Background Triple CFTR modulator therapy (elexacaftor/tezacaftor/ivacaftor) improves lung function, weight, exacerbation rates and quality of life in people with Cystic Fibrosis. CF is a multisystem disease and there is increasing interest in the extrapulmonary effects of CFTR modulators. Chronic rhinosinusitis and gastroesophageal reflux are common in people with CF and cause a high level of sino-nasal and laryngopharyngeal symptoms. We assessed the effect of triple CFTR modulator therapy on these symptoms in a cohort of patients with advanced CF lung disease.
Method In a prospective study, we used the Sino-Nasal Outcome Test (SNOT), the Reflux Symptom Index (RSI) and the Hull Airway Reflux Questionnaire (HARQ) as patient-reported outcome measures (PROMs) to assess the effect of triple CFTR modulators on sino-nasal and laryngoesophageal reflux symptoms. Questionnaires, lung function and weight were recorded at baseline before starting treatment and after 6 months on treatment.
Results 32 patients (23 male) starting elexacaftor/tezacaftor/ivacaftor were studied. Their baseline characteristics were mean age 34.3 (range 20–65) years, FEV1% predicted 24.8 (11–40), weight 63.2 kg (35–99.8) and BMI 21.28 kg/m2 (13.2–31.1). All patients continued with treatment throughout the study period. At 6 months there was an improvement in mean FEV1% predicted of 8.63 and BMI 2.6 kg/m2. Patient reported outcome measures showed significant improvement (table 1): median scores RSI 10, HARQ 19.5 and SNOT 16 (p<0.001 for all outcomes).
Discussion This study shows significant improvement in lung function, weight and sino-nasal and laryngopharyngeal reflux PROMs in patients with advanced CF. The SNOT-20, RSI and HARQ scores showed improvement that exceeded recognised clinically significant changes in these metrics.
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