Development of a preoperative risk-scoring system for predicting poor responders to peroral endoscopic myotomy

doi:10.1016/j.gie.2020.06.028

Gastrointestinal Endoscopy

Volume 93, Issue 2, February 2021, Pages 398-405

https://doi.org/10.1016/j.gie.2020.06.028 Get rights and content

Background and Aims

Peroral endoscopic myotomy (POEM) is an effective treatment for esophageal motility disorder. However, some people are poor responders who will probably need retreatments, such as endoscopic pneumatic dilation or re-POEM, and a scoring system for the prediction of poor responders preoperatively has not yet been established. We aimed to develop and validate a preoperative scoring system for predicting poor responders.

Methods

Overall, 244 patients who underwent POEM for esophageal motility disorders in our hospital from April 2015 to March 2019 were retrospectively included in this study. Poor responders were defined as patients with any of following: (1) Eckardt score ≥3 at 1-year follow-up, (2) endoscopic findings of food retention at 1-year follow-up, and (3) retreatments within 1 year after POEM. A risk-scoring system for poor responders was developed based on multiple logistic regression analysis, and its performance was internally validated using bootstrapping.

Results

Forty patients were diagnosed as poor responders at the 1-year follow-up. In the multivariate study, points for risk scores were assigned for 4 independent risk factors as follows: pretreatment Eckardt score (1-point increments), previous treatments (4 points), sigmoid-type esophagus (4 points), and esophageal dilation grade ≥II (4 points). The scoring system could predict an estimated risk for poor responders and provided satisfactory discrimination (area under the receiver operating characteristic curve, 0.78; 95% confidence interval, 0.68-0.88) and calibration (slope = 0.93; 95% confidence interval, 0.62-1.31).

Conclusions

A validated risk-scoring system for predicting poor responders preoperatively was established; this system could be useful for selecting treatment strategies and postoperative surveillance.

Graphical abstract

Introduction

Esophageal motility disorders (EMDs), such as achalasia and its variants, are a group of diseases characterized by progressive dysphagia, chest pain, regurgitation, and weight loss.¹ Peroral endoscopic myotomy (POEM) is a highly safe and clinically effective treatment for EMD with short-term follow-up²; however, several prospective multicenter studies have shown that treatment of approximately 10% of patients who undergo POEM eventually fails 1 to 2 years after POEM.³^,⁴ Furthermore, some patients with significant recurrent or persistent symptoms after POEM need retreatments within 3 years.⁵

Some studies have suggested that risk factors for POEM failure include disease duration, previous treatments, and preoperative Eckardt score, among others^;6, 7, 8 however, they were not validated and predicted a degree of risk. Liu et al⁹ reported a risk-scoring system for predicting POEM failure based on previous treatment, intraprocedural mucosal injury, and clinical reflex. However, it was not useful in preoperative prediction because the system included postoperative factors.

This study aimed to develop and validate a preoperative risk-scoring system consisting of only preoperative factors for predicting poor responders who are likely to need retreatments. This risk-scoring system could assist in the selection of a treatment plan based on estimated risk for retreatments and to determine postoperative surveillance strategies leading to a reduction in unnecessary endoscopic procedures and follow-up visits in high-volume (ie, experienced) centers.

Section snippets

Patients

The clinical data of consecutive patients who underwent POEM for achalasia and its variants in our hospital from April 2015 to March 2019 were collected retrospectively from prospectively maintained institutional database. Achalasia variants, such as diffuse esophageal spasm, jackhammer esophagus, and esophagogastric junction (EGJ) outflow obstruction, were included. However, patients who had already undergone POEM and were not subjected to esophagogastroduodenoscopy (EGD) at 1-year follow-up

Baseline characteristics of the patients

In total, 344 consecutive patients with achalasia and its variants who underwent POEM were enrolled. We excluded 6 patients who had already undergone POEM, and 94 patients who did not undergo EGD and HRM at the 1-year follow-up or were lost to follow-up. Finally, 244 patients (111 men [45.5%], median age, 56.3 years [IQR, 39.2-73.4; range, 5-88]) were included in this study (Fig. 1).

There were 225 patients (92.2%) with achalasia, including 141 with type I, 50 with type II, and 15 with type III.

Discussion

POEM is highly effective in the treatment of EMD with a short-term follow-up,² but treatment of approximately 10% of patients eventually fails 1 to 2 years after POEM,⁴ and some patients with significant recurrent or persistent symptoms after POEM require retreatments within 3 years.⁵ Few studies have reported a preoperative risk-scoring system for predicting POEM failure. In this study, poor responders were defined as those who were likely to need retreatments after POEM, and a validated

References (19)

