The combination of elexacaftor, tezacaftor, and ivacaftor is effective at improving sinonasal outcomes in patients with cystic fibrosis (CF), according to results of a new study published in the Journal of Cystic Fibrosis.

The study was a pre/post, observational cohort carried out on 34 patients aged at least 12 years at 2 centers over a 9-month treatment period. Elexacaftor-tezacaftor-ivacaftor is a modulator therapy approved for individuals aged 6 years or older with CF.

“Symptoms improved within 7 days of therapy and plateaued by day 28,” the authors wrote. “Endoscopic crusting resolved and nasal polyposis improved, with a decrease in size or resolution of polyps. Sinus opacification and mucosal thickening improved on computed tomography [CT] radiographs with treatment.”

This study is unique because it includes patients (≥12 years) as well as an endoscopic evaluation of sinonasal disease. The primary endpoint was the change in the sinonasal quality of life, measured using the 22-Item Sinonasal Outcome Test scale, a patient-reported assessment of 22 sinus symptoms. The patients’ symptoms improved quickly and the improvement lasted at least 180 days.

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The origin of sinonasal disease in patients with CF is a defect in the cystic fibrosis transmembrane conductance regulator protein (CFTR). Elexacaftor, tezacaftor, and ivacaftor were approved by the US Food and Drug Administration specifically as a highly effective CFTR corrector for patients with CF who are at least 6 years old and who have at least 1 drug-sensitive allele, typically F508del.

The authors expect highly symptomatic patients with CF and lung transplant recipients with sinonasal disease or severe nasal polyps to benefit from elexacaftor, tezacaftor, and ivacaftor treatment in terms of symptoms, quality of life, and a reduced need for surgical interventions.

Patients with CF (40%-60%) experience chronic rhinosinusitis (including anosmia, congestion, rhinorrhea, and facial pain) with or without nasal polyps, and 90% have sinus mucosal thickening on CT, all of which add to their medical burden and reduce the quality of life.

Reference

Stapleton AL, Kimple AJ, Goralski, JL, et al. Elexacaftor-tezacaftor- ivacaftor improves sinonasal outcomes in cystic fibrosis. J Cyst Fibros. Published online March 14, 2022. doi:10.1016/j.jcf.2022.03.002