Outcome reporting in studies of paediatric achalasia: A systematic review

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.

Contributors

Jonathan J. Neville, Sierra Schaffer, Simon Eaton, Nigel J. Hall

Publication

Journal: Journal of Pediatric Gastroenterology and Nurtition
Volume:
Issue:
Pages: -
Year: 2025
DOI: 10.1002/jpn3.70128

Further Study Information

Current Stage: Completed
Date:
Funding source(s): JJN is supported by funding from the NIHRGOSH BRC. The views expressed are thoseof the author(s) and not necessarily those ofthe NHS, the NIHR or the Department ofHealth

Health Area

Disease Category: Child health

Disease Name: Achalasia

Target Population

Age Range: 0 - 16

Sex: Either

Nature of Intervention: Drug, Surgery

Stakeholders Involved

Study Type

- Systematic review of outcomes measured in trials

Method(s)

- Systematic review

Linked Studies



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