Peroral endoscopic myotomy for pediatric achalasia: current evidence, clinical outcomes, and long term perspectives

Overview

Pediatric achalasia is an uncommon esophageal motility disorder characterized by impaired lower esophageal sphincter relaxation and absent esophageal peristalsis. Children commonly present with dysphagia, regurgitation, chest pain, feeding difficulties, weight loss, and recurrent respiratory symptoms. Conventional therapeutic approaches include pneumatic balloon dilation, botulinum toxin injection, and laparoscopic Heller myotomy. During the last decade, peroral endoscopic myotomy (POEM) has emerged as a minimally invasive therapeutic option for both adults and children with achalasia. This narrative review summarizes the currently available evidence regarding the efficacy, safety, and long‑term outcomes of POEM in pediatric achalasia based on published clinical studies and observational cohorts. Available evidence consistently demonstrates high technical and clinical success rates, with substantial reductions in Eckardt scores and lower esophageal sphincter pressure after treatment. Most studies also report durable symptom improvement during long‑term follow up. Adverse events are generally mild and self‑limited; however, postoperative reflux and reflux esophagitis remain important concerns requiring surveillance. Current evidence suggests that POEM is an effective and relatively safe treatment option for pediatric achalasia, and may represent an attractive alternative to surgical myotomy in selected patients. Nevertheless, most available studies are retrospective and originate from specialized centers. Additional prospective multicenter studies with standardized outcome assessment and extended follow-up are still required to define the long‑term role of POEM in children.

Keywords:

peroral endoscopic myotomy – POEM – pediatric achalasia – dysphagia – esophageal motility disorders

Introduction

Achalasia is a primary esophageal motility disorder characterized by absent or severely impaired esophageal peristalsis together with defective relaxation of the lower esophageal sphincter (LES) [1–8]. Although the disorder is uncommon in childhood, its clinical consequences may be substantial because prolonged dysphagia and regurgitation can lead to malnutrition, impaired growth, recurrent respiratory complications, and reduced quality of life [1,6,7,9]. Reported annual incidence rates in children range from approximately 0.02 to 0.31 per 100,000 children [1,6–8]. Children with achalasia frequently present with progressive dysphagia, regurgitation of undigested food, chest discomfort, weight loss, nocturnal cough, and aspiration-related symptoms [1,6,7].

Historically, treatment strategies for pediatric achalasia focused on reducing LES pressure to facilitate esophageal emptying [4,9,10]. Pneumatic balloon dilation (PBD) and botulinum toxin injection have been used for many years, but repeated interventions are often required and treatment durability may be limited [9,11]. Laparoscopic Heller myotomy (LHM), frequently combined with fundoplication, has traditionally been regarded as the definitive surgical treatment for pediatric achalasia [9,10]. Although effective, LHM remains an invasive surgical procedure associated with postoperative pain, hospitalization, and potential surgical complications [9].

Peroral endoscopic myotomy (POEM) was introduced as a less invasive endoscopic alternative [12,13]. Initially developed in experimental models and later applied in humans, POEM involves the creation of a submucosal tunnel followed by selective myotomy of the circular muscle fibers extending across the gastroesophageal junction [12,13]. Experience in adults rapidly demonstrated favorable outcomes, and POEM has subsequently gained increasing acceptance in pediatric practice [1,13]. Over the past decade, several pediatric studies have evaluated the technical feasibility, symptom improvement, physiologic changes, and long-term durability of POEM [2–8,10–12,14–20].

Herein, we discuss the available literature regarding POEM in pediatric achalasia, focusing on clinical efficacy, symptom relief, manometric improvement, safety profile, reflux-related complications, quality of life, and long-term outcomes.

Technical aspects and clinical rationale of POEM

POEM is performed endoscopically under general anesthesia [2,4,12,13]. After submucosal injection, a mucosal incision is created to allow tunneling within the submucosal space. The endoscopist subsequently performs myotomy of the circular muscle fibers extending the dissection across the LES into the proximal stomach before closure of the mucosal entry with endoscopic clips [12,13]. This approach avoids external incisions while directly addressing the functional obstruction responsible for achalasia symptoms.

The minimally invasive nature of POEM has generated considerable interest in pediatric populations. Several investigators have emphasized that POEM may be particularly attractive in children because it preserves future therapeutic options, avoids abdominal incisions, and permits tailored myotomy lengths according to achalasia subtype [6,15,17]. Furthermore, the procedure can be performed even in patients with prior interventions such as balloon dilation or previous myotomy [10].

