A recent study led by researchers at CU Anschutz has found that people with cystic fibrosis (CF) who begin the triple-drug therapy elexacaftor/tezacaftor/ivacaftor (ETI) can safely reduce many of their daily lung treatments without negative effects on their health over several years. The findings were published in the Journal of Cystic Fibrosis.
"This is incredibly meaningful for individuals and families living with CF," said Scott Sagel, MD, PhD, professor of pediatrics-pulmonary medicine at the CU Anschutz School of Medicine and director of the University of Colorado Cystic Fibrosis Center. "For decades people with CF have spent hours every day managing their disease. Our findings show that many have stepped back from some of those time-consuming therapies thanks to ETI."
The ETI therapy targets the underlying protein malfunction responsible for CF, improving lung function from within rather than only addressing symptoms such as thick mucus or frequent infections. As more patients are treated with ETI, researchers sought to determine if long-standing respiratory therapies like inhaled antibiotics and mucus-thinning treatments remain necessary at previous levels.
To answer this question, the research team monitored over 600 children and adults after they started ETI treatment. Children aged 6 to 11 were observed for up to three years, while adolescents and adults were followed for up to four and a half years. The use of chronic respiratory therapies—including hypertonic saline, dornase alfa, inhaled antibiotics, and oral azithromycin—declined steadily across all age groups during this period. Teenagers and adults reduced these treatments by nearly half; younger children showed similar patterns.
Importantly, stopping multiple daily therapies did not result in loss of lung function or increased respiratory symptoms regardless of age, sex, or initial lung function level. Those who discontinued therapies generally had higher lung function when starting ETI and were less likely to be infected with Pseudomonas aeruginosa—a common infection among CF patients. However, since the study did not collect data on why individuals stopped certain therapies, Sagel highlighted the importance of consulting a clinician before making changes.
"For many families, the daily time commitment required for CF care can be overwhelming," Sagel said. "Seeing people maintain good health while doing fewer respiratory therapies is incredibly encouraging. A reduction in treatment burden can have a profound impact on quality of life."
Sagel also stressed that decisions about ongoing therapy should be personalized: "The opportunity to simplify daily treatment is exciting but decisions to stop or continue therapies should be made through shared decision-making with a clinician who understands each person's overall health."
Future research will examine whether some treatments may still play a role during respiratory illnesses and if mechanical airway clearance remains necessary for those no longer using inhaled mucus-thinning medications.
"The goal is to help people with CF and their care teams maintain strong long-term health while easing daily treatment demands," said Sagel.
This work was part of PROMISE—one of the largest ongoing projects studying how this therapy performs in routine care at cystic fibrosis centers throughout the United States.
Additional contributors came from Seattle Children's Research Institute; University of Washington; University of Alabama at Birmingham; Children's Hospital of Philadelphia; Washington University in St. Louis; and The Hospital for Sick Children (SickKids) in Toronto.
