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Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
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Novel end points for clinical trials in young children with cystic fibrosis

Shannon J Simpson1, Lauren S Mott, Charles R Esther

  • 1Telethon Institute for Child Health Research, Centre for Child Health Research, University of Western Australia, Perth, Australia.

Expert Review of Respiratory Medicine
|June 6, 2013
PubMed

Insights

New methods can now measure early cystic fibrosis (CF) lung disease in young children. Further standardization is needed for these techniques to be used in clinical trials for CF patients.

Area of Science:

  • Pulmonology
  • Pediatric Medicine
  • Biomarkers Research

Background:

  • Cystic fibrosis (CF) lung disease begins early and is a leading cause of mortality.
  • Clinical trials for new CF therapies face challenges in young children due to a lack of suitable endpoints.
  • Objective measures are needed to assess disease progression and treatment efficacy in pediatric CF patients.

Purpose of the Study:

  • To review recent advances in assessing early lung disease in young children with CF.
  • To highlight the potential of new techniques for monitoring CF progression in clinical trials.
  • To identify areas needing further development for robust pediatric CF trial endpoints.

Main Methods:

  • Review of recent literature on infant and preschool lung function testing.
  • Analysis of advancements in chest imaging techniques for pediatric CF.
  • Evaluation of biochemical biomarkers for quantifying mild lung disease in young children.

Main Results:

  • Progress has been made in quantifying mild lung disease in young children with CF using lung function tests, imaging, and biomarkers.
  • These advancements offer the possibility of objectively monitoring disease progression during interventions.
  • Current techniques require further standardization and development for reliable use in clinical trials.

Conclusions:

  • Objective measures for assessing early lung disease in young children with CF are becoming available.
  • Standardization and validation are crucial for implementing these measures in clinical trials.
  • Improved endpoints will facilitate the evaluation of new CF therapies in the pediatric population.

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