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

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

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

3 Citations (Scopus)

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).

Original language	English
Pages (from-to)	581-587
Number of pages	7
Journal	Journal of Cystic Fibrosis
Volume	21
Issue number	4
DOIs	https://doi.org/10.1016/j.jcf.2021.11.010
Publication status	Published - 22 Jul 2022
Externally published	Yes

Bibliographical note

Funding Information:
This research was supported by a Perth Children's Hospital Foundation New Investigator Grant (9772). 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 (9757). MM was supported by a study grant from the Medical Research Future Fund (GNT1152376). TS was 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).

Funding Information:
This research was supported by a Perth Children's Hospital Foundation New Investigator Grant (9772). 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 (9757). MM was supported by a study grant from the Medical Research Future Fund (GNT1152376). TS was 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:
© 2021

Keywords

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

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Pulmonary and Respiratory Medicine

Access to Document

10.1016/j.jcf.2021.11.010Licence: Unspecified

Cite this

@article{b56349dc679b41a8bb5e6f02df937374,

title = "Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis",

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",

author = "Charlie McLeod and Jamie Wood and Siobhain Mulrennan and Sue Morey and Andr{\'e} Schultz and Mitch Messer and Kate Spaapen and Yue Wu and Steven Mascaro and Smyth, {Alan R.} and Blyth, {Christopher C.} and Steve Webb and Snelling, {Thomas L.} and Richard Norman",

note = "Funding Information: This research was supported by a Perth Children's Hospital Foundation New Investigator Grant (9772). 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 (9757). MM was supported by a study grant from the Medical Research Future Fund (GNT1152376). TS was 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). Funding Information: This research was supported by a Perth Children's Hospital Foundation New Investigator Grant (9772). 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 (9757). MM was supported by a study grant from the Medical Research Future Fund (GNT1152376). TS was 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} 2021",

year = "2022",

month = jul,

day = "22",

doi = "10.1016/j.jcf.2021.11.010",

language = "English",

volume = "21",

pages = "581--587",

journal = "Journal of Cystic Fibrosis",

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number = "4",

}

TY - JOUR

T1 - Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis

AU - McLeod, Charlie

AU - Wood, Jamie

AU - Mulrennan, Siobhain

AU - Morey, Sue

AU - Schultz, André

AU - Messer, Mitch

AU - Spaapen, Kate

AU - Wu, Yue

AU - Mascaro, Steven

AU - Smyth, Alan R.

AU - Blyth, Christopher C.

AU - Webb, Steve

AU - Snelling, Thomas L.

AU - Norman, Richard

N1 - Funding Information: This research was supported by a Perth Children's Hospital Foundation New Investigator Grant (9772). 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 (9757). MM was supported by a study grant from the Medical Research Future Fund (GNT1152376). TS was 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). Funding Information: This research was supported by a Perth Children's Hospital Foundation New Investigator Grant (9772). 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 (9757). MM was supported by a study grant from the Medical Research Future Fund (GNT1152376). TS was 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: © 2021

PY - 2022/7/22

Y1 - 2022/7/22

N2 - 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).

AB - 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).

KW - Cystic fibrosis

KW - Discrete choice experiment

KW - Outcomes

KW - Patient preferences

KW - Patient-reported outcome measures

U2 - 10.1016/j.jcf.2021.11.010

DO - 10.1016/j.jcf.2021.11.010

M3 - Article

C2 - 35033463

AN - SCOPUS:85122970630

SN - 1569-1993

VL - 21

SP - 581

EP - 587

JO - Journal of Cystic Fibrosis

JF - Journal of Cystic Fibrosis

IS - 4

ER -

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

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

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