Cystic fibrosis and the role of gastrointestinal outcome measures in the new era of therapeutic CFTR modulation.

Abstract	With the development of new drugs that directly affect CFTR protein function, clinical trials are being designed or initiated for a growing number of patients with cystic fibrosis. The currently available and accepted clinical endpoints, FEV1 and BMI, have limitations. The aim of this report is to draw attention to the need and the ample possibilities for the development and validation of relevant gastrointestinal clinical endpoints for scientific evaluation of CFTR modulation treatment, particularly in young children and infants. The gastrointestinal tract offers very good opportunities to measure CFTR protein function and systematically evaluate CF related clinical outcomes based on the principal clinical gastrointestinal manifestations of CF: intestinal pH, intestinal transit time, intestinal bile salt malabsorption, intestinal inflammation, exocrine pancreatic function and intestinal fat malabsorption. We present a descriptive analysis of a variety of gastrointestinal outcome measures for clinical relevance, reliability, validity, responsiveness to interventions, feasibility in particular in young children and the availability of reference values.
Authors	Frank A J A Bodewes, Henkjan J Verkade, Jan A J M Taminiau, Drucy Borowitz, Michael Wilschanski, Working group Cystic Fibrosis and Pancreatic Disease of the European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN)
Journal	Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society (J Cyst Fibros) Vol. 14 Issue 2 Pg. 169-77 (Mar 2015) ISSN: 1873-5010 [Electronic] Netherlands
PMID	25677689 (Publication Type: Journal Article, Review)
Copyright	Copyright © 2015 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
Chemical References	Cystic Fibrosis Transmembrane Conductance Regulator
Topics	Child, Preschool Cystic Fibrosis (complications, drug therapy, genetics) Cystic Fibrosis Transmembrane Conductance Regulator (genetics) Diagnostic Techniques, Digestive System Drug Monitoring (methods) Gastrointestinal Diseases (diagnosis, etiology, metabolism, physiopathology) Genetic Therapy (methods) Humans Infant Outcome Assessment, Health Care Reproducibility of Results

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