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.