ONE-YEAR EXPERIENCE WITH ELEXACAFTOR / TEZACAFTOR / IVACAFTOR THERAPY AT THE ADULT CYSTIC FIBROSIS CENTRE

Abstract

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have fundamentally changed the treatment landscape of cystic fibrosis (CF). Real-world clinical data are important for evaluating their effectiveness and safety across different patient populations. Aim. To evaluate the one-year experience with elexacaftor / tezacaftor / ivacaftor therapy at the Adult Cystic Fibrosis Centre of the Hospital of Lithuanian University of Health Sciences Kauno klinikos. Methods. A retrospective analysis of demographic and clinical data from adult patients with CF treated with CFTR modulators between 2024 and 2026 was performed. Changes in lung function, sweat chloride concentration, body mass index (BMI), exocrine pancreatic function, emotional health, respiratory microbiology, and CF pulmonary exacerbations, as well as treatment safety, were assessed. Outcomes were evaluated at baseline and after 1, 3, 6, and 12 months of treatment. Results. The analysis included 21 patients with a mean age of 28.48±6.91 years. FEV1 increased as early as after one month of treatment, from 53.10±29.16% predicted to 61.75±29.57% predicted (p=0.005), while FVC increased from 65.48±24.53% predicted to 72.45±23.07% predicted (p=0.002); these improvements were sustained throughout the 12-month treatment period. Sweat chloride concentration decreased from 74.86±25.41 to 41.86±19.34 mmol/L after one month (p=0.008) and was 30.30±15.22 mmol/L after 12 months. BMI increased steadily from 20.51±2.99 to 22.42±2.72 kg/m². The median number of CF pulmonary exacerbations decreased from 1 (Q1–Q3: 0–3) to 0 (0–1) (p=0.005), while the median number of days spent in hospital due to CF pulmonary exacerbations decreased from 22 (0–53.25) to 0 (0–10) days (p=0.002). The proportion of patients in whom Staphylococcus aureus was detected at least once decreased from 85.0% to 20.0% (p<0.001), while the proportion with Pseudomonas aeruginosa decreased from 55.0% to 25.0% (p=0.031). CFTR modulators were well tolerated. Adverse events were reported in 4 patients (19.0%); they were transient and did not recur after treatment was resumed. Conclusions. One-year real-world clinical experience demonstrated that CFTR modulator therapy was associated with early and sustained improvements in lung function, a marked reduction in sweat chloride concentration, an increase in BMI, a decrease in the number of CF pulmonary exacerbations, and fewer days spent in the hospital due to exacerbations. The proportions of patients with detection of the major respiratory pathogens Staphylococcus aureus and Pseudomonas aeruginosa also decreased. Treatment was well tolerated.