Long-term outcomes of peroral endoscopic myotomy for achalasia in pediatric patients: a prospective, single-center study

doi:10.1016/j.gie.2014.06.035

Gastrointestinal Endoscopy

Volume 81, Issue 1, January 2015, Pages 91-100

https://doi.org/10.1016/j.gie.2014.06.035 Get rights and content

Background

Peroral endoscopic myotomy (POEM) has been developed to provide a less-invasive myotomy for achalasia in adults but seldom has been used in pediatric patients.

Objective

To evaluate the feasibility, safety, and efficacy of POEM for pediatric patients with achalasia.

Design

Single-center, prospective study.

Setting

Academic medical center.

Patients

A total of 27 pediatric patients (mean age 13.8 years, range 6-17 years) with achalasia.

Interventions

POEM.

Main Outcome Measurements

The primary outcome was symptom relief during follow-up, defined as an Eckardt score of ≤3. Secondary outcomes were procedure-related adverse events, clinical reflux adverse events, and lower esophageal sphincter (LES) pressure on manometry before and after POEM.

Results

A total of 26 cases (96.3%) underwent successful POEM. A submucosal tunnelling attempt failed in 1 case because of serious inflammation and adhesion. No serious adverse events related to POEM were encountered. During a mean follow-up period of 24.6 months (range 15-38 months), treatment success was achieved in all patients (mean score before vs after treatment 8.3 vs 0.7; P < .001). Mean LES pressure also decreased from a mean of 31.6 mm Hg to 12.9 mm Hg after POEM (P < .001). Five patients developed clinical reflux adverse events (19.2%).

Limitations

Single center and lack of some objective evaluations.

Conclusion

This relatively long-term follow-up study adds to the evidence that POEM seems to be a promising new treatment for pediatric patients with achalasia, resulting in long-term symptom relief in all cases and without serious adverse events.

Section snippets

Patients

After institutional review board approval was obtained (approval number 2010-135, ethical committee of Zhongshan Hospital, Fudan University), the study included consecutive patients in the Endoscopy Center and Endoscopy Research Institute, Zhongshan Hospital, Fudan University between August 2010 and July 2012. The study also has been registered in ClinicalTrials.gov (identifier: NCT01649843). Patients aged <18 years were eligible for enrollment in the study if they reported persistence of

Patient characteristics

As shown in Table 1, the study cohort consisted of 11 boys and 16 girls, aged 6 to 17 years (mean 13.8 years). The mean duration of symptoms before they underwent POEM was 1.7 years (range 0.2-3 years). Nine patients presented with severe growth retardation and weight at less than the third percentile curve, 13 patients presented with weight at the third to 25th percentile curve, whereas 5 patients presented with weight at the 25th to 75th percentile curve. The mean total weight loss to patient

Discussion

The optimal management of achalasia in children and adolescents remains unclear, and the literature lacks standardized technical procedures and follow-up protocols. Currently, treatments for pediatric achalasia can be endoscopic or surgical. Treatments such as intraesophageal botulinum toxin injection and pneumatic dilation alone are unlikely to provide long-term resolution of symptoms, with most patients clinically relapsing, warranting repeated procedures, and inevitably necessitating

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Cited by (99)

