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

Research output: Contribution to journalArticleResearchpeer-review

3 Citations (Scopus)


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).

Original languageEnglish
Pages (from-to)581-587
Number of pages7
JournalJournal of Cystic Fibrosis
Issue number4
Publication statusPublished - Jul 2022


  • Cystic fibrosis
  • Discrete choice experiment
  • Outcomes
  • Patient preferences
  • Patient-reported outcome measures

Cite this