifiers

Local EPrints ID: 451259
URI: http://eprints.soton.ac.uk/id/eprint/451259
ISSN: 2312-0541
PURE UUID: ac8d7a81-9cf3-4816-baae-dc82737db6e1
ORCID for Bruna Rubbo: ORCID iD orcid.org/0000-0002-1629-8601

Catalogue record

Date deposited: 15 Sep 2021 16:31
Last modified: 16 Sep 2021 01:47

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Contributors

Author: Jane Lucas
Author: Florian Gahleitner
Author: James Thompson
Author: Claire Jackson
Author: Jana, Franziska Hueppe
Author: Laura Behan
Author: Eleonora Dehlink
Author: Myrofora Goutaki
Author: Florian S. Halbeisen
Author: Ana Paula Lima de Queiroz
Author: Guillaume Thouvenin
Author: Claudia E. Kuehni
Author: Philipp Latzin
Author: Bruna Rubbo ORCID iD

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