People with cystic fibrosis highly value freedom from pain associated with breathing and difficulty with breathing, according to Australian-led research into preferred outcomes.

The study of 162 adolescents and adults with CF and 186 carers of children with CF comprised a discrete choice experiment (DCE) to quantify the relative importance of potential outcomes following hypothetical treatment of pulmonary exacerbations.

The study, published in the Journal of Cystic Fibrosis, found difficult/painful breathing had the greatest influence on the preferred health state for people with CF and carers alike.

“Avoidance of gastrointestinal problems were also strongly influential.”

“This should be considered when deciding whether to extend the duration of treatment for pulmonary exacerbation beyond 10-14 days, give the paucity of data to support clear clinical benefit with prolongation of therapy, and the likelihood that extending treatment may result in gastrointestinal (and other) side-effects,” the researchers said.

Also important to people with CF was when treatment always or often interfered with their lives (‘things you like to do’) or impacted on school, studies or work.

Carers of children with CF were less concerned about these outcomes and more concerned with issues such as pain unrelated to breathing and anxiety or depression experienced by children.

Coughing up mucus was considered a negative outcome of treatment and important to both people with CF and carers when it occurred ‘a lot’ or in ‘huge amounts.’

“Paradoxically, production of sputum might also be a therapeutic goal, given that clearance of mucus is considered desirable to avoid stasis of airways secretions and concomitant infection,” the study said

“Understanding the perceived causal mechanisms which give rise to outcomes might help explain discrepancies in how some stakeholders value some attributes.”

The researchers said their findings should help inform treatment at both an individual patient level and at a population level when evaluating future treatments for pulmonary exacerbations.

“They should be used to guide joint decision making between physicians and their patients when setting therapeutic goals.”