Effect of peroral endoscopic myotomy on growth and esophageal motility for pediatric esophageal achalasia
2024, Journal of the Formosan Medical Association
Peroral endoscopic myotomy (POEM), a novel minimally invasive treatment for esophageal achalasia, has been shown to be effective and safe for both adult and pediatric patients. However, studies on its application in children in Taiwan and its impact on growth and esophageal motility are lacking.
We conducted a retrospective study on consecutive pediatric patients who were diagnosed with esophageal achalasia at National Taiwan University Hospital and underwent POEM during 2015–2022. Disease characteristics and treatment outcomes were analyzed.
Ten patients (age 16.9 ± 3.1 years), nine newly diagnosed and one previously treated with pneumatic dilatation, underwent POEM for achalasia (type I/II/III: 3/7/0). Average symptom duration before diagnosis was 19.4 ± 19.9 months, mean POEM procedure time was 83.6 ± 30.7 min, and clinical success (Eckardt score ≤3) was achieved in all patients. Eight patients experienced mild adverse events during POEM, but none required further endoscopic or surgical intervention. Over a mean follow-up period of 3.7 ± 1.6 years, mean Eckardt score decreased significantly from 5.7 ± 2.4 to 1.1 ± 0.7 (p = 0.0001). The BMI z-score also increased significantly after POEM (p = 0.023). Five patients received follow-up high-resolution impedance manometry (HRIM), and all had improved lower esophageal sphincter resting pressures (p = 0.011), body contractility, and bolus transit (p = 0.019).
POEM is an effective and safe treatment for pediatric achalasia in Taiwan. Early diagnosis and treatment with POEM may help to restore esophageal function and nutrition status in children.
Lessons learned from early experience of per oral endoscopic myotomy in children
2023, Journal of Pediatric Surgery
Achalasia cardia (AC) is rare in children, and the standard treatment is open or Laparoscopic Heller's myotomy with or without fundoplication if pneumatic dilatation has failed. Per oral endoscopic myotomy (POEM) is a novel technique for management of achalasia with good results in adults. We report POEM in four children and the outcome with special emphasis on the technique and management of intra-operative complications.
Four children aged 7–15 years presenting with progressive dysphagia, cough, night-time aspirations, and weight loss of six months to one year were investigated with upper GI contrast study, flexible endoscopy and biopsy, oesophageal manometry, and a diagnosis of Type 1 & 2 AC was made. An experienced adult endoscopist in collaboration with the paediatric surgical team performed POEM.
POEM was performed successfully using ERBE HYBRID knife setup and waterjet injection for the submucosal tunnelling. Operative time was 25–40 min (mean 31 min). The hospital stay was 3–8 days with last 3 patients discharged on day three. No major intraoperative or post-operative complications were seen. The Eckardt score changed from above 4 to 0 at one-month follow-up. All four are well at one year post-operatively and beyond. Two patients had subcutaneous emphysema post-operatively. One developed pneumoperitoneum intra-operatively.
POEM was successfully performed with only minor adverse events in experienced hands. Anticipation and preparation for potential intraoperative complications and assigned responsibilities to each team helped the safe completion in the shortest time. Rectifying pneumoperitoneum concurrently without interruption of the operation exemplified teamwork.
III
Endoscopic management of spontaneous esophageal and postoperative motility disorders
2022, Journal of Visceral Surgery
Considerable progress has been made in therapeutic endoscopy over recent years and new techniques now offer access to a new space, the submucosal space, thus paving the way for new therapeutic perspectives, in particular, for functional endoscopy. The first of these applications, POEM (Per-oral endoscopic myotomy), has become, since the beginning of the 2010's, an endoscopic alternative to Heller myotomy for the treatment of achalasia. The results of this technique have been fully documented, and the technique has replaced pneumatic dilatation and become the standard treatment for this indication with equivalent effectiveness compared to traditional surgery, but with less morbidity. Indeed, the overall efficacy is around 90%, results are stable over time, and severe morbidity is less than 1%. Later, other motility disorders, such as spastic disorders, have been treated, certainly with less impressive outcomes than for achalasia, but still, of interest. This approach has also been applied to post-fundoplication motility disorders, whose de novo prevalence can be as high as 20%, and for which management is complex; this represents another new non-surgical perspective. The POEM procedure for this indication, in addition to myotomy, allows performance of a partial incision of the wrap with substantial symptomatic relief. In conclusion, the technique seems to have a bright future as it is progressively applied to more and more motility disorders.
Single-dose versus short-course prophylactic antibiotics for peroral endoscopic myotomy: a randomized controlled trial
2021, Gastrointestinal Endoscopy
Peroral endoscopic myotomy (POEM) has been recommended for achalasia treatment. To prevent the potential of infective risk, antibiotic prophylaxis is usually administered, whereas the additional need of antibiotic therapy after POEM is uncertain. The primary endpoint was to determine whether prophylaxis versus prophylaxis plus short therapy was needed after POEM.
