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

J Cyst Fibros. 2017 Jan;16(1):124-131. doi: 10.1016/j.jcf.2016.07.004. Epub 2016 Jul 27.

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<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.

Keywords: CFTR potentiator; G551D; Gastrointestinal; Real life data.

Publication types

  • Observational Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aminophenols / administration & dosage*
  • Chloride Channel Agonists / administration & dosage
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
  • Cystic Fibrosis* / diagnosis
  • Cystic Fibrosis* / drug therapy
  • Cystic Fibrosis* / genetics
  • Cystic Fibrosis* / physiopathology
  • Drug Monitoring / methods
  • Female
  • Gastroesophageal Reflux* / complications
  • Gastroesophageal Reflux* / diagnosis
  • Gastroesophageal Reflux* / physiopathology
  • Humans
  • Lung / physiopathology*
  • Male
  • Mutation
  • Quinolones / administration & dosage*
  • Respiratory Aspiration* / diagnosis
  • Respiratory Aspiration* / etiology
  • Respiratory Aspiration* / physiopathology
  • Respiratory Aspiration* / prevention & control
  • Respiratory Function Tests / methods
  • Treatment Outcome
  • United Kingdom / epidemiology

Substances

  • Aminophenols
  • Chloride Channel Agonists
  • Quinolones
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • ivacaftor