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

作者：Zeybel Gemma L; Pearson Jeffrey P; Krishnan Amaran; Bourke Stephen J; Doe Simon; Anderson Alan; Faruqi Shoaib; Morice Alyn H; Jones Rhys; McDonnell Melissa; Zeybel Mujdat; Dettmar Peter W; Brodlie Malcolm; Ward Chris^*

来源：Journal of Cystic Fibrosis, 2017, 16(1): 124-131.

DOI：10.1016/j.jcf.2016.07.004

• G551D
• CFTR potentiator
• Gastrointestinal
• Real life data

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 (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)).
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 < 0.04 versus baseline) denoted by the reflux symptom index and Hull airway reflux questionnaire.
Conclusion: Ivacaftor treatment was beneficial for patients with symptoms of EOR, thought to be a precursor to microaspiration.

• 出版日期2017-1