Comparative studies remain limited in children; however, existing evidence suggests that POEM provides clinical outcomes comparable to or potentially superior to traditional approaches in selected patients [11,16]. Tan and colleagues reported favorable results when comparing POEM with endoscopic balloon dilation for primary pediatric achalasia [11]. Other investigators have proposed that POEM may evolve into an important first-line treatment option in specialized centers because of its high technical success rate and relatively rapid postoperative recovery [3,5,16].

 

Clinical efficacy and symptom relief

Published pediatric studies consistently demonstrate substantial clinical improvement after POEM [2–8,10–12,14–20]. Across multiple cohorts, dysphagia, regurgitation, vomiting, and chest pain improved markedly following the procedure [3,6,8,12]. Technical success rates were generally very high, and most investigators reported successful completion of the procedure in nearly all patients [3,15,16].

One of the most commonly used outcome measures in achalasia research is the Eckardt score, which evaluates dysphagia, regurgitation, chest pain, and weight loss. Most pediatric studies reported dramatic reductions in Eckardt scores after POEM [2–8,10–12,14–20]. Kethman and colleagues observed a reduction in mean scores from approximately 7 before treatment to 2.4 after intervention [2]. Nabi and coworkers similarly demonstrated substantial decreases in symptom burden one year after POEM [4]. Long-term follow-up studies also support sustained symptom control. Peng et al. reported a decrease in mean Eckardt score from 7.67 (SD 1.62) before treatment to 0.86 (1.28) after extended follow-up exceeding five years [7]. Nabi et al. likewise reported durable symptom improvement with long-term clinical success rates approaching 95% [15].

In addition to symptomatic benefit, several studies documented improved nutritional status and growth parameters after successful treatment [6,19]. Hsu and colleagues demonstrated improvement in body mass index and growth-related indicators after POEM, emphasizing the importance of effective symptom control during childhood and adolescence [19]. These findings suggest that POEM may positively influence not only esophageal symptoms, but also overall pediatric development and nutritional recovery.

Currently available evidence also suggests that POEM remains effective in patients who previously underwent other interventions. Nabi et al. evaluated the impact of prior treatment on long-term outcomes and found that POEM remained a valuable therapeutic option even in previously treated children [10].

Manometric and physiologic outcomes

Objective physiologic improvement after POEM has been demonstrated in several studies through esophageal manometry and imaging [3,4,7,12–14,19]. LES pressure consistently decreased after treatment. Li and colleagues observed a reduction in the mean pressure from 26.8 to approximately 9 mmHg after POEM [12]. Similar findings were reported by Nabi et al. and Peng et al., both of whom documented significant long--term reductions in LES pressure [4,7].

Integrated relaxation pressure and other manometric parameters also improved after treatment, indicating restoration of esophageal emptying and relief of functional obstruction [14,19]. Radiographic and endoscopic evaluations frequently demonstrated improved esophageal emptying and enlargement of the gastroesophageal junction after the procedure [3,12]. These physiologic improvements correlate well with the symptomatic benefits observed in clinical follow-up.

Most importantly, several long-term studies suggested that these physiologic benefits remain durable over time [6,7,13,15]. In patients undergoing repeated manometric evaluation years after POEM, LES pressure remained significantly lower than preoperative values, supporting the long-term efficacy of the procedure [7].

 

Safety and adverse events

Current evidence indicates that POEM is generally safe in children when performed by experienced endoscopists in specialized centers [3,5–7,15–17]. Most reported adverse events were mild, transient, and managed conservatively without major intervention [4,6,7,18].

Gas-related events represent some of the most frequently described complications. Studies reported transient subcutaneous emphysema, capnoperitoneum, retroperitoneal air, and pneumoperitoneum [4,7,18]. These events were usually self-limited and resolved without surgical intervention [4,7]. Occasional mucosal injuries were reported, but were generally recognized immediately and managed successfully with endoscopic clip closure [4,18].

Large multicenter studies demonstrated relatively low rates of serious complications. Choné and colleagues reported significant intraoperative bleeding in only a small proportion of patients, and postoperative complications rarely required intensive care admission or surgical intervention [17]. Similarly, Wood et al. and Liu et al. reported favorable short-term safety profiles in pediatric cohorts [5,18].

