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Development of a Core Outcome Set for Infant Gastroesophageal Reflux Disease
Maartje M J Singendonk1, Robyn Rexwinkel1, Nina F Steutel1
1Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Pediatric Gastroenterology, Amsterdam, The Netherlands.
Insights
A new core outcome set (COS) for infant gastroesophageal reflux disease (GERD) was developed. This standardized set of 9 outcomes will improve consistency in infant GERD therapeutic trials.
Area of Science:
- Pediatric Gastroenterology
- Clinical Trial Methodology
- Outcome Measurement
Background:
- Therapeutic trials for infant gastroesophageal reflux disease (GERD) lack standardized outcome measures.
- This heterogeneity complicates the comparison of study results and hinders evidence-based practice.
Purpose of the Study:
- To develop a consensus-based core outcome set (COS) for infant GERD.
- To reduce study heterogeneity and improve comparability in future infant GERD therapeutic trials.
Main Methods:
- The Delphi technique and consensus meetings were employed, following the Outcome Measures in Rheumatology Initiative 2.0 recommendations.
- Healthcare professionals and parents of infants with GERD identified and prioritized key therapeutic goals and treatment failure indicators.
- A 9-item COS was finalized through expert and patient representative consensus.
Main Results:
- A 9-item core outcome set (COS) for infant GERD was established.
- The COS includes: Adequate Growth, Adequate Relief, Adverse events, Crying, Evidence of Esophagitis, Feeding Difficulties, Hematemesis, No Escalation of Therapy, and Sleep Problems.
- High participation rates were achieved from healthcare professionals (76%) and parents (97% in phase 1).
Conclusions:
- A standardized 9-item COS for infant GERD has been developed.
- This COS will serve as a minimum standard for outcome measurement in infant GERD therapeutic trials.
- Implementation of this COS is expected to decrease study heterogeneity and facilitate result comparability.
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.
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