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Updated: Mar 8, 2026

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients
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Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients

Published on: October 15, 2017

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Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor

Michael S Stalvey1, Jesse Pace2, Minoo Niknian3

  • 1Department of Pediatrics, mstalvey@peds.uab.edu.

Pediatrics
|February 2, 2017
PubMed

Insights

Ivacaftor treatment improved growth in children with cystic fibrosis (CF). This CFTR modulator therapy may help address short stature and growth velocity issues in affected children.

Area of Science:

  • Pediatric Endocrinology
  • Genetic Disorders
  • Pharmacology

Background:

  • Cystic fibrosis (CF) commonly causes impaired growth in children, alongside lung and pancreatic issues.
  • The biological defect in the CF transmembrane conductance regulator (CFTR) protein is implicated in CF complications.
  • Targeting the CFTR protein defect may offer a therapeutic strategy for growth abnormalities in CF.

Purpose of the Study:

  • To evaluate the effects of ivacaftor, a CFTR potentiator, on linear growth and weight in children with CF.
  • To assess if targeting the CFTR protein defect can improve growth outcomes in prepubescent children with CF.

Main Methods:

  • Post hoc analysis of 83 children (aged 6-11 years) from two clinical trials (GOAL and ENVISION).
  • Assessment of height and weight z scores and growth velocities (GVs) in children treated with ivacaftor.
  • Comparison of growth parameters between ivacaftor-treated groups and placebo (in ENVISION study).

Main Results:

  • Ivacaftor treatment significantly increased height and weight z scores in children from baseline in both GOAL and ENVISION studies.
  • Significant increases in height growth velocity were observed with ivacaftor treatment.
  • Height and weight GVs were significantly higher in ivacaftor-treated children compared to placebo.

Conclusions:

  • Ivacaftor therapy shows potential in addressing short stature and altered growth velocity in prepubescent children with CF.
  • These findings suggest an intrinsic growth defect in CF that can be improved by CFTR modulation.
  • CFTR potentiation represents a promising therapeutic avenue for growth disturbances in cystic fibrosis.
Abstract

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