Randomized trial evaluates sustained lung function and nutritional improvements in cystic fibrosis, suggesting varied responses to therapy.
Key Points
The study aims to evaluate the long-term effectiveness of elexacaftor/tezacaftor/ivacaftor (ETI) on lung function and nutrition in people with cystic fibrosis (PwCF).
Analyzed data from PwCF ≥ 12 years with F508del enrolled in the CF Foundation Patient Registry from 2018 to 2022.
Compared mean percent predicted forced expiratory volume in 1 second (ppFEV1) and body mass index (BMI) before and after ETI initiation over the course of four years.
Utilized linear regression and negative binomial modeling to evaluate response and pulmonary exacerbation rates.
Mean ppFEV1 increased by 9.9 (95% CI 9.6-10.2) after three years of ETI administration.
Mean BMI increased by 1.03 kg/m2 (95% CI 0.98—1.07) in the same period.
32.8% of participants exhibited significant improvements in both ppFEV1 and BMI, with reduced pulmonary exacerbation rates (adjusted IRR 0.62, 95% CI 0.57—0.66).