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Real-Life Impact of ETI Therapy in Cystic Fibrosis: Insights From a Large Treatment Center

Real-Life Impact of ETI Therapy in Cystic Fibrosis: Insights From a Large Treatment Center 706 470 Pam Georgiana

Greater clinical gains observed in patients with severe lung disease, regardless of genotype.

A recent prospective observational study led by Shahid I. Sheikh, MD, pediatric pulmonologist at Nationwide Children’s Hospital and professor of Pediatrics at The Ohio State University College of Medicine, offers insights into the effectiveness of a new medication for adolescents and adults with cystic fibrosis. The findings, published in Pulmonary Pharmacology and Therapeutics, examined how genotype and lung disease severity influence the outcomes of elexacaftor-tezacaftor-ivacaftor (ETI) therapy.

“ETI has revolutionized how we care for cystic fibrosis patients, improving both life expectancy and quality of life,” says Dr. Sheikh. “However, existing clinical trial data often lack the detail on how different patient groups respond to treatment in real-life clinical settings.”

The study followed 115 adolescents and adults with cystic fibrosis treated at Nationwide Children’s, all carrying at least one copy of the F508del mutation. Approximately 58% were homozygous (two copies of the F508del mutation), and 66% had moderate to severe lung disease at the beginning of the study. The researchers utilized a range of measures, including lung function tests (ppFEV1), body mass index (BMI), exacerbations and bacterial throat/sputum culture analysis, to compare clinical outcomes before and after one year of ETI therapy. They specifically examined how outcomes differed by genotype (homozygous and heterozygous) and lung function.

“We observed significant improvements across the entire cohort, including increases in lung function and BMI, fewer pulmonary exacerbations and reductions in bacterial culture positivity on throat cultures,” Dr. Sheikh notes.

Genotype did not influence response to therapy.

“Whether patients were homozygous or heterozygous for F508del did not significantly change their likelihood of clinical improvement,” Dr. Sheikh explains. “This is encouraging and suggests ETI will benefit a broad range of patients.”

However, baseline lung function made a notable difference. Patients with more severe lung disease experienced greater improvements in lung function and BMI, as well as fewer pulmonary exacerbations.

Dr. Sheikh elaborates, “It makes sense as those with more advanced disease have more room for improvement, but it also highlights how transformative ETI can be.”

One of the most promising findings was the reduction in treatment burden. Antibiotic use and hospital admissions declined, and nearly half of the patients were able to discontinue daily dornase alfa and hypertonic saline after one year of therapy.

“ETI has the potential to substantially reduce treatment complexity, which could improve patients’ quality of life,” says Dr. Sheikh.

Despite the positive outcomes, Dr. Sheikh emphasizes the need for more research.

“Our study does not include a control group and is based at one center, so the results may not apply broadly,” he notes. Additionally, because patients produced less sputum while on ETI therapy, the findings may underestimate existing airway pathogens.

Dr. Sheikh concludes, “Our study affirms that ETI improves health outcomes for many cystic fibrosis patients, regardless of homozygosity. What is really exciting is that those with more severe lung disease appear to experience the greatest clinical gains.”

 

 

Reference:

Sheikh S, Holtzlander M, Eisner M, Gushue C, Palacios S, Kotha K, Imran S, McCoy KS. Real-life impact of genotype and severity of lung disease on efficacy of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis. Pulm Pharmacol Ther. 2025 Mar;88:102345.

Image credit: Nationwide Children’s

About the author

Pam Georgiana is a brand marketing professional and writer located in Bexley, Ohio. She believes that words bind us together as humans and that the best stories remind us of our humanity. She specialized in telling engaging stories for healthcare, B2B services, and nonprofits using classic storytelling techniques. Pam has earned an MBA in Marketing from Capital University in Columbus, Ohio.

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