Outcome reporting in studies of paediatric achalasia: a systematic review
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.
achalasia, core outcome set, gastroenterology, outcome measures, surgery
523-529
Neville, Jonathan J.
de26913d-d2f9-4a45-ac7c-80ab0f210927
Schaffer, Sierra
2929ed19-f727-47ee-b170-ddfbf355626f
Eaton, Simon
cb6f3a32-3e77-495a-b932-bf59700c6afa
Hall, Nigel J.
6919e8af-3890-42c1-98a7-c110791957cf
September 2025
Neville, Jonathan J.
de26913d-d2f9-4a45-ac7c-80ab0f210927
Schaffer, Sierra
2929ed19-f727-47ee-b170-ddfbf355626f
Eaton, Simon
cb6f3a32-3e77-495a-b932-bf59700c6afa
Hall, Nigel J.
6919e8af-3890-42c1-98a7-c110791957cf
Neville, Jonathan J., Schaffer, Sierra, Eaton, Simon and Hall, Nigel J.
(2025)
Outcome reporting in studies of paediatric achalasia: a systematic review.
Journal of Pediatric Gastroenterology & Nutrition, 81 (3), .
(doi:10.1002/jpn3.70128).
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.
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J pediatr gastroenterol nutr - 2025 - Neville - Outcome reporting in studies of paediatric achalasia A systematic
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Accepted/In Press date: 11 June 2025
e-pub ahead of print date: 22 June 2025
Published date: September 2025
Keywords:
achalasia, core outcome set, gastroenterology, outcome measures, surgery
Identifiers
Local EPrints ID: 503395
URI: http://eprints.soton.ac.uk/id/eprint/503395
ISSN: 0277-2116
PURE UUID: e60feb8e-ebc9-4f92-b1aa-fa0052cec934
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Date deposited: 30 Jul 2025 16:48
Last modified: 18 Sep 2025 01:44
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Author:
Jonathan J. Neville
Author:
Sierra Schaffer
Author:
Simon Eaton
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