title: Ivacaftor and symptoms of extra-oesophageal reflux in patients with cystic fibrosis and G551D mutation.
abstract: 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.
  
  
  
  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)).
  
  
  
  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.
  
  
  
  Ivacaftor treatment was beneficial for patients with symptoms of EOR, thought to be a precursor to microaspiration.
authors.affiliation: Institute for Cell and Molecular Bioscience, Newcastle University, Catherine Cookson Building, Framlington Place, Newcastle Upon Tyne NE2 4HH, United Kingdom.
authors.forename: Gemma L
authors.initials: GL
authors.lastname: Zeybel
authors.affiliation: Institute for Cell and Molecular Bioscience, Newcastle University, Catherine Cookson Building, Framlington Place, Newcastle Upon Tyne NE2 4HH, United Kingdom.
authors.forename: Jeffrey P
authors.initials: JP
authors.lastname: Pearson
authors.affiliation: Northern Aerodigestive group, Royal Victoria Infirmary, Newcastle upon Tyne NE1 4LP, United Kingdom.
authors.forename: Amaran
authors.initials: A
authors.lastname: Krishnan
authors.affiliation: Department of Respiratory Medicine, Royal Victoria Infirmary, Newcastle upon Tyne NE1 4LP, United Kingdom.
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Bourke
authors.affiliation: Department of Respiratory Medicine, Royal Victoria Infirmary, Newcastle upon Tyne NE1 4LP, United Kingdom.
authors.forename: Simon
authors.initials: S
authors.lastname: Doe
authors.affiliation: Department of Respiratory Medicine, Royal Victoria Infirmary, Newcastle upon Tyne NE1 4LP, United Kingdom.
authors.forename: Alan
authors.initials: A
authors.lastname: Anderson
authors.affiliation: Academic Department of Respiratory Medicine, Hull York Medical School, University of Hull, Castle Hill Hospital, Cottingham, United Kingdom.
authors.forename: Shoaib
authors.initials: S
authors.lastname: Faruqi
authors.affiliation: Academic Department of Respiratory Medicine, Hull York Medical School, University of Hull, Castle Hill Hospital, Cottingham, United Kingdom.
authors.forename: Alyn H
authors.initials: AH
authors.lastname: Morice
authors.affiliation: Northern Aerodigestive group, Royal Victoria Infirmary, Newcastle upon Tyne NE1 4LP, United Kingdom.
authors.forename: Rhys
authors.initials: R
authors.lastname: Jones
authors.affiliation: Institute for Cell and Molecular Bioscience, Newcastle University, Catherine Cookson Building, Framlington Place, Newcastle Upon Tyne NE2 4HH, United Kingdom.
authors.forename: Melissa
authors.initials: M
authors.lastname: McDonnell
authors.affiliation: Institute for Cellular Medicine, Medical School, Newcastle University, Catherine Cookson Building, Framlington Place, Newcastle Upon Tyne NE2 4HH, United Kingdom; School of Medicine, Koç University, Istanbul, Turkey.
authors.forename: Mujdat
authors.initials: M
authors.lastname: Zeybel
authors.affiliation: RD Biomed Ltd, Castle Hill Hospital, Cottingham, United Kingdom.
authors.forename: Peter W
authors.initials: PW
authors.lastname: Dettmar
authors.affiliation: Institute for Cellular Medicine, Medical School, Newcastle University, Catherine Cookson Building, Framlington Place, Newcastle Upon Tyne NE2 4HH, United Kingdom; Department of Paediatric Respiratory Medicine, Great North Children's Hospital, Queen Victoria Road, Newcastle upon Tyne NE1 4LP, United Kingdom.
authors.forename: Malcolm
authors.initials: M
authors.lastname: Brodlie
authors.affiliation: Institute for Cellular Medicine, Medical School, Newcastle University, Catherine Cookson Building, Framlington Place, Newcastle Upon Tyne NE2 4HH, United Kingdom; Northern Aerodigestive group, Royal Victoria Infirmary, Newcastle upon Tyne NE1 4LP, United Kingdom. Electronic address: Chris.Ward@newcastle.ac.uk.
authors.forename: Chris
authors.initials: C
authors.lastname: Ward
mesh_terms: D000328:Adult; D000627:Aminophenols / Q000008:administration & dosage*; D065101:Chloride Channel Agonists / Q000008:administration & dosage; D003550:Cystic Fibrosis / Q000175:diagnosis / Q000188:drug therapy / Q000235:genetics / Q000503:physiopathology; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics*; D016903:Drug Monitoring / Q000379:methods; D005260:Female; D005764:Gastroesophageal Reflux / Q000150:complications / Q000175:diagnosis / Q000503:physiopathology; D006801:Humans; D008168:Lung / Q000503:physiopathology*; D008297:Male; D009154:Mutation; D015363:Quinolones / Q000008:administration & dosage*; D053120:Respiratory Aspiration / Q000175:diagnosis / Q000209:etiology / Q000503:physiopathology / Q000517:prevention & control; D012129:Respiratory Function Tests / Q000379:methods; D016896:Treatment Outcome; D006113:United Kingdom / Q000453:epidemiology
chemical_list: D000627:Aminophenols; D065101:Chloride Channel Agonists; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor
keywords: CFTR potentiator; G551D; Gastrointestinal; Real life data