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Development of a core outcome set for paediatric achalasia: a joint ERNICA, ESPGHAN and EUPSA study protocol
Jonathan J Neville1, Iris den Uijl2, Willemijn Irvine3
1Great Ormond Street Institute of Child Health, University College London, London, UK jonathan.neville@nhs.net.
Insights
This study aims to establish a core outcome set (COS) for pediatric achalasia, standardizing outcome reporting in research for this rare disease. This will improve data comparability and facilitate meta-analyses for better treatment efficacy evaluation.
Area of Science:
- Pediatric Gastroenterology
- Rare Disease Research
- Clinical Trial Methodology
Background:
- Achalasia in children is rare, with studies often being small, retrospective, and reporting heterogeneous outcomes.
- Lack of standardized outcomes impedes meta-analysis and risks under-reporting patient-relevant data like quality of life.
- A Core Outcome Set (COS) is crucial for rare pediatric diseases to ensure consistent and relevant outcome reporting.
Purpose of the Study:
- To define a standardized Core Outcome Set (COS) for pediatric achalasia.
- To facilitate consistent outcome reporting in clinical research for pediatric achalasia.
- To enable data pooling and meta-analysis for improved understanding of treatment efficacy.
Main Methods:
- Systematic literature review of reported outcomes in pediatric achalasia studies.
- Three-stage Delphi consensus process involving healthcare professionals, patients, and parent representatives.
- Consensus meeting to finalize the Core Outcome Set (COS).
Main Results:
- The study will result in a defined Core Outcome Set (COS) for pediatric achalasia.
- The COS will be disseminated through professional societies, patient groups, and the COMET initiative website.
- The systematic review and Delphi process will identify and prioritize key outcomes.
Conclusions:
- Establishing a COS for pediatric achalasia is essential for advancing research in this rare condition.
- Standardized outcomes will enhance the quality and comparability of research data.
- This COS will guide future efficacy studies and disease registries.
Introduction:
Achalasia is a rare disease in children. Studies investigating the efficacy of interventions and disease outcomes in paediatric achalasia are predominantly retrospective, consist of small cohorts and report heterogeneous outcomes. The variation in the use and definition of reported outcomes impedes meta-analysis, which is problematic in a rare paediatric condition. Similarly, there is a risk of under-reporting patient-relevant outcomes, such as quality of life. To overcome these issues, a minimum set of important and patient-relevant outcomes should be reported in all studies of paediatric achalasia. Core outcome sets (COS) are a standardised set of outcomes that can guide further research and facilitate data pooling and meta-analysis. The development of a COS in rare paediatric disease is essential, prior to conducting efficacy studies or creating a disease registry, to ensure that the most important outcomes are reported. Currently, no COS exists for children with achalasia. In this study, we aim to define a COS for paediatric achalasia for use in clinical research.
Methods And Analysis:
This study will consist of three parts. The first will be a systematic review of the literature, evaluating the outcomes and outcome definitions reported in published clinical research studies investigating paediatric achalasia. Second, a three-stage Delphi consensus process will be undertaken to identify and prioritise outcomes. This process will involve healthcare professionals, patients and parent representatives. Third, a consensus meeting will be held, during which the final COS will be defined.
Dissemination:
The results of this study will be disseminated to stakeholders via the European Reference Network for Rare Inherited Congenital Anomalies, European Society for Pediatric Gastroenterology Hepatology and Nutrition, European Paediatric Surgeons' Association, and patient groups. The COS will be published in a peer-reviewed journal and uploaded to the Core Outcome Measures in Effectiveness Trials (COMET) initiative website.
Trial Registration Number:
The study was pre-registered with the COMET initiative in July 2024 (https://www.comet-initiative.org/Studies/Details/2568). The systematic review component of the study was pre-registered on PROSPERO (CRD42024509855).
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