Effects of Elexacaftor-Tezacaftor-Ivacaftor on Nasal and Sinus Symptoms in Children With Cystic Fibrosis.

Abstract

Background: New CFTR Modulator triple therapy Elexacaftor-Ivacaftor-Tezacaftor (ETI) prove efficacy in pulmonary outcomes. However, its impact on nasal sinus symptoms in children has not been specifically studied. The aim of this study is to evaluate the impact of this therapy on nasal sinus symptomatology in children aged 6-12 years.

Methods: A prospective, single-center cohort study was conducted over a 12-month follow-up period in children aged 6-12 years at the initiation of ETI therapy. The primary outcome was evolution of the SN-5 score, a validated pediatric questionnaire measuring quality of life related to nasal sinus symptoms. A decrease of 0.5 points is considered clinically significant. Secondary outcomes included changes in clinical examination findings (obstructive turbinate hypertrophy, polyps, presence of pus in the middle meatus, and externalized mucocele), quality of life measured by the Visual Analog Scale (VAS), and number of antibiotic courses during the study period.

Results: Twenty-six patients were included between March and September 2023, with no lost to follow-up. The initial mean SN-5 score was 2.88 (95% CI {1.91; 3.85}). After 1 year, the mean SN-5 score was significantly lower (1.41, 95% CI {1.00; 1.88}, Delta = 1.47, p < 0.001). The VAS related to symptoms also improved (Delta = 1.7, p < 0.001), and the number of antibiotic courses decreased (25 vs. 69, p < 0.001). A trend toward improvement in clinical examination parameters was observed.

Conclusion: ETI therapy appears to significantly improve nasal sinus symptoms in children aged 6-12 years, as evidenced by improved quality-of-life scales and reduced antibiotic use.