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Outcome reporting in studies of paediatric achalasia: A systematic review

Neville, Jonathan J; Schaffer, Sierra; Eaton, Simon; Hall, Nigel J; (2025) Outcome reporting in studies of paediatric achalasia: A systematic review. Journal of Pediatric Gastroenterology and Nutrition , 81 (3) pp. 523-529. 10.1002/jpn3.70128. Green open access

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Abstract

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.

Type: Article
Title: Outcome reporting in studies of paediatric achalasia: A systematic review
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1002/jpn3.70128
Publisher version: https://doi.org/10.1002/jpn3.70128
Language: English
Additional information: This work is licensed under a Creative Commons License. The images or other third-party material in this article are included in the Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
Keywords: Science & Technology, Life Sciences & Biomedicine, Gastroenterology & Hepatology, Nutrition & Dietetics, Pediatrics, achalasia, core outcome set, gastroenterology, outcome measures, surgery, PERORAL ENDOSCOPIC MYOTOMY, ESOPHAGEAL ACHALASIA, BALLOON DILATION, HELLER MYOTOMY, CHILDREN, EFFICACY, DILATATION
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Biology and Cancer Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10219022
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