A new multisite study led by researchers at CU Anschutz shows that people with cystic fibrosis (CF) who start the three-drug elexacaftor/tezacaftor/ivacaftor (ETI) therapy can safely reduce many of their daily lung treatments while maintaining good health for years.
The study was published today in Journal of Cystic Fibrosis.
“This is incredibly important for individuals and families living with cystic fibrosis,” said lead author Scott Sagel, MD, PhD., professor of pediatrics-pulmonary medicine at CU Anschutz School of Medicine and director of the University of Colorado Cystic Fibrosis Center. “For decades people with cystic fibrosis have spent hours every day managing their disease. Our findings show that many have moved away from some of these time-consuming treatments thanks to ETI.”
ETI helps the dysfunctional protein that causes CF to work more efficiently, allowing the lungs to work better from the inside out instead of just dealing with symptoms like thick mucus or frequent infections. As more people with cystic fibrosis are treated with ETI, researchers were keen to understand whether long-term respiratory treatments such as inhaled antibiotics and mucus-thinning treatments are still being used at the same level.
To investigate this question, the team followed more than 600 children and adults after starting ETI. Children aged 6 to 11 were followed for up to three years. Adolescents and adults were followed for up to four and a half years. In all age groups, the use of chronic respiratory therapies, including hypertonic saline, dornase alfa, inhaled antibiotics, and oral azithromycin steadily decreased over time. Adolescents and adults cut their use of these treatments almost in half, and younger children showed similar trends.
A key finding was that stopping multiple daily treatments did not appear to lead to loss of lung function or more respiratory symptoms. This was true for age, sex, and baseline lung function. Discontinuers tended to have higher lung function at the start of ETI and were less likely to be infected by Pseudomonas aeruginosaa common lung infection associated with CF. Because the study didn’t record why people stopped taking treatments, Sagel stresses the importance of discussing any changes with a CF clinician.
“For many families, the daily time commitment required for CF care can be overwhelming,” Sagel said. “Seeing people maintain good health while taking fewer respiratory treatments is incredibly encouraging. Reducing treatment burden can have a profound impact on quality of life.”
Sagel emphasized the need for individualized care in those undergoing long-term ETI therapy.
“The opportunity to simplify daily treatment is exciting, but decisions to stop or continue treatments should be made through shared decision-making with a clinician who understands each individual’s overall health,” Sagel said.
Next, the researchers will investigate whether certain treatments may still be useful when used only during respiratory illness, and whether mechanical forms of airway clearance remain necessary for people who are no longer dependent on inhaled mucus-thinning medications.
“The goal is to help people with cystic fibrosis and their care teams maintain strong long-term health while easing the day-to-day demands of treatment,” said Sagel.
The study was part of PROMISE, one of the largest long-term projects looking at how this treatment works in routine care at CF centers in the United States.
Additional authors from the following institutions participated in the study: Seattle Children’s Research Institute, University of Washington, University of Alabama at Birmingham, Children’s Hospital of Philadelphia, Washington University at St. Louis and The Hospital for Sick Children (SickKids) in Toronto.
