# Management and outcomes of paediatric achalasia: multicentre retrospective study in the UK

## TL;DR

This study examines the treatment and outcomes of children with achalasia in the UK, finding that Heller’s cardiomyotomy has higher success rates but also more complications.

## Contribution

The study provides insights into management variation and treatment outcomes for pediatric achalasia in the UK.

## Key findings

- Heller’s cardiomyotomy had a higher success rate (72%) compared to endoscopic balloon dilatation (6%).
- Many children remain symptomatic after treatment, requiring multiple interventions.

## Abstract

Achalasia is rare disease in children and young people (CYP) that causes significant symptoms and often requires invasive interventions. There is currently no consensus on the optimal management strategy. This study investigated the current management and outcomes of CYP with achalasia in the UK.

A retrospective study was conducted of CYP (aged ≤ 16 years) diagnosed with achalasia between 2011 and 2021 in the UK. The study was co-designed with the patient group Achalasia Action. Data were collected from patient records. The primary outcome was treatment success.

In all, 126 patients were included from 13 UK centres; 64 of the patients (50.8%) were male and the median age at diagnosis was 12 (interquartile range (i.q.r.) 9–14) years. The most frequent presenting features were dysphagia (73.8%), vomiting (53.2%), and weight loss (38.9%). The median time from symptom onset to diagnosis was 11 (i.q.r. 6–24) months. Treatment success was achieved in 55 of 120 patients (45.8%) after first-line intervention. Heller’s cardiomyotomy (HCM) as the first-line intervention had a higher success rate than endoscopic balloon dilatation (EBD; (52 of 72 (72%) versus 3 of 48 (6%), respectively; P < 0.001). However, overall HCM had a higher frequency of complications than EBD (17 of 98 (17%) versus 3 of 57 (5%), respectively; P = 0.045). In the entire cohort, 53% of patients reported symptoms at the 1-year follow-up.

Variation exists in the management of CYP with achalasia in the UK. The highest rates of treatment success were associated with HCM. Many CYP remain symptomatic after treatment and require multiple interventions. The present data can be used to inform management decisions in CYP with achalasia.

Achalasia is rare disease in children and young people (CYP) that causes significant symptoms and often requires invasive interventions. This retrospective study of 126 CYP with achalasia diagnosed between 2011 and 2021 at 13 centres in the UK found variation in the management of CYP with achalasia. The highest rates of treatment success were associated with Heller’s cardiomyotomy, but many CYP remain symptomatic after treatment and require multiple interventions.