Consecutive patients scheduled for POEM were randomly assigned (1:1) to group A (prophylactic cefazolin 2 g IV) or group B (prophylaxis + cefazolin 2 g IV × 3 followed by oral amoxicillin/clavulanate 3 g/day). Infective risk was assessed by means of host response, namely body temperature and serum levels of white blood cells and C-reactive protein; immune response (the cytokines interleukin [IL]-6, IL-1β, and tumor necrosis factor-α and microbial translocation mediators lipopolysaccharide binding protein and soluble CD14); and blood cultures at time points before (t0) and after (t1, t2) POEM.
After POEM, none of the 124 enrolled patients (54.6 ± 12.6 years old; 64 men) developed any fever (body temperature: t0, 36.56± .49°C; t1, 36.53± .52°C; t2, 36.48± .41°C), without any differences between groups at any time point. Regarding systemic inflammation, no difference was reported between groups in serum levels of C-reactive protein and white blood cells. Considering microbial translocation mediated response, lipopolysaccharide binding protein (group A: t0, 1539 ± 168.6 pg/mL; t1, 1321 ± 149.1 pg/mL; t2, 2492 ± 283.2 pg/mL; group B: t0, 1318 ± 115.9 pg/mL; t1, 1492 ± 163.8 pg/mL; t2, 2600 ± 328.2 pg/mL) and soluble CD14 (group A: t0, 2.16 ± .15 μg/mL; t1, 1.89 ± .15 μg/mL; t2, 2.2 ± .15 μg/mL; group B: t0, 2.1 ± .13 μg/mL; t1, 2 ± .13 μg/mL; t2, 2.5 ± .2 μg/mL) were similar between the 2 groups; the immune response cytokines IL-6, IL-1β, and tumor necrosis factor-α also were similar in the 2 groups. In relation to blood cultures, at t1 the group B bacteremia rate was 3.2% (2/62) and group A was 1.6% (1/62) with no difference (P = .6). All subsequent blood cultures were negative at t2.
According to our study, postprophylactic short-term antimicrobial therapy after POEM is not required because of a very low residual infective risk. (Clinical trial registration number: NCT03587337.)
Landscape of Adverse Events Related to Peroral Endoscopic Myotomy in 3135 Patients and a Risk-Scoring System to Predict Major Adverse Events
2021, Clinical Gastroenterology and Hepatology
Citation Excerpt :
All patients were intubated. The standard POEM procedure was performed as previously reported,12 including mucosal incision, submucosal tunneling, myotomy, and mucosal closure. Intraprocedural mucosal injury (including type I and type II mucosal injury) and mucosal edema were defined as previously described.13
This article systemically describes the landscape of peroral endoscopic myotomy (POEM)-related adverse events (AEs) and compares the different grading systems; and establishes and validates a combined risk factor model and a simplified risk-scoring system to predict POEM-related major AEs.
A total of 3135 patients with achalasia treated with POEM were included and the AEs were systemically described and graded. A predictive model and risk-scoring system was developed using logistic regression and then internally validated using bootstrapping approaches.
A total of 258 out of 3135 patients, accounting for 8.23% of the total patients, presented with 297 AEs. According to Clavien-Dindo grading, 175 (67.83%), 23 (8.91%), 56 (21.71%), 4 (1.55%), and 0 (0.00%) patients were graded as grade I–V, respectively. By American Society of Gastrointestinal Endoscopy lexicon, 175 (67.83%) patients were classified with mild AE, 66 (25.58%) were classified with moderate AE, and 17 (6.59%) were classified with severe AE, respectively. Sixty-eight (2.17%) patients were classified with major AE. Air insufflation, selective myotomy, mucosal injury, and long operation time were selected into the predictive model with an area under the curve of 0.795. They were assigned with scores of 18, 5, 3, and 5 in the risk-scoring system, respectively. By applying the risk scoring system, patients with higher scores had higher rates of major AEs. The model showed little evidence for overfitting and was well-calibrated.
Based on a systematic landscape analysis, POEM is a safe procedure with low rates of severe AEs. Our prediction model and risk-scoring system demonstrated good performance in predicting major AEs.
Per oral endoscopic myotomy (POEM) in pediatric patients with esophageal achalasia: First Latin-American experience
2021, Journal of Pediatric Surgery
Achalasia is the most common primary motor disorder of the esophagus, but its incidence in pediatric patients is low. Laparoscopic Heller myotomy (LHM) is the current surgical standard of care treatment. Per-oral endoscopic myotomy (POEM) has emerged as a safe and effective therapeutic alternative in adult patients. We herein report the outcomes of a cohort of pediatric patients with achalasia treated by POEM at a Chilean medical center.
This is a retrospective analysis of prospectively collected data on children who underwent POEM for esophageal achalasia. Clinical follow-up was evaluated by recording the Eckardt score, a high-resolution esophageal manometry (HREM) three months after the procedure, and an annual upper gastrointestinal endoscopy.
Five patients with esophageal achalasia confirmed by HREM and with a mean age of 11 (5 to 15) years underwent POEM between 2017 and 2019. One patient had a previous LHM. No morbidity or mortality was observed. All patients resolved their dysphagia and no patient required further interventions. Mean Eckardt score reduced from 10 points preoperatively to 1 point postoperatively.
Two patients currently have mild esophagitis (confirmed by endoscopy).
Our results support the previously reported safety and effectiveness of POEM. Longer follow-up and larger cohorts will be important to confirm its role in the treatment of children with esophageal achalasia.
Treatment study.
Level IV.