H. Inoue et al.
Per-oral endoscopic myotomy: a series of 500 patients
J Am Coll Surg
(2015)
Q.L. Li et al.
Outcomes of per-oral endoscopic myotomy for treatment of esophageal achalasia with a median follow-up of 49 months
Gastrointest Endosc
(2018)
X.Y. Liu et al.
A risk-scoring system to predict clinical failure for patients with achalasia after peroral endoscopic myotomy
Gastrointest Endosc
(2020)
M.P. Sweet et al.
The outcome of laparoscopic Heller myotomy for achalasia is not influenced by the degree of esophageal dilation
J Gastrointest Surg
(2008)
S.J. Spechler et al.
Classification of oesophageal motility abnormalities
Gut
(2001)
S. Ngamruengpong et al.
Long-term outcomes of per-oral endoscopic myotomy in patient with achalasia with a minimum follow-up of 49 months
Gastrointest Endosc
(2018)
D. Von Renteln et al.
Peroral endoscopic myotomy for the treatment of achalasia: an international prospective multicenter study
Gastroenterology
(2013)
F.B. Hoeji et al.
Management of recurrent symptoms after per-oral endoscopic myotomy
Gastrointest Endosc
(2018)
H.O. Kristensen et al.
Long-term outcome of peroral endoscopic myotomy for esophageal achalasia in patients with previous Heller myotomy
Surg Endosc
(2017)

There are more references available in the full text version of this article.

Cited by (18)

Propensity score matching is needed to evaluate the clinical success rate of peroral endoscopic myotomy
2024, Gastrointestinal Endoscopy
Factors associated with early failure of peroral endoscopic myotomy in achalasia
2024, Gastrointestinal Endoscopy
Achalasia can be treated very effectively with peroral endoscopic myotomy (POEM), but factors associated with early failure remain to be determined, especially in European cohorts.
All consecutive adult patients who underwent a first POEM to treat primary achalasia were included in this multicenter retrospective study. Early failure was defined by an Eckardt score (ES) >3 at 3 months after POEM. When evaluating factors predictive of early failure, 2 cohorts were considered: one consisted of the total population, for whom only basic variables were collected, and the other a cohort built for a case-control study that included matched early-failure and early-success patients (ratio, 1:2).
Among 746 patients, the early failure rate was 9.4%. Predictive factors were age ≤45 years (P = .019), achalasia types I and III (P < .001), and the development of a severe adverse event during the procedure (P = .023). In the case-control study, the only additional independent risk factor for early failure was a high pre-POEM ES (P = .001). Only the retrosternal pain subscore was significantly associated with the early failure rate.
The early failure rate of POEM used to treat primary achalasia is <10%. Younger age, type I/III achalasia, and a high pre-POEM ES were significantly associated with failure.
POEM, long-term outcomes, and gastroesophageal reflux: All is well!
2021, Gastrointestinal Endoscopy
GIE Editorial Board top 10 topics: advances in GI endoscopy in 2020
2021, Gastrointestinal Endoscopy
Citation Excerpt :
Liu et al31 proposed a scoring system based on 4 (previous treatment, intraprocedural type 1 mucosal injury, type II mucosal injury, and postprocedure clinical reflux) of 17 proposed risk factors that can be used to predict likelihood of POEM failure. Urakami et al32 also performed a retrospective study involving 244 patients and found pretreatment Eckardt score, sigmoid esophagus, history of previous treatment, and esophageal dilation grade to be predictive of poor outcomes. The Liu study also evaluated the factors identified in the Urakami study but did not find these to be significantly associated with POEM efficacy.31
The American Society for Gastrointestinal Endoscopy’s Gastrointestinal Endoscopy Editorial Board reviewed a systematic literature search of original endoscopy-related articles published during 2020 in Gastrointestinal Endoscopy and 10 other high-impact medical and gastroenterology journals. Votes from each individual board member were tallied to identify a consensus list of the 10 most significant topic areas in GI endoscopy over the calendar year of study using 4 criteria: significance, novelty, impact on national health, and impact on global health. The 10 areas identified were as follows: artificial intelligence in endoscopy, coronavirus disease 2019 and GI practice, third-space endoscopy, lumen-apposing metal stents, single-use duodenoscopes and other disposable equipment, endosonographic needle technology and techniques, endoscopic closure devices, advances in GI bleeding management, improvements in polypectomy techniques, and bariatric endoscopy. Each board member contributed a summary of important articles relevant to 1 to 2 topic areas, leading to a collective summary that is presented in this document of the “top 10” endoscopic advances of 2020.
Preoperative predictors of poor response to peroral endoscopic myotomy: Have we found the crystal ball?
2021, Gastrointestinal Endoscopy
Long-term efficacy of peroral endoscopic myotomy for achalasia under different criteria
2024, Surgical Endoscopy

View all citing articles on Scopus

: DISCLOSURE: All authors disclosed no financial relationships.

: See CME section; p. 495.

View full text

Original articleClinical endoscopyDevelopment of a preoperative risk-scoring system for predicting poor responders to peroral endoscopic myotomy

Background and Aims

Methods

Results

Conclusions

Graphical abstract

Introduction

Section snippets

Patients

Baseline characteristics of the patients

Discussion

J Am Coll Surg

Gastrointest Endosc

Gastrointest Endosc

J Gastrointest Surg

Classification of oesophageal motility abnormalities

Gut

Long-term outcomes of per-oral endoscopic myotomy in patient with achalasia with a minimum follow-up of 49 months

Gastrointest Endosc

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

Gastroenterology

Management of recurrent symptoms after per-oral endoscopic myotomy

Gastrointest Endosc

Long-term outcome of peroral endoscopic myotomy for esophageal achalasia in patients with previous Heller myotomy

Surg Endosc

Original article
Clinical endoscopy
Development of a preoperative risk-scoring system for predicting poor responders to peroral endoscopic myotomy