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

Charlie McLeod; Jamie Wood; André Schultz; Richard Norman; Sherie Smith; Christopher C. Blyth; Steve Webb; Alan R. Smyth; Thomas L. Snelling

doi:10.1016/j.jcf.2020.08.015

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

Charlie McLeod^*, Jamie Wood, André Schultz, Richard Norman, Sherie Smith, Christopher C. Blyth, Steve Webb, Alan R. Smyth, Thomas L. Snelling

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

14 Citations (Scopus)

22 Downloads (Pure)

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.

Original language	English
Pages (from-to)	858-867
Number of pages	10
Journal	Journal of Cystic Fibrosis
Volume	19
Issue number	6
DOIs	https://doi.org/10.1016/j.jcf.2020.08.015
Publication status	Published - 01 Nov 2020
Externally published	Yes

Bibliographical note

Funding Information:
This research did not receive any specific grant from funding agencies in the public, commercial or not-for-profit sectors. CM is supported by an NHMRC post-graduate scholarship ( GNT1150996 ), and top-up grants from the Wesfarmers Centre (top-up grant) and the Perth Children's Hospital Foundation (9772 & 9757). TS is supported by a Career Development Fellowship from the National Health and Medical Research Council ( GNT1111657 ). CB is also supported by the National Health and Medical Research Council ( 1111596/1173163 ).

Publisher Copyright:
© 2020

Keywords

Cystic fibrosis
Endpoint measure
Outcome assessment
Outcome variables
Patient reported outcome measures
Pulmonary exacerbation

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Pulmonary and Respiratory Medicine

Access to Document

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Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic review
Copyright 2020 Elsevier. This manuscript is distributed under a Creative Commons Attribution-NonCommercial-NoDerivs License (https://creativecommons.org/licenses/by-nc-nd/4.0/), which permits distribution and reproduction for non-commercial purposes, provided the author and source are cited.
Accepted author manuscript, 832 KBLicence: CC BY-NC-ND

Cite this

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

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.",

keywords = "Cystic fibrosis, Endpoint measure, Outcome assessment, Outcome variables, Patient reported outcome measures, Pulmonary exacerbation",

author = "Charlie McLeod and Jamie Wood and Andr{\'e} Schultz and Richard Norman and Sherie Smith and Blyth, {Christopher C.} and Steve Webb and Smyth, {Alan R.} and Snelling, {Thomas L.}",

note = "Funding Information: This research did not receive any specific grant from funding agencies in the public, commercial or not-for-profit sectors. CM is supported by an NHMRC post-graduate scholarship ( GNT1150996 ), and top-up grants from the Wesfarmers Centre (top-up grant) and the Perth Children's Hospital Foundation (9772 & 9757). TS is supported by a Career Development Fellowship from the National Health and Medical Research Council ( GNT1111657 ). CB is also supported by the National Health and Medical Research Council ( 1111596/1173163 ). Publisher Copyright: {\textcopyright} 2020",

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doi = "10.1016/j.jcf.2020.08.015",

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T1 - Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic review

AU - McLeod, Charlie

AU - Wood, Jamie

AU - Schultz, André

AU - Norman, Richard

AU - Smith, Sherie

AU - Blyth, Christopher C.

AU - Webb, Steve

AU - Smyth, Alan R.

AU - Snelling, Thomas L.

N1 - Funding Information: This research did not receive any specific grant from funding agencies in the public, commercial or not-for-profit sectors. CM is supported by an NHMRC post-graduate scholarship ( GNT1150996 ), and top-up grants from the Wesfarmers Centre (top-up grant) and the Perth Children's Hospital Foundation (9772 & 9757). TS is supported by a Career Development Fellowship from the National Health and Medical Research Council ( GNT1111657 ). CB is also supported by the National Health and Medical Research Council ( 1111596/1173163 ). Publisher Copyright: © 2020

PY - 2020/11/1

Y1 - 2020/11/1

N2 - 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.

AB - 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.

KW - Cystic fibrosis

KW - Endpoint measure

KW - Outcome assessment

KW - Outcome variables

KW - Patient reported outcome measures

KW - Pulmonary exacerbation

U2 - 10.1016/j.jcf.2020.08.015

DO - 10.1016/j.jcf.2020.08.015

M3 - Review article

C2 - 33191129

AN - SCOPUS:85096019340

SN - 1569-1993

VL - 19

SP - 858

EP - 867

JO - Journal of Cystic Fibrosis

JF - Journal of Cystic Fibrosis

IS - 6

ER -

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

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

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Cite this