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: DISCLOSURE: This study was supported by grants from the Major Project of Shanghai Municipal Science and Technology Committee (11411950502 and 13411950801), Academic Leader Training Project of Shanghai Municipal Commission of Health and Family Planning (13B038), National Natural Science Foundation of China (81302098, 81370588 and 81201902), and Natural Science Foundation of Shanghai (13ZR1452300). All other authors disclosed no financial relationships relevant to this article.

: If you would like to chat with an author of this article, you may contact Dr Zhou at [email protected].

: See CME section; p. 214.

^∗: Drs Chen and Li contributed equally to this article.

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Original articleClinical endoscopyLong-term outcomes of peroral endoscopic myotomy for achalasia in pediatric patients: a prospective, single-center study

Background

Objective

Design

Setting

Patients

Interventions

Main Outcome Measurements

Results

Limitations

Conclusion

Section snippets

Patients

Patient characteristics

Discussion

J Pediatr Surg

J Am Coll Surg

Gastroenterology

Gastrointest Endosc

J Gastrointest Surg

Gastroenterology

Gastrointest Endosc

J Pediatr Surg

J Pediatr Surg

J Am Coll Surg

Treatment and surveillance strategies in achalasia: an update

Nat Rev Gastroenterol Hepatol

Epidemiological study of achalasia in children

Gut

A review of achalasia in 33 children

Dig Dis Sci

Peroral endoscopic myotomy (POEM) for esophageal achalasia

Endoscopy

Peroral endoscopic myotomy for the treatment of achalasia: an international prospective multicenter study

Gastroenterology

Peroral endoscopic remyotomy for failed Heller myotomy: a prospective single-center study

Endoscopy

Original article
Clinical endoscopy
Long-term outcomes of peroral endoscopic myotomy for achalasia in pediatric patients: a prospective, single-center study