Highlights
- •Preferred outcome states after treatment for pulmonary exacerbations are reported.
- •Difficult/painful breathing had the greatest impact on preferred health states.
- •Avoidance of gastrointestinal problems also heavily influenced decision-making.
- •These data should be considered when considering goals of therapy and trial design.
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
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Article info
Publication history
Published online: January 12, 2022
Accepted:
November 19,
2021
Received in revised form:
November 17,
2021
Received:
September 5,
2021
Identification
Copyright
© 2021 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
