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. 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.
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).
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).
Authors: 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 Journal: BMJ Open Date: 2022-09-23 Impact factor: 3.006