Multicenter Evaluation of Clinical Efficacy and Safety of Per‐oral Endoscopic Myotomy in Children

Abstract

Objectives: Per-oral endoscopic myotomy (POEM) is a recommended treatment modality for achalasia, but there is little published data for its use in children. The objective of the present study was to evaluate whether POEM is clinically effective and safe for children. Methods: International multicenter retrospective study conducted in 14 tertiary centers that included consecutive children who underwent POEM between January 2012 and August 2018. Outcomes, such as clinical response were assessed whenever available. Adverse events and factors associated with clinical failure were also investigated. Results: A total of 117 patients (mean ± SD age: 14.2 ± 3.7 years) underwent POEM for achalasia (type I, n = 36; type II n=66; type III, n=8). Among these, 30 (26%) were pretreated (botulinum injection and/or pneumatic dilatation). Mean ± SD baseline Eckardt score was 7.5 ± 2.0. Clinical success was achieved in 90.6% of cases (95%CI [83.8%;95.2%]) in the intention-to-treat analysis. The mean ± SD Eckardt score post-POEM was 0.9 ± 1.2 (P < 0.001). The mean duration of follow-up time 545 days (range: 100–1612). A total of 7 adverse events occurred (4 mucosotomies, 2 subcutaneous emphysema, 1 esopleural fistula). Gastroesophageal reflux symptoms were seen in 17 patients (15%); missing data for 10 patients (9%). There was a trend towards more frequent clinical failure in achalasia associated with genetic disorders (40% vs 8%, P = 0.069). Conclusions: POEM in pediatric patients appears to be effective and safe, although there was a trend towards more frequent clinical failure achalasia associated with genetic disorders. Further studies are needed to assess the long-term outcomes, especially the consequences of GERD.

Long-term clinical results of per-oral endoscopic myotomy (POEM) for achalasia: First report of more than 10-year patient experience as assessed with a questionnaire-based survey

“…Since the introduction of POEM into clinical practice based on the evidence of an experimental report on the safety of endoscopic myotomy in a porcine model by Pasricha et al [16], indications initially were restricted for only typical achalasia; hence, POEM has been safely completed in all cases regardless of prior treatment. Based on primary reports regarding efficiency and safety associated with short-term results of POEM, indications for the procedure have been expanded [15,[17][18][19], and to date, modifications and discussions are still being done among endoscopists all over the world to achieve safer and better results [20,21].…”

Current management of pediatric achalasia

“…After demonstrating its minimally invasive benefit for adult patients, the POEM procedure has become an important treatment for achalasia in children (30). Overall, the clinical success and safety profile appears to be similar to that described in adults (16,31). Major steps of the POEM are depicted in Figure 4.…”

Achalasia

“…Most recently, the successful development of natural orifice endoscopic surgery (NOTES) in human clinical use has also inspired surgeons to perform trans-esophageal endoscopic myotomy as a treatment for achalasia (Choné et al 2019;Nabi et al 2019;Lee et al 2019). Indeed, endoscopic myotomy has become the most widely Fig.…”