title: CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation.
abstract: To investigate the effect of treatment with ivacaftor on insulin secretion in patients with cystic fibrosis (CF) (ΔF508\S549R) having CFRD/impaired insulin secretion.
  
  
  
  A standard OGTT was performed before and after 16weeks of treatment with ivacaftor in 2 sibling patients with CF carrying the S549R gating mutation. The area under the curve (AUC) for glucose and insulin was calculated using the trapezoidal estimation.
  
  
  
  Before treatment, the OGTT of case 1 showed indeterminate glycemia; the OGTT of case 2 indicated CFRD. After ivacaftor treatment the OGTT demonstrated improved insulin secretion pattern mainly by increased first phase early insulin secretion, resulting in reduction of the AUC of glucose in both cases.
  
  
  
  The treatment with ivacaftor in patients with CF carrying gating mutation can ameliorate impaired insulin secretion. Further studies and larger cohorts are needed to evaluate the impact of ivacaftor on insulin secretion in patients with CF carrying gating or other mutations.
authors.affiliation: Division of Pediatric Pulmonology and CF center, Jerusalem 91240, Israel.
authors.forename: Reuven
authors.initials: R
authors.lastname: Tsabari
authors.affiliation: Division of Pediatric Pulmonology and CF center, Jerusalem 91240, Israel.
authors.forename: Hila Iron
authors.initials: HI
authors.lastname: Elyashar
authors.affiliation: Division of Pediatric Pulmonology and CF center, Jerusalem 91240, Israel.
authors.forename: Malena Cohen
authors.initials: MC
authors.lastname: Cymberknowh
authors.affiliation: Division of Pediatric Pulmonology and CF center, Jerusalem 91240, Israel.
authors.forename: Oded
authors.initials: O
authors.lastname: Breuer
authors.affiliation: Division of Pediatric Pulmonology and CF center, Jerusalem 91240, Israel.
authors.forename: Shoshana
authors.initials: S
authors.lastname: Armoni
authors.affiliation: Israel CF center, Carmel Medical Center, Haifa, Israel.
authors.forename: Galit
authors.initials: G
authors.lastname: Livnat
authors.affiliation: Division of Pediatric Pulmonology and CF center, Jerusalem 91240, Israel.
authors.forename: Eitan
authors.initials: E
authors.lastname: Kerem
authors.affiliation: Division of Pediatric Endocrinology, Hadassah Hebrew University Medical Center, Jerusalem 91240, Israel. Electronic address: zangend@hadassah.org.il.
authors.forename: David Haim
authors.initials: DH
authors.lastname: Zangen
mesh_terms: D000627:Aminophenols / Q000008:administration & dosage / Q000493:pharmacokinetics; D001786:Blood Glucose / Q000032:analysis; D065101:Chloride Channel Agonists / Q000008:administration & dosage / Q000493:pharmacokinetics; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D005260:Female; D006801:Humans; D007328:Insulin / Q000378:metabolism*; D000078790:Insulin Secretion; D008297:Male; D009154:Mutation; D015363:Quinolones / Q000008:administration & dosage / Q000493:pharmacokinetics; D035781:Siblings; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000627:Aminophenols; D001786:Blood Glucose; D065101:Chloride Channel Agonists; D007328:Insulin; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor
keywords: CFRD; CFTR potentiator; Cystic fibrosis; Gating mutation; Impaired OGTT; Ivacaftor