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Development of a Core Outcome Set for Infant Gastroesophageal Reflux Disease

Singendonk, Maartje M.J.*; Rexwinkel, Robyn*; Steutel, Nina F.*; Gottrand, Frederic; McCall, Lisa; Orsagh-Yentis, Danielle K.§; Rosen, Rachel||; Strisciuglio, Caterina; Thapar, Nikhil#; Vandenplas, Yvan**; Benninga, Marc A.*; Tabbers, Merit M.*

Journal of Pediatric Gastroenterology and Nutrition: May 2019 - Volume 68 - Issue 5 - p 655–661
doi: 10.1097/MPG.0000000000002245
Original Articles: Gastroenterology
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Objective: In therapeutic trials for infant gastroesophageal reflux disease (GERD), ways to define GERD and measure and report study outcomes vary widely. The aim of this study was to develop a core outcome set (COS) for infant GERD.

Methods: The COS was developed using the Delphi technique, adhering to the Outcome Measures in Rheumatology Initiative 2.0 recommendations. Healthcare professionals (HCPs) (predominantly pediatric gastroenterologists and general pediatricians) and parents of infants (age 0–12 months) with GERD, listed up to 5 primary goals of therapy from their perspective and up to 5 persistent signs or symptoms that would signify inadequate treatment. Outcomes mentioned by >10% of participants were included in 2 shortlists. Next, HCPs and parents rated and prioritized outcomes on these shortlists. Outcomes with the highest rank formed the draft COS. The final COS was created after 2 consensus meetings between an expert panel and patient representatives.

Results: In total, 125 of 165 HCPs (76%) and 139 of 143 parents (97%) of infants with GERD completed the first phase. The second phase was completed by 83 of 139 HCPs (60%) and 127 of 142 different parents (89%). Outcomes of these phases were discussed during the consensus meetings and a 9-item COS was formed: “Adequate Growth,” “Adequate Relief,” “Adverse events,”, “Crying,” “Evidence of Esophagitis,” “Feeding Difficulties,” ”Hematemesis,“ ”No Escalation of Therapy,“ and ”Sleep Problems.”

Conclusions: We developed a COS for infant GERD consisting of 9 items that should minimally be measured in future therapeutic trials to decrease study heterogeneity and ease comparability of results.

*Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Pediatric Gastroenterology, Amsterdam, The Netherlands

Division of Pediatric Gastroenterology, Hepatology and Nutrition, CHU Lille, University Lille, France.

Gastroenterology Unit, Women's and Children's Health Network, School of Medicine, North Adelaide, Australia

§Division of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Nationwide Children's Hospital, Columbus, OH

||Division of Gastroenterology, Aerodigestive Center, Children's Hospital Boston, Boston, MA

Department of Woman, Child and General and Specialistic Surgery, University of Campania “Luigi Vanvitelli,” Naples, Italy

#Great Ormond Street Hospital for Children, London, UK

**KidZ Health Castle, UZ Brussel, Vrije Universiteit Brussel, Brussels, Belgium.

Address correspondence and reprint requests to Merit M. Tabbers, MD, PhD, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, H7-228, PO Box 22700, 1100 DD Amsterdam, The Netherlands (e-mail: m.m.tabbers@amc.nl).

Received 12 December, 2018

Accepted 12 December, 2018

Maartje M.J. Singendonk and Robyn Rexwinkel: Indicates shared first authorship.

Supplemental digital content is available for this article. Direct URL citations appear in the printed text, and links to the digital files are provided in the HTML text of this article on the journal's Web site (www.jpgn.org).

This study was funded by a Networking Grant from the European Society for Pediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN).

Disclaimer: Although this article is produced by the ESPGHAN Motility Working group, it does not necessarily represent ESPGHAN policy and is not endorsed by ESPGHAN.

The authors report no conflicts of interest.

© 2019 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology,