title: Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
abstract: OBJECTIVE
  Cystic fibrosis (CF) is known for its impact on the lung and pancreas of individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may affect growth outcomes.
  
  
  METHODS
  In this post hoc analysis, we assessed linear growth and weight in 83 children (aged 6-11 years) enrolled in 2 clinical trials, the longitudinal-observation GOAL study and the placebo-controlled ENVISION study, to evaluate the effects of ivacaftor, a CFTR potentiator. We calculated height and weight z scores and height and weight growth velocities (GVs).
  
  
  RESULTS
  In ivacaftor-treated children in GOAL, height and weight z scores increased significantly from baseline to 6 months (increases of 0.1 [P < .05] and 0.26 [P < .0001], respectively); height GV increased significantly from 3 to 6 months (2.10-cm/year increase; P < .01). In ivacaftor-treated children in ENVISION, height and weight z scores increased significantly from baseline to 48 weeks (increases of 0.17 [P < .001] and 0.35 [P < .001], respectively). Height and weight GVs from baseline to 48 weeks were also significantly higher with ivacaftor than with placebo (differences of 1.08 cm/year [P < .05] and 3.11 kg/year [P < .001], respectively).
  
  
  CONCLUSIONS
  Ivacaftor treatment in prepubescent children may help to address short stature and altered GV in children with CF; results from these analyses support the existence of an intrinsic defect in the growth of children with CF that may be ameliorated by CFTR modulation.
authors.affiliation: Department of Pediatrics, mstalvey@peds.uab.edu.
authors.forename: Michael S
authors.initials: MS
authors.lastname: Stalvey
authors.affiliation: Pediatric Pulmonary Center, and.
authors.forename: Jesse
authors.initials: J
authors.lastname: Pace
authors.affiliation: Vertex Pharmaceuticals Incorporated, Boston, Massachusetts.
authors.forename: Minoo
authors.initials: M
authors.lastname: Niknian
authors.affiliation: Vertex Pharmaceuticals (Europe) Ltd, London, United Kingdom.
authors.forename: Mark N
authors.initials: MN
authors.lastname: Higgins
authors.affiliation: Department of Pediatrics.|Pediatric Pulmonary Center, and.
authors.forename: Valerie
authors.initials: V
authors.lastname: Tarn
authors.affiliation: Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama.
authors.forename: Joy
authors.initials: J
authors.lastname: Davis
authors.affiliation: Department of Pediatrics, University of Washington, Seattle, Washington; and.|CF Foundation Therapeutics Development Network, Seattle Children's Research Institute, Seattle, Washington.
authors.forename: Sonya L
authors.initials: SL
authors.lastname: Heltshe
authors.affiliation: Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama.
authors.forename: Steven M
authors.initials: SM
authors.lastname: Rowe
mesh_terms: D002648:Child; D005260:Female; D006801:Humans; D008297:Male; D000627:Aminophenols / Q000627:therapeutic use; D001827:Body Height / Q000187:drug effects; D001835:Body Weight / Q000187:drug effects; D003550:Cystic Fibrosis / Q000188:drug therapy; D004311:Double-Blind Method; D005500:Follow-Up Studies; D008137:Longitudinal Studies; D015363:Quinolones / Q000627:therapeutic use; D016896:Treatment Outcome; D017326:Clinical Trials, Phase III as Topic; D064887:Observational Studies as Topic; D016032:Randomized Controlled Trials as Topic
chemical_list: D000627:Aminophenols; C545203:ivacaftor; D015363:Quinolones