Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis

Charlie McLeod; Jamie Wood; Siobhain Mulrennan; Sue Morey; André Schultz; Mitch Messer; Kate Spaapen; Yue Wu; Steven Mascaro; Alan R Smyth; Christopher C Blyth; Steve Webb; Thomas L Snelling; Richard Norman

doi:10.1016/j.jcf.2021.11.010

Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis

J Cyst Fibros. 2022 Jul;21(4):581-587. doi: 10.1016/j.jcf.2021.11.010. Epub 2022 Jan 13.

Authors

Charlie McLeod¹, Jamie Wood², Siobhain Mulrennan³, Sue Morey⁴, André Schultz⁵, Mitch Messer⁶, Kate Spaapen⁶, Yue Wu⁷, Steven Mascaro⁸, Alan R Smyth⁹, Christopher C Blyth¹⁰, Steve Webb¹¹, Thomas L Snelling¹², Richard Norman¹³

Affiliations

¹ Infectious Diseases Implementation Research Division, Telethon Kids Institute, Nedlands 6009, Australia; Infectious Diseases Department, Perth Children's Hospital, Nedlands 6009, Australia. Electronic address: charlie.mcleod81@gmail.com.
² Abilities Research Center, Department of Rehabilitation and Human Performance, Icahn School of Medicine at Mount Sinai, New York, NY 10029, United States; Respiratory Department, Sir Charles Gairdner Hospital, Nedlands 6009, Australia.
³ Respiratory Department, Sir Charles Gairdner Hospital, Nedlands 6009, Australia; Faculty of Health and Medical Sciences, University of Western Australia, Crawley 6009, Australia.
⁴ Respiratory Department, Sir Charles Gairdner Hospital, Nedlands 6009, Australia.
⁵ Division of Paediatrics, Faculty of Medicine, University of Western Australia, Crawley 6009, Australia; Wal-yan Respiratory Research Centre, Telethon Kids Institute, University of Western Australia, Nedlands 6009, Australia; Department of Respiratory Medicine, Perth Children's Hospital, Nedlands 6009, Australia.
⁶ Infectious Diseases Implementation Research Division, Telethon Kids Institute, Nedlands 6009, Australia.
⁷ Sydney School of Public Health, The University of Sydney, Sydney 2052, Australia.
⁸ Bayesian Intelligence, Upwey 3158, Australia.
⁹ Evidence Based Child Health Group, School of Medicine, University of Nottingham, NottinghamNG7 2RD, United Kingdom.
¹⁰ Division of Paediatrics, Faculty of Medicine, University of Western Australia, Crawley 6009, Australia; Infectious Diseases Department, Perth Children's Hospital, Nedlands 6009, Australia; Wesfarmers Centre for Vaccines and Infectious Diseases, Telethon Kids Institute, Nedlands 6009, Australia; Pathwest Laboratory Medicine WA, QEII Medical Centre, Nedlands 6009, Australia.
¹¹ Department of Intensive Care, St John of God Hospital, Subiaco 6008, Australia; School of Population Health and Preventive Medicine, Monash University, St Kilda 3004, Australia.
¹² Sydney School of Public Health, The University of Sydney, Sydney 2052, Australia; Menzies School of Health Research, Royal Darwin Hospital Campus, Tiwi 0810, Australia.
¹³ School of Population Health, Curtin University, Bentley 6102, Australia.

PMID: 35033463
DOI: 10.1016/j.jcf.2021.11.010

Abstract

Background: Treatment for pulmonary exacerbations of cystic fibrosis (CF) can produce a range of positive and negative outcomes. Understanding which of these outcomes are achievable and desirable to people affected by disease is critical to agreeing to goals of therapy and determining endpoints for trials. The relative importance of outcomes resulting from treatment of these episodes are not reported. We aimed to (i) quantify the relative importance of outcomes resulting from treatment for pulmonary exacerbations and (ii) develop patient and proxy carer-reported weighted outcome measures for use in adults and children, respectively.

Methods: A discrete choice experiment (DCE) survey was conducted. Participants were asked to make a series of hypothetical decisions about treatment for pulmonary exacerbations to assess how they make trade-offs between different attributes of health. Data were analysed using a conditional logistic regression model. The correlation coefficients from these data were rescaled to enable generation of a composite health outcome score between 0 and 100 (worst to best health state).

Results: 362 individuals participated (167 people with CF and 195 carers); of these, 206 completed the survey (56.9%). Most participants were female and resided in Australia. Difficult/painful breathing had the greatest impact on the preferred health state amongst people with CF and carers alike. Avoidance of gastrointestinal problems also heavily influenced decision-making.

Conclusions: These data should be considered when making treatment decisions and determining endpoints for trials. Further research is recommended to quantify the preferences of children and to determine whether these align with those of their carer(s).

Keywords: Cystic fibrosis; Discrete choice experiment; Outcomes; Patient preferences; Patient-reported outcome measures.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Adult
Australia / epidemiology
Child
Cystic Fibrosis* / complications
Cystic Fibrosis* / epidemiology
Cystic Fibrosis* / therapy
Female
Humans
Lung
Male
Outcome Assessment, Health Care