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

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• 作者：Gemma L. Zeybel^a ; Jeffrey P. Pearson^a ; Amaran Krishnan^c ; Stephen J. Bourke^d ; Simon Doe^d ; Alan Anderson^d ; Shoaib Faruqi^e ; Alyn H. Morice^e ; Rhys Jones^c ; Melissa McDonnell^a ; Mujdat Zeybel^b ; ^f ; Peter W. Dettmar^g ; Malcolm Brodlie^b ; ^h ; Chris Ward^b ; ^c ; ^{Chris.Ward@newcastle.ac.uk}
• 关键词：G551D ; CFTR potentiator ; Gastrointestinal ; Real life data
• 刊名：Journal of Cystic Fibrosis
• 出版年：2017
• 出版时间：January 2017
• 年：2017
• 卷：16
• 期：1
• 页码：124-131
• 全文大小：472 K
• 卷排序：16

文摘

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.MethodsData from 12 patients with CF and the G551D mutation prescribed ivacaftor (150 mg bd) was collected at baseline, 6, 26 and 52 weeks. The changes in symptoms of EOR were assessed by questionnaire (reflux symptom index (RSI) and Hull airway reflux questionnaire (HARQ)).ResultsSix 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 < 0 ∙ 04 versus baseline) denoted by the reflux symptom index and Hull airway reflux questionnaire.ConclusionIvacaftor treatment was beneficial for patients with symptoms of EOR, thought to be a precursor to microaspiration.