Although the overall safety profile appears reassuring, postoperative gastroesophageal reflux disease (GERD) remains an important issue [5,6,15,20]. Reflux symptoms and reflux esophagitis have been documented in a notable proportion of patients after POEM [5,20]. In one multicenter study, reflux symptoms occurred in approximately 14% of patients, while postoperative endoscopy identified reflux esophagitis in a larger subgroup [5]. Most cases were mild according to the Los Angeles classification system [5]. Nevertheless, the risk of long-term reflux-related injury underscores the importance of postoperative monitoring and appropriate acid suppression therapy when indicated.

 

Long-term outcomes and quality of life

One of the major concerns in pediatric achalasia management is the durability of treatment benefits because affected children have many decades of life expectancy. Encouragingly, currently available long-term studies suggest that POEM provides sustained symptom relief in many patients [6,7,13,15,20].

Several cohorts reported follow-up periods extending beyond four or five years [6,7,13,15]. Peng et al. described excellent long-term symptom control with very low retreatment rates after a minimum follow-up of five years [7]. Nabi et al. similarly reported durable clinical success in children and adolescents after prolonged observation [15]. Bi and colleagues confirmed favorable long-term efficacy and safety in a retrospective cohort with follow-up extending beyond five years [6].

Quality-of-life assessments have also produced encouraging results. Kuipers and colleagues reported that pediatric patients treated with POEM achieved quality-of-life scores comparable to those observed in the general population and in patients treated with other therapeutic modalities [20]. More importantly, persistent symptoms were associated with significantly poorer disease--related quality of life, emphasizing the clinical relevance of sustained symptom control [20].

Despite these promising results, long--term pediatric literature remains relatively limited compared with adult studies. Most available studies originate from tertiary referral centers with substantial procedural expertise, and long-term surveillance data regarding reflux complications, recurrent dysphagia, and esophageal remodeling remain incomplete [6,7,15,20].

 

Current limitations of the evidence

Although published studies consistently support the efficacy of POEM in pediatric achalasia, several important limitations should be acknowledged. Most available reports are retrospective observational studies or case series involving relatively small numbers of patients [2–8,10–12,14–20]. Randomized controlled trials comparing POEM with LHM or pneumatic dilation in children are lacking [11,16].

Another important limitation is heterogeneity of the study design, patient selection, follow-up duration, and outcome assessment. Definitions of clinical success varied among studies, and postoperative reflux evaluation was not uniformly performed [5,15,20]. Some cohorts relied primarily on symptom assessment without standardized physiologic or endoscopic follow-up.

Publication bias is also possible because many reports originate from high-volume centers with advanced endoscopic expertise [6,13,17]. Outcomes achieved in specialized institutions may not be generalizable to all clinical settings. Furthermore, because pediatric achalasia is rare, multicenter collaboration remains essential to generate adequately powered prospective studies.

Finally, although long-term results appear encouraging, the available evidence does not yet fully clarify the lifetime durability of POEM in children or the long-term implications of postoperative reflux exposure [5,6,20].

 

Conclusion

Available evidence indicates that POEM is an effective and relatively safe treatment for pediatric achalasia. Most studies demonstrate high technical success rates together with marked improvements in dysphagia, regurgitation, Eckardt scores, and LES pressure. Current data also suggest that many children experience durable symptom relief over prolonged follow-up periods. The minimally invasive nature of POEM, combined with favorable clinical outcomes, has positioned the procedure as an important therapeutic option in pediatric achalasia management.

Nevertheless, reflux-related complications remain a relevant concern, and continued postoperative surveillance is essential. In addition, most currently available studies are retrospective and originate from specialized centers. Further prospective multicenter investigations with standardized reporting, comparative designs, and extended follow-up are required to better define the long-term role of POEM in children and adolescents with achalasia.

Abbreviations

GERD      gastroesophageal reflux disease

IRP          integrated relaxation pressure

LES          lower esophageal sphincter

LHM        laparoscopic Heller myotomy

PBD         pneumatic balloon dilation

POEM      peroral endoscopic myotomy

Submitted/Doručeno: 5. 2. 2026

Accepted/Přijato: 6. 4. 2026

 

Corresponding author

Gholam Reza Sivandzadeh, MD

Gastroenterohepatology Research Center

Shiraz University of Medical Sciences

Namazi Sq.

71937-11351 Shiraz

Iran

ghsivand@sums.ac.ir