Well-defined and reliable clinical outcome assessments for pediatric Crohn disease: a critical need for drug development

Journal of Pediatric Gastroenterology and Nutrition
Haihao SunAndrew E Mulberg

Abstract

The aim of the present study was to identify areas for further development of clinical outcome assessment (COA) in pediatric Crohn disease (CD). The study analyzed the measurement properties of all existing COA tools for pediatric CD in literature and published registration trials of approved drugs for pediatric CD based on criteria described in Food and Drug Administration guidance for patient-reported outcome (PRO) development. The Pediatric Crohn's Disease Activity Index (PCDAI) and its derivatives (abbreviated, short, modified, and weighted PCDAIs) were reviewed. The Crohn's Disease Activity Index (CDAI) and Harvey-Bradshaw index (HBI), designed for adult patients, have been adapted for use in a few pediatric CD studies. The use of PCDAI as an endpoint in Remicade and Humira trials led to the Food and Drug Administration-approved indication in pediatric CD. Common issues in measurement properties of COA tools included the absence of direct patient or caregivers' input to generate the items measuring signs and symptoms; absence of evidence demonstrating correlation with clinically relevant inflammation observed with endoscopic measures; lack of standardization in measurement, age-appropriate interviewer script, and response ...Continue Reading

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Citations

Aug 24, 2017·Journal of Pediatric Gastroenterology and Nutrition·Kay DiederenAngelika Kindermann
Jun 3, 2016·Journal of Pediatric Gastroenterology and Nutrition·Haihao SunUNKNOWN International Inflammatory Bowel Disease (i-IBD) Working Group
Aug 3, 2017·Clinical Trials : Journal of the Society for Clinical Trials·Virginia KwitkowskiPaul Kluetz

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