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Lower airway clinical outcome measures for use in primary ciliary dyskinesia research: a scoping review

Lower airway clinical outcome measures for use in primary ciliary dyskinesia research: a scoping review
Lower airway 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 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.

2312-0541
Gahleitner, Florian
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Thompson, James
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Jackson, Claire
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Hueppe, Jana, Franziska
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Behan, Laura
cf1a7b5e-64c5-4b02-8db2-7ad96781d40d
Dehlink, Eleonora
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Goutaki, Myrofora
60fbeefc-dbb1-429c-b81a-3c35d368db64
Halbeisen, Florian S.
d4282cb2-a4d2-4f60-a471-daf86fc41020
de Queiroz, Ana Paula Lima
c73fd08d-3b0b-42ef-8ad1-36806d87dc89
Thouvenin, Guillaume
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Kuehni, Claudia E.
ac67c925-ee32-429d-a3b5-c244daa314b4
Latzin, Philipp
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Lucas, Jane
5cb3546c-87b2-4e59-af48-402076e25313
Rubbo, Bruna
dc31cd48-3d84-41ab-a8b8-351c9914dca4
Gahleitner, Florian
9e2e7ea7-ccf5-4e9a-928e-9937e10a446c
Thompson, James
a0a1e940-d720-47de-81d7-ebcd48738239
Jackson, Claire
64cdd6fa-74c3-4ac6-94ef-070620a6efd9
Hueppe, Jana, Franziska
e5062ce1-72de-4a12-9025-6676ed7470a4
Behan, Laura
cf1a7b5e-64c5-4b02-8db2-7ad96781d40d
Dehlink, Eleonora
1f60f07d-8a85-4a11-b5e5-5ea8e048e8ed
Goutaki, Myrofora
60fbeefc-dbb1-429c-b81a-3c35d368db64
Halbeisen, Florian S.
d4282cb2-a4d2-4f60-a471-daf86fc41020
de Queiroz, Ana Paula Lima
c73fd08d-3b0b-42ef-8ad1-36806d87dc89
Thouvenin, Guillaume
6f2d08a4-3875-48a6-a4fb-f5aee08ebd94
Kuehni, Claudia E.
ac67c925-ee32-429d-a3b5-c244daa314b4
Latzin, Philipp
3b0bef77-dcdd-4360-8422-8c877dea657e
Lucas, Jane
5cb3546c-87b2-4e59-af48-402076e25313
Rubbo, Bruna
dc31cd48-3d84-41ab-a8b8-351c9914dca4

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, Lucas, Jane and Rubbo, Bruna (2021) Lower airway clinical outcome measures for use in primary ciliary dyskinesia research: a scoping review. ERJ Open Research, 7 (4). (doi:10.1183/23120541.00320-2021).

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 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.

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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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Accepted/In Press date: 31 July 2021
Published date: 29 November 2021
Additional Information: Copyright ©The authors 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 James Thompson: ORCID iD orcid.org/0000-0002-9285-1317
ORCID for Claire Jackson: ORCID iD orcid.org/0000-0002-1200-0935
ORCID for Jane Lucas: ORCID iD orcid.org/0000-0001-8701-9975
ORCID for Bruna Rubbo: ORCID iD orcid.org/0000-0002-1629-8601

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Date deposited: 15 Sep 2021 16:31
Last modified: 17 Mar 2024 03:39

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Contributors

Author: Florian Gahleitner
Author: James Thompson ORCID iD
Author: Claire Jackson ORCID iD
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: Jane Lucas ORCID iD
Author: Bruna Rubbo ORCID iD

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