Effect of Peroral Endoscopic Myotomy vs Pneumatic Dilation on Symptom Severity and Treatment Outcomes among Treatment-Naive Patients with Achalasia: A Randomized Clinical Trial

Fraukje A. Ponds, Paul Fockens, Aaltje Lei, Horst Neuhaus, Torsten Beyna, Jennis Kandler, Thomas Frieling, Philip W.Y. Chiu, Justin C.Y. Wu, Vivien W.Y. Wong, Guido Costamagna, Pietro Familiari, Peter J Kahrilas, John Erik Pandolfino, André J.P.M. Smout, Albert J. Bredenoord*

*Corresponding author for this work

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

Importance: Case series suggest favorable results of peroral endoscopic myotomy (POEM) for treatment of patients with achalasia. Data comparing POEM with pneumatic dilation, the standard treatment for patients with achalasia, are lacking. Objective: To compare the effects of POEM vs pneumatic dilation as initial treatment of treatment-naive patients with achalasia. Design, Setting, and Participants: This randomized multicenter clinical trial was conducted at 6 hospitals in the Netherlands, Germany, Italy, Hong Kong, and the United States. Adult patients with newly diagnosed achalasia and an Eckardt score greater than 3 who had not undergone previous treatment were included. The study was conducted between September 2012 and July 2015, the duration of follow-up was 2 years after the initial treatment, and the final date of follow-up was November 22, 2017. Interventions: Randomization to receive POEM (n = 67) or pneumatic dilation with a 30-mm and a 35-mm balloon (n = 66), with stratification according to hospital. Main Outcomes and Measures: The primary outcome was treatment success (defined as an Eckardt score ≤3 and the absence of severe complications or re-treatment) at the 2-year follow-up. A total of 14 secondary end points were examined among patients without treatment failure, including integrated relaxation pressure of the lower esophageal sphincter via high-resolution manometry, barium column height on timed barium esophagogram, and presence of reflux esophagitis. Results: Of the 133 randomized patients, 130 (mean age, 48.6 years; 73 [56%] men) underwent treatment (64 in the POEM group and 66 in the pneumatic dilation group) and 126 (95%) completed the study. The primary outcome of treatment success occurred in 58 of 63 patients (92%) in the POEM group vs 34 of 63 (54%) in the pneumatic dilation group, a difference of 38% ([95% CI, 22%-52%]; P <.001). Of the 14 prespecified secondary end points, no significant difference between groups was demonstrated in 10 end points. There was no significant between-group difference in median integrated relaxation pressure (9.9 mm Hg in the POEM group vs 12.6 mm Hg in the pneumatic dilation group; difference, 2.7 mm Hg [95% CI, -2.1 to 7.5]; P =.07) or median barium column height (2.3 cm in the POEM group vs 0 cm in the pneumatic dilation group; difference, 2.3 cm [95% CI, 1.0-3.6]; P =.05). Reflux esophagitis occurred more often in the POEM group than in the pneumatic dilation group (22 of 54 [41%] vs 2 of 29 [7%]; difference, 34% [95% CI, 12%-49%]; P =.002). Two serious adverse events, including 1 perforation, occurred after pneumatic dilation, while no serious adverse events occurred after POEM. Conclusions and Relevance: Among treatment-naive patients with achalasia, treatment with POEM compared with pneumatic dilation resulted in a significantly higher treatment success rate at 2 years. These findings support consideration of POEM as an initial treatment option for patients with achalasia. Trial Registration: Netherlands Trial Register number: NTR3593.

Original languageEnglish (US)
Pages (from-to)134-144
Number of pages11
JournalJAMA - Journal of the American Medical Association
Volume322
Issue number2
DOIs
StatePublished - Jul 9 2019

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Esophageal Achalasia
Dilatation
Randomized Controlled Trials
Barium
Therapeutics
Peptic Esophagitis
Netherlands
Pressure
Lower Esophageal Sphincter
Manometry
Hong Kong
Random Allocation
Treatment Failure
Italy
Multicenter Studies
Germany

