Researchers at the University of Colorado (CU) Anschutz have confirmed that cystic fibrosis (CF) patients who start the triple-drug therapy ETI (elexacaftor/tezacaftor/ivacaftor) can safely reduce their daily lung treatments while maintaining long-term health. Results from the multisite study, “Elexacaftor/Tezacaftor/Ivacaftor Is Associated With Long-Term Reduction in Use of Chronic Respiratory Therapies in Cystic Fibrosis,” were published in the Journal of Cystic Fibrosis.
ETI is a combination drug that increases the function of the CF-causing, malfunctional protein. It improves overall lung function rather than simply treating individual symptoms like infection or thickened mucus.
Scott Sagel, MD, PhDUniversity of Colorado Anschutz
The study evaluated more than 600 children and adults after they began ETI to see if the drug allowed them to decrease the dosage frequency of other respiratory therapies, such as inhaled antibiotics and mucus-thinning treatments. Researchers followed children ages six to 11 for up to three years and followed adolescents and adults for up to four and a half years.
Patients across all three age groups were able to steadily decrease their use of chronic respiratory therapies, including oral azithromycin, inhaled antibiotics, hypertonic saline and dornase alfa, by nearly half over time.
Additional key findings include:
- Patients who stopped multiple daily therapies did not experience loss of lung function or reoccurrence of respiratory symptoms. This was true for all ages and sexes and across baseline lung function.
- Patients who discontinued therapy for unknown reasons tended to have higher lung function when they began taking ETI. They were also less likely to become infected with a common CF-related lung infection, Pseudomonas aeruginosa.
Researchers said they will continue to investigate CF therapies, such as whether mechanical forms of airway clearance are still beneficial for patients who no longer rely on inhaled mucus-thinning medications.
The study was part of PROMISE — one of the largest long-term projects examining how ETI works in care facilities throughout the United States. Dr. Sagel is professor of pediatrics-pulmonary medicine at CU Anschutz School of Medicine and director of the University of Colorado Cystic Fibrosis Center in Aurora.
