title: Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up.
abstract: OBJECTIVE
  Ivacaftor is a revolutionary treatment option for cystic fibrosis (CF) patients with G551D and other gating mutations. The aim of this study was to evaluate the clinical status of patients on ivacaftor who were followed for up to 6 years together with an evaluation of ivacaftor therapy in one patient with an initial FEV1 less than 40% of predicted value.
  
  
  METHODS
  Data on development of clinical status and sinopulmonary-related therapies were obtained from patient health records during ivacaftor treatment lasting for up to six years and were compared with an equivalent period before ivacaftor administration.
  
  
  RESULTS
  Five CF adults with a median age 28.6 years (range 21.4-35.6 years) with median FEV1 45% pred. (range 16-85% pred.) were included in the study. Four subjects were also participants in the STRIVE and PERSIST studies. Altogether, twenty-four patient-years of ivacaftor treatment were analyzed. The median FEV1 decline per year decreased from -4.5 to -0.9% pred. (P = 0.043). Reduction in number of days on antibiotic treatment and hospital stays was 21% (P < 0.001) and 75% (P = 0.003), respectively. Improvement and stabilization of lung function was observed for up to six years of treatment. In a patient with severe airway obstruction, an increase in the FEV1 value (30.4% from baseline) was documented during the first twelve months of treatment.
  
  
  CONCLUSIONS
  Ivacaftor therapy resulted in improved and stabilized lung function in up to six years of treatment with a reduction in number of days on antibiotic treatment and hospital stays. Its efficiency was also displayed in a patient with severe airway obstruction.
authors.affiliation: Department of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic.|Cystic Fibrosis Centre, University Hospital Motol, Prague, Czech Republic.
authors.forename: Libor
authors.initials: L
authors.lastname: Fila
authors.affiliation: Department of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic.|Cystic Fibrosis Centre, University Hospital Motol, Prague, Czech Republic.
authors.forename: Lucie
authors.initials: L
authors.lastname: Valentova Bartakova
authors.affiliation: Department of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic.|Cystic Fibrosis Centre, University Hospital Motol, Prague, Czech Republic.
authors.forename: Alzbeta
authors.initials: A
authors.lastname: Grandcourtova
authors.affiliation: Department of Pneumology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic.
authors.forename: Miloslav
authors.initials: M
authors.lastname: Marel
authors.affiliation: Division of Health Insurance, University Hospital Motol, Prague, Czech Republic.
authors.forename: Radovan
authors.initials: R
authors.lastname: Drnek
authors.affiliation: Cystic Fibrosis Centre, University Hospital Motol, Prague, Czech Republic.|Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic.
authors.forename: Alena
authors.initials: A
authors.lastname: Bilkova
authors.affiliation: Cystic Fibrosis Centre, University Hospital Motol, Prague, Czech Republic.|Department of Biology and Medical Genetics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic.
authors.forename: Milan
authors.initials: M
authors.lastname: Macek
authors.affiliation: Cystic Fibrosis Centre, University Hospital Motol, Prague, Czech Republic.|Department of Medical Microbiology, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic.
authors.forename: Pavel
authors.initials: P
authors.lastname: Drevinek
mesh_terms: D000328:Adult; D000359:Aftercare; D000627:Aminophenols / Q000627:therapeutic use*; D065101:Chloride Channel Agonists / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000503:physiopathology; D005260:Female; D005541:Forced Expiratory Volume / Q000502:physiology; D006801:Humans; D008297:Male; D015363:Quinolones / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000627:Aminophenols; D065101:Chloride Channel Agonists; D015363:Quinolones; C545203:ivacaftor
keywords: adults; cystic fibrosis; ivacaftor; lung disease