Development of a Core Outcome Set for Children Aged 1-18 Years with Gastroesophageal Reflux Disease

Robyn Rexwinkel,Vera W.A Van Berge Henegouwen,Maartje M.J Singendonk,Usha Krishnan,Yvan Vandenplas,Caterina Strisciuglio,Rachel Rosen,Arnaud Lalanne,Shaman Rajindrajith,Marc A Benninga,Merit M Tabbers

doi:10.1016/j.jpeds.2022.01.043

Abstract

To develop a core outcome set for clinical studies assessing gastroesophageal reflux disease (GERD) in children. This core outcome set was developed using a 2-round Delphi technique and adhering to the Outcome Measures in Rheumatology Initiative (OMERACT 2.0) recommendations. Healthcare professionals (HCPs) and (parents of) children (age 1-18years) with a GERD diagnosis (ie, the presence of bothersome symptoms), listed up to 5 harmful and/or beneficial outcomes that they considered important in the treatment of GERD. Outcomes mentioned by more than 10% of participants were put forward and rated and prioritized by HCPs, parents, and children in a second round. Outcomes with the highest rank formed the draft core outcome set. The final core outcome set was created during an online consensus meeting between an expert panel. The first round was completed by 118 of 125 HCPs (94%), 146 of 146 parents (100%), and 69 of 70 children (99%). A total of 80 of 118 HCPs (68%), 130 of 140 parents (93%), and 77 children (100%) completed round 2. "Adequate relief," "evidence of esophagitis," "feeding difficulties," "heartburn (≥4years)," "hematemesis," "regurgitation," "sleeping difficulties," "vomiting," and "adverse events" were included in the final core outcome set for GERD in children aged 1-18years. We identified a total set of 9 core outcomes and suggest these outcomes to be minimally measured in clinical studies assessing GERD in children. Implementation of this core outcome set is likely to increase comparison between studies and may thus provide future recommendations to improve treatment of GERD in children.

Highlights

We identified a total set of nine core outcomes and suggest these outcomes to be minimally measured in clinical studies assessing Gastroesophageal reflux disease (GERD) in children
Gastroesophageal reflux disease (GERD) is a complex disease with a heterogeneous symptom profile, and is clinically defined as gastroesophageal reflux (GER) causing bothersome symptoms and/or complications based on international expert consensus.[1]
Journal Pre-proof Comparability of clinical trials on GERD in children is hampered as they do not share a uniform set of outcome measures, assessment time points nor measurement tools, even when they are addressing similar clinical questions.[2,3,4] outcomes in clinical trials are often chosen to meet the needs of researchers and results may be statistically significant, this does not directly imply clinical relevance

Summary

Introduction

This is a PDF file of an article that has undergone enhancements after acceptance, such as the addition of a cover page and metadata, and formatting for readability, but it is not yet the definitive version of record. This version will undergo additional copyediting, typesetting and review before it is published in its final form, but we are providing this version to give early visibility of the article. Objective to develop a core outcome set (COS) for clinical studies assessing GERD in children.

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