Effects of CFTR-modulator triple therapy on sinunasal symptoms in children and adults with cystic fibrosis

PurposeSinunasal symptoms and chronic rhinusinutitis are common in patients with cystic fibrosis. Cystic fibrosis transmembrane regulator (CFTR) modulators have led to dramatic improvements of respiratory symptoms and quality of life in patients with cystic fibrosis. This study aims to evaluate subjective and objective sinunasal symptoms after start of CFTR-modulator triple therapy.Methods43 patients (n = 6 < 18 years), treated with highly effective CFTR-modulator therapy with elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) were included, as were 20 controls with cystic fibrosis but without CFTR-modulator therapy (n = 6 < 18 years). All assessed their sinunasal symptoms retrospectively and the intervention group at a mean of 9.3 (2-16) months after start of ELX/TEZ/IVA.ResultsImprovements in SNOT-22 overall score from m = 32.7 to m = 15.7 points (p < 0.0001) as well in the nasal, emotional, otologic, and sleep subdomains could be demonstrated in the intervention group. No changes were found in the control group. Children showed lower SNOT-22 scores than adults and a reduction of SNOT-22 total score from m = 9.4 to m = 2.2 (p = 0.25) was found. 8 patients were evaluated by an otorhinolaryngologist before and after start of ELX/TEZ/IVA and showed pronounced objective clinical improvement.ConclusionsHighly effective CFTR-modulator therapy has a significant positive impact on both subjective and objective sinunasal symptoms in patients with CF and some improvement could be demonstrated in children < 18 years as well.

Automated summary

This study aims to evaluate subjective and objective sinunasal symptoms after start of CFTR-modulator triple therapy. The results show that highly effective CFTR-modulator therapy has a significant positive impact on both subjective and objective sinunasal symptoms in patients with CF, including children.