title: The predictive potential of the sweat chloride test in cystic fibrosis patients with the G551D mutation.
abstract: BACKGROUND
  Ivacaftor, a cystic fibrosis transmembrane regulator (CFTR) potentiator, decreased sweat chloride concentrations and improved clinical measures in cystic fibrosis (CF) patients with the G551D mutation.
  
  
  RESULTS
  Sweat chloride measurements at day 15 had an overall positive predictive value (PPV) of 86.3%, a negative predictive value (NPV) of 65.5%, sensitivity of 73.9%, and specificity of 80.9% for an FEV1 improvement of ≥5% from baseline at week 16. For ivacaftor patients the median FEV1 improvement was 16.7%; for placebo patients 0.4%. For patients aged 6-11 years who received ivacaftor and who had a sweat chloride decrease of ≥40 mmol/L from baseline at day 15, a median weight gain of 11.2% at week 16, compared to 6% for those with a smaller decrease was observed.
  
  
  CONCLUSIONS
  Changes in sweat chloride concentration at day 15 following treatment with ivacaftor may have sufficient predictive potential to identify individuals that show improvement in pulmonary function and weight gain after 16 weeks of treatment.
authors.affiliation: Vertex Pharmaceuticals Incorporated, 130 Waverly Street, Cambridge, MA 02139-4242, USA; Faculty of Medicine, University of Ulm, Albert-Einstein-Allee 7, 89081 Ulm, Germany. Electronic address: verena_seliger@vrtx.com.
authors.forename: Verena I
authors.initials: VI
authors.lastname: Seliger
authors.forename: David
authors.initials: D
authors.lastname: Rodman
authors.forename: Fredrick
authors.initials: F
authors.lastname: Van Goor
authors.forename: Andreas
authors.initials: A
authors.lastname: Schmelz
authors.forename: Peter
authors.initials: P
authors.lastname: Mueller
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000627:Aminophenols / Q000627:therapeutic use*; D002648:Child; D002712:Chlorides / Q000032:analysis*; D003550:Cystic Fibrosis / Q000188:drug therapy / Q000235:genetics*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D005260:Female; D005541:Forced Expiratory Volume; D006801:Humans; D008297:Male; D009154:Mutation; D015363:Quinolones / Q000627:therapeutic use*; D012680:Sensitivity and Specificity; D013542:Sweat / Q000737:chemistry*; D015430:Weight Gain
chemical_list: D000627:Aminophenols; C505032:CFTR protein, human; D002712:Chlorides; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor
keywords: Clinical outcomes; FEV(1); Ivacaftor; PPV; Weight