Protocol for establishing a core outcome set for evaluation in studies of pulmonary exacerbations in people with cystic fibrosis

Charlie McLeod, Alan Robert Smyth, Mitch Messer, Andre Schultz, Jamie Wood, Richard Norman, Christopher C Blyth, Steve Webb, Zoe Elliott, Donald Van Devanter, Anne L Stephenson, Allison Tong, Thomas L Snelling, Charlie McLeod, Alan Robert Smyth, Mitch Messer, Andre Schultz, Jamie Wood, Richard Norman, Christopher C Blyth, Steve Webb, Zoe Elliott, Donald Van Devanter, Anne L Stephenson, Allison Tong, Thomas L Snelling

Abstract

Introduction: Pulmonary exacerbations are associated with increased morbidity and mortality in people with cystic fibrosis (CF). There is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations or how these outcomes should be measured. Outcomes of importance to people with lived experience of the disease are frequently omitted or inconsistently reported in studies, which limits the value of such studies for informing practice and policy. To better standardise outcome reporting and measurement, we aim to develop a core outcome set for studies of pulmonary exacerbations in people with CF (COS-PEX) and consensus recommendations for measurement of core outcomes.

Methods and analysis: Preliminary work for development of COS-PEX has been reported, including (1) systematic reviews of outcomes and methods for measurement reported in existing studies of pulmonary exacerbations; (2) workshops with people affected by CF within Australia; and (3) a Bayesian knowledge expert elicitation workshop with health professionals to ascertain outcomes of importance. Here we describe a protocol for the additional stages required for COS-PEX development and consensus methods for measurement of core outcomes. These include (1) an international two-round online Delphi survey and (2) consensus workshops to review and endorse the proposed COS-PEX and to agree with methods for measurement.

Ethics and dissemination: National mutual ethics scheme approval has been provided by the Child and Adolescent Health Service Human Research Ethics Committee (RGS 4926). Results will be disseminated via consumer and research networks and peer-reviewed publications. This study is registered with the Core Outcome Measures in Effectiveness Trials database.

Keywords: Cystic fibrosis; QUALITATIVE RESEARCH; RESPIRATORY MEDICINE (see Thoracic Medicine).

Conflict of interest statement

Competing interests: None declared.

© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Figures

Figure 1
Figure 1
Method for development of core outcome set for studies of pulmonary exacerbations in people with CF (COS-PEX).
Figure 2
Figure 2
Core outcome set for studies of pulmonary exacerbations in people with CF (COS-PEX).

