A new multi-site study led by researchers at CU Anschutz shows that people with cystic fibrosis (CF) who start the triple-drug therapy elexacaftor/tezacaftor/ivacaftor (ETI) can safely reduce many of their daily lung treatments while maintaining good health for years.
The study was published today in the Journal of Cystic Fibrosis .
"This is incredibly meaningful for individuals and families living with CF," said lead author 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."
ETI helps the malfunctioning protein that causes CF work more effectively, allowing the lungs to function better from the inside rather than simply treating symptoms like thick mucus or frequent infections. As more people with CF are being treated with ETI, researchers have been eager to understand whether long-standing respiratory therapies such as inhaled antibiotics and mucus-thinning treatments are still being used at the same level.
To explore this question, the team followed more than 600 children and adults after they began ETI. Children ages 6 to 11 were tracked for up to three years. Adolescents and adults were followed up to four and a half years. Across all age groups, use of chronic respiratory therapies including hypertonic saline, dornase alfa, inhaled antibiotics and oral azithromycin fell steadily over time. Teens and adults cut their use of these treatments by nearly half and younger children showed similar trends.
A key finding was that stopping multiple daily therapies did not appear to lead to loss of lung function or more respiratory symptoms. This was true across age, sex, and baseline lung function. Those who discontinued therapies tended to have higher lung function at the start of ETI and were less likely to be infected with Pseudomonas aeruginosa, a common CF-related lung infection. Because the study did not capture individuals' reasons for stopping therapies, 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 doing fewer respiratory therapies is incredibly encouraging. A reduction in treatment burden can have a profound impact on quality of life."
Sagel emphasized the need for personalized care in those on long-term ETI therapy.
"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," said Sagel.
Next, researchers will investigate whether some therapies may still be useful when used only during respiratory illnesses and whether mechanical forms of airway clearance remain necessary for people who no longer depend on 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.
The study was part of PROMISE, one of the largest long-term projects examining how this therapy works in everyday care at CF centers across the United States.
Additional authors from the following institutions were involved in the study: Seattle Children's Research Institute, the University of Washington, the University of Alabama at Birmingham, the Children's Hospital of Philadelphia, Washington University in St. Louis, and The Hospital for Sick Children (SickKids) in Toronto.
About the University of Colorado Anschutz
The University of Colorado Anschutz is a world-class academic medical campus at the forefront of transformative science, medicine, education and patient care. The campus encompasses the University of Colorado health professional schools, more than 60 centers and institutes and two nationally ranked independent hospitals - UCHealth University of Colorado Hospital and Children's Hospital Colorado – which see more than two million adult and pediatric patient visits yearly. Innovative, interconnected and highly collaborative, CU Anschutz delivers life-changing treatments, patient care and professional training and conducts world-renowned research fueled by $910 million in annual research funding, including $757 million in sponsored awards and $153 million in philanthropic gifts.
