How Do Patients Decide on Treatment for Cystic Fibrosis Exacerbations?

cystic fibrosis
When choosing among alternative treatments for cystic fibrosis exacerbations, what are patients’ and caregivers’ greatest health concerns?

In a study of patient decisionmaking involving people with cystic fibrosis (CF), researchers found that concerns over difficulty or pain with breathing had the greatest impact on patients’ decisionmaking with respect to the treatment of pulmonary exacerbations, and that gastrointestinal problems also figured prominently in influencing treatment decisions. These were among study findings recently published in the Journal of Cystic Fibrosis.

Given the varying toxicities and burdens of therapeutics for cystic fibrosis, researchers sought to determine how these patients valued different aspects of their health status and the trade-offs they made when choosing between alternative therapies. Toward that end, researchers conducted a discrete choice experiment survey (DCE) in order quantify how those with CF and their caregivers hypothetically viewed the relative importance of outcomes resulting from treatment of CF pulmonary exacerbations. Researchers’ ultimate goal was creation of patient-centered instruments for comparing treatment strategies for children and adults with CF.  

The study involved 362 participants recruited from CF clinics at Perth Children’s Hospital and Sir Charles Gairdner Hospital in Australia and via consumer and research networks across Australia and the United Kingdom. Participants included 99 people with CF and 107 caregivers of children with CF, who completed all study tasks in the survey.

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 then analyzed using a conditional logistic regression model. The outcomes studied were: breathing difficulty/pain, fatigue, pain unrelated to breathing, lack of motivation/demoralization, reduced appetite, presence of fevers/night sweats, high treatment burden, inability to meet goals, sputum production and clearance, and gastrointestinal symptoms. Participants ranged from 13 to 63 years of age.

The study found that for people with CF, breathing difficulty/pain had the greatest influence on their preferred health outcome states followed by gastrointestinal symptoms, then presence of fevers/night sweats. For caregivers, the greatest influence on their preferred health outcome for their child was breathing difficulty/pain, followed by pain unrelated to breathing, then gastrointestinal symptoms.

The researchers concluded, “The health outcome preferences reported here should guide decisionmaking when seeking agreement on personal goals of therapy between patients and clinicians for treatment of pulmonary exacerbations.”

The investigators noted several limitations to their study, including lack of diversity among study participants, who were largely adult females residing in Australia, and confusion among participants resulting from the study’s use of hypothetical scenarios.

Disclosure: One study author declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.


McLeod C, Wood J, Mulrennan S, et al. Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis. J Cyst Fibros. Published online January 12, 2022. doi:10.1016/j.jcf.2021.11.010