References

    1. Hurley MN, Prayle AP, Flume P. Intravenous antibiotics for pulmonary exacerbations in people with cystic fibrosis. Cochrane Database Syst Rev 2015;7:CD009730. 10.1002/14651858.CD009730.pub2
    1. Abbott L, Plummer A, Hoo ZH, et al. . Duration of intravenous antibiotic therapy in people with cystic fibrosis. Cochrane Database Syst Rev 2019;9:CD006682. 10.1002/14651858.CD006682.pub6
    1. Bhatt J, Jahnke N, Smyth AR. Once-Daily versus multiple-daily dosing with intravenous aminoglycosides for cystic fibrosis. Cochrane Database Syst Rev 2019;9:CD002009. 10.1002/14651858.CD002009.pub7
    1. Smith S, Rowbotham NJ, Charbek E. Inhaled antibiotics for pulmonary exacerbations in cystic fibrosis. Cochrane Database Syst Rev 2018;10:CD008319. 10.1002/14651858.CD008319.pub3
    1. Waters V, Ratjen F. Combination antimicrobial susceptibility testing for acute exacerbations in chronic infection of Pseudomonas aeruginosa in cystic fibrosis. Cochrane Database Syst Rev 2015;11:CD006961. 10.1002/14651858.CD006961.pub3
    1. Elphick HE, Scott A. Single versus combination intravenous anti-pseudomonal antibiotic therapy for people with cystic fibrosis. Cochrane Database Syst Rev 2016;12:CD002007. 10.1002/14651858.CD002007.pub4
    1. Jain K, Wainwright C, Smyth AR. Bronchoscopy-guided antimicrobial therapy for cystic fibrosis. Cochrane Database Syst Rev 2018;9:CD009530. 10.1002/14651858.CD009530.pub4
    1. Lord R, Jones AM, Horsley A. Antibiotic treatment for Burkholderia cepacia complex in people with cystic fibrosis experiencing a pulmonary exacerbation. Cochrane Database Syst Rev 2020;4:CD009529. 10.1002/14651858.CD009529.pub4
    1. Smith S, Ratjen F, Remmington T, et al. . Combination antimicrobial susceptibility testing for acute exacerbations in chronic infection of Pseudomonas aeruginosa in cystic fibrosis. Cochrane Database Syst Rev 2020;5:CD006961. 10.1002/14651858.CD006961.pub5
    1. Smith S, Waters V, Jahnke N, et al. . Standard versus biofilm antimicrobial susceptibility testing to guide antibiotic therapy in cystic fibrosis. Cochrane Database Syst Rev 2020;6:CD009528. 10.1002/14651858.CD009528.pub5
    1. Prinsen CAC, Vohra S, Rose MR, et al. . Core Outcome Measures in Effectiveness Trials (COMET) initiative: protocol for an international Delphi study to achieve consensus on how to select outcome measurement instruments for outcomes included in a 'core outcome set'. Trials 2014;15:247. 10.1186/1745-6215-15-247
    1. Core Outcome Measures in Effectiveness Trials (COMET) database [Internet], 2020. Available:
    1. McLeod C, Wood J, Schultz A, et al. . Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: a systematic review. J Cyst Fibros 2020;19:858–67. 10.1016/j.jcf.2020.08.015
    1. McLeod C, Wood J, Tong A, et al. . The measurement properties of tests and tools used in cystic fibrosis studies: a systematic review. Eur Respir Rev 2021;30:200354. 10.1183/16000617.0354-2020
    1. McLeod C, Wood J, Mulrennan S. Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis. J Cys Fib 2021. 10.1016/j.jcf.2021.11.010
    1. Moher D, Glasziou P, Chalmers I, et al. . Increasing value and reducing waste in biomedical research: who’s listening? The Lancet 2016;387:1573–86. 10.1016/S0140-6736(15)00307-4
    1. Smyth A. The core outcome set Taskforce for CF (COST-CF): core outcome measures in effectiveness trials (COSMIN), 2017. Available:
    1. Kirkham JJ, Davis K, Altman DG, et al. . Core outcome Set-STAndards for development: the COS-STAD recommendations. PLoS Med 2017;14:1002447. 10.1371/journal.pmed.1002447
    1. Kirkham JJ, Gorst S, Altman DG, et al. . Core outcome Set-STAndards for reporting: the COS-STAR statement. PLoS Med 2016;13:e1002148. 10.1371/journal.pmed.1002148
    1. Dodd S, Clarke M, Becker L, et al. . A taxonomy has been developed for outcomes in medical research to help improve knowledge discovery. J Clin Epidemiol 2018;96:84–92. 10.1016/j.jclinepi.2017.12.020
    1. McLeod C, Norman R, Wood J, et al. . Novel method to select meaningful outcomes for evaluation in clinical trials. BMJ Open Respir Res 2021;8:e000877. 10.1136/bmjresp-2021-000877
    1. Williamson PR, Altman DG, Bagley H, et al. . The comet Handbook: version 1.0. Trials 2017;18:1–50. 10.1186/s13063-017-1978-4
    1. Chevance A, Tran V-T, Ravaud P. Controversy and debate series on core outcome sets. paper 1: improving the generalizability and credibility of core outcome sets (COS) by a large and international participation of diverse stakeholders. J Clin Epidemiol 2020;125:206–12. 10.1016/j.jclinepi.2020.01.004
    1. Sinha IP, Smyth RL, Williamson PR. Using the Delphi technique to determine which outcomes to measure in clinical trials: recommendations for the future based on a systematic review of existing studies. PLoS Med 2011;8:e1000393. 10.1371/journal.pmed.1000393
    1. Birko S, Dove ES, Özdemir V. Evaluation of nine consensus indices in Delphi foresight research and their dependency on Delphi survey characteristics: a simulation study and debate on Delphi design and interpretation. PLoS One 2015;10:e0135162. 10.1371/journal.pone.0135162
    1. tool QS, 2021. Available:
    1. Akl E, Mustafa R, Wiercioch N. Grade Handbook: grade group, 2021.
    1. Cho Y, Sautenet B, Rangan G, et al. . Standardised outcomes in Nephrology-Polycystic kidney disease (SONG-PKD): study protocol for establishing a core outcome set in polycystic kidney disease. Trials 2017;18:560. 10.1186/s13063-017-2298-4
    1. Boers M, Kirwan JR, Wells G, et al. . Developing core outcome measurement sets for clinical trials: OMERACT filter 2.0. J Clin Epidemiol 2014;67:745–53. 10.1016/j.jclinepi.2013.11.013

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