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Outcome reporting in studies of paediatric achalasia: A systematic review
Jonathan J Neville1, Sierra Schaffer2, Simon Eaton1
1Great Ormond Street Institute of Child Health, University College London, London, UK.
Insights
Developing a core outcome set for pediatric achalasia is crucial. Current studies lack standardized reporting, with under-reported patient-relevant outcomes like survival, necessitating a new approach for better research and care.
Area of Science:
- Pediatric Gastroenterology
- Clinical Trial Methodology
Background:
- Pediatric achalasia is a rare condition with significant morbidity.
- Standardized reporting is needed to compare data and improve research for children with achalasia.
Purpose of the Study:
- To identify outcomes currently reported in pediatric achalasia studies.
- To inform the development of a core outcome set (COS) for pediatric achalasia.
Main Methods:
- A systematic review following PRISMA guidelines was conducted.
- Studies included children (≤18 years) diagnosed with achalasia.
- Outcomes were recorded and categorized using OMERACT core areas.
Main Results:
- Sixty-two studies (54 retrospective, 8 prospective) were analyzed.
- Forty-eight unique outcomes were reported; common ones included intra-operative complications, post-operative complications, and length of stay.
- Few studies specified a primary outcome, often using the unvalidated Eckardt score in children. Outcomes related to pathophysiology, life impact, and survival were under-reported.
Conclusions:
- Studies on pediatric achalasia are often small and retrospective.
- The Eckardt score, commonly used as a primary outcome, is unvalidated in pediatric populations.
- A stakeholder-involved COS is needed to ensure reporting of patient-relevant outcomes, reduce heterogeneity, and facilitate meta-analysis.
Objectives:
Paediatric achalasia is a rare condition associated with significant morbidity. A core outcome set (COS) would standardise reporting, enable comparison of data sets, and focus research efforts; ultimately improving care for children with achalasia. We aimed to identify outcomes currently reported in studies of paediatric achalasia to inform outcomes for a COS.
Methods:
A systematic review was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-analysis guidelines. Studies investigating children ≤18 years of age with a diagnosis of achalasia were included. Primary and secondary outcomes were recorded and assigned to OMERACT core areas. The study was pre-registered (PROSPERO: CRD42024509855).
Results:
Sixty-two studies were included in this review, consisting of 54 retrospective and 8 prospective studies. Median cohort size was 20 patients (inter-quartile range: 13-28). Forty-eight unique outcomes were reported. The most common outcomes reported were intra-operative complications (65%, 40 studies), post-operative complications (58%, 36 studies) and length of stay (58%, 36 studies). A primary outcome was specified in 12 studies (19%), the most common was the Eckardt score (13%) in 8 studies. Studies least frequently reported outcomes in the death (21%, 13 studies) and pathophysiological manifestations (35%, 22 studies) core areas.
Conclusions:
The studies included in this review were predominantly small and retrospective. Of the few studies that specified a primary outcome, the majority used the Eckardt score, which is unvalidated in children. Outcomes relevant to pathophysiological manifestations, life impact and survival were under-reported. A COS for paediatric achalasia, involving key stakeholders, would ensure that patient-relevant outcomes were reported, reduce heterogeneity and facilitate meta-analysis.
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