Ivacaftor and symptoms of extra-oesophageal reflux in patients with cystic fibrosis and G551D mutation

Abstract Background Extra-oesophageal reflux (EOR) may lead to microaspiration in patients with cystic fibrosis (CF), a probable cause of deteriorating lung function. Successful clinical trials of ivacaftor highlight opportunities to understand EOR in a real world study. Methods Data from 12 patients with CF and the G551D mutation prescribed ivacaftor (150mg bd) was collected at baseline, 6, 26 and 52weeks. The changes in symptoms of EOR were assessed by questionnaire (reflux symptom index (RSI) and Hull airway reflux questionnaire (HARQ)). Results Six patients presented EOR at baseline (RSI >13; median 13; range 2–29) and 5 presented airway reflux (HARQ >13; median 12; range 3 to 33). Treatment with ivacaftor was associated with a significant reduction of EOR symptoms (P Conclusion Ivacaftor treatment was beneficial for patients with symptoms of EOR, thought to be a precursor to microaspiration.