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Ponds, Fraukje A. ; Fockens, Paul ; Lei, Aaltje ; Neuhaus, Horst ; Beyna, Torsten ; Kandler, Jennis ; Frieling, Thomas ; Chiu, Philip W.Y. ; Wu, Justin C.Y. ; Wong, Vivien W.Y. ; Costamagna, Guido ; Familiari, Pietro ; Kahrilas, Peter J ; Pandolfino, John Erik ; Smout, André J.P.M. ; Bredenoord, Albert J. / Effect of Peroral Endoscopic Myotomy vs Pneumatic Dilation on Symptom Severity and Treatment Outcomes among Treatment-Naive Patients with Achalasia : A Randomized Clinical Trial. In: JAMA - Journal of the American Medical Association. 2019 ; Vol. 322, No. 2. pp. 134-144.
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title = "Effect of Peroral Endoscopic Myotomy vs Pneumatic Dilation on Symptom Severity and Treatment Outcomes among Treatment-Naive Patients with Achalasia: A Randomized Clinical Trial",
abstract = "Importance: Case series suggest favorable results of peroral endoscopic myotomy (POEM) for treatment of patients with achalasia. Data comparing POEM with pneumatic dilation, the standard treatment for patients with achalasia, are lacking. Objective: To compare the effects of POEM vs pneumatic dilation as initial treatment of treatment-naive patients with achalasia. Design, Setting, and Participants: This randomized multicenter clinical trial was conducted at 6 hospitals in the Netherlands, Germany, Italy, Hong Kong, and the United States. Adult patients with newly diagnosed achalasia and an Eckardt score greater than 3 who had not undergone previous treatment were included. The study was conducted between September 2012 and July 2015, the duration of follow-up was 2 years after the initial treatment, and the final date of follow-up was November 22, 2017. Interventions: Randomization to receive POEM (n = 67) or pneumatic dilation with a 30-mm and a 35-mm balloon (n = 66), with stratification according to hospital. Main Outcomes and Measures: The primary outcome was treatment success (defined as an Eckardt score ≤3 and the absence of severe complications or re-treatment) at the 2-year follow-up. A total of 14 secondary end points were examined among patients without treatment failure, including integrated relaxation pressure of the lower esophageal sphincter via high-resolution manometry, barium column height on timed barium esophagogram, and presence of reflux esophagitis. Results: Of the 133 randomized patients, 130 (mean age, 48.6 years; 73 [56{\%}] men) underwent treatment (64 in the POEM group and 66 in the pneumatic dilation group) and 126 (95{\%}) completed the study. The primary outcome of treatment success occurred in 58 of 63 patients (92{\%}) in the POEM group vs 34 of 63 (54{\%}) in the pneumatic dilation group, a difference of 38{\%} ([95{\%} CI, 22{\%}-52{\%}]; P <.001). Of the 14 prespecified secondary end points, no significant difference between groups was demonstrated in 10 end points. There was no significant between-group difference in median integrated relaxation pressure (9.9 mm Hg in the POEM group vs 12.6 mm Hg in the pneumatic dilation group; difference, 2.7 mm Hg [95{\%} CI, -2.1 to 7.5]; P =.07) or median barium column height (2.3 cm in the POEM group vs 0 cm in the pneumatic dilation group; difference, 2.3 cm [95{\%} CI, 1.0-3.6]; P =.05). Reflux esophagitis occurred more often in the POEM group than in the pneumatic dilation group (22 of 54 [41{\%}] vs 2 of 29 [7{\%}]; difference, 34{\%} [95{\%} CI, 12{\%}-49{\%}]; P =.002). Two serious adverse events, including 1 perforation, occurred after pneumatic dilation, while no serious adverse events occurred after POEM. Conclusions and Relevance: Among treatment-naive patients with achalasia, treatment with POEM compared with pneumatic dilation resulted in a significantly higher treatment success rate at 2 years. These findings support consideration of POEM as an initial treatment option for patients with achalasia. Trial Registration: Netherlands Trial Register number: NTR3593.",
author = "Ponds, {Fraukje A.} and Paul Fockens and Aaltje Lei and Horst Neuhaus and Torsten Beyna and Jennis Kandler and Thomas Frieling and Chiu, {Philip W.Y.} and Wu, {Justin C.Y.} and Wong, {Vivien W.Y.} and Guido Costamagna and Pietro Familiari and Kahrilas, {Peter J} and Pandolfino, {John Erik} and Smout, {Andr{\'e} J.P.M.} and Bredenoord, {Albert J.}",
year = "2019",
month = "7",
day = "9",
doi = "10.1001/jama.2019.8859",
language = "English (US)",
volume = "322",
pages = "134--144",
journal = "JAMA - Journal of the American Medical Association",
issn = "0098-7484",
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}

Ponds, FA, Fockens, P, Lei, A, Neuhaus, H, Beyna, T, Kandler, J, Frieling, T, Chiu, PWY, Wu, JCY, Wong, VWY, Costamagna, G, Familiari, P, Kahrilas, PJ, Pandolfino, JE, Smout, AJPM & Bredenoord, AJ 2019, 'Effect of Peroral Endoscopic Myotomy vs Pneumatic Dilation on Symptom Severity and Treatment Outcomes among Treatment-Naive Patients with Achalasia: A Randomized Clinical Trial', JAMA - Journal of the American Medical Association, vol. 322, no. 2, pp. 134-144. https://doi.org/10.1001/jama.2019.8859

Effect of Peroral Endoscopic Myotomy vs Pneumatic Dilation on Symptom Severity and Treatment Outcomes among Treatment-Naive Patients with Achalasia : A Randomized Clinical Trial. / Ponds, Fraukje A.; Fockens, Paul; Lei, Aaltje; Neuhaus, Horst; Beyna, Torsten; Kandler, Jennis; Frieling, Thomas; Chiu, Philip W.Y.; Wu, Justin C.Y.; Wong, Vivien W.Y.; Costamagna, Guido; Familiari, Pietro; Kahrilas, Peter J; Pandolfino, John Erik; Smout, André J.P.M.; Bredenoord, Albert J.

In: JAMA - Journal of the American Medical Association, Vol. 322, No. 2, 09.07.2019, p. 134-144.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Effect of Peroral Endoscopic Myotomy vs Pneumatic Dilation on Symptom Severity and Treatment Outcomes among Treatment-Naive Patients with Achalasia

T2 - A Randomized Clinical Trial

AU - Ponds, Fraukje A.

AU - Fockens, Paul

AU - Lei, Aaltje

AU - Neuhaus, Horst

AU - Beyna, Torsten

AU - Kandler, Jennis

AU - Frieling, Thomas

AU - Chiu, Philip W.Y.

AU - Wu, Justin C.Y.

AU - Wong, Vivien W.Y.

AU - Costamagna, Guido

AU - Familiari, Pietro

AU - Kahrilas, Peter J

AU - Pandolfino, John Erik

AU - Smout, André J.P.M.

AU - Bredenoord, Albert J.

PY - 2019/7/9

Y1 - 2019/7/9

N2 - Importance: Case series suggest favorable results of peroral endoscopic myotomy (POEM) for treatment of patients with achalasia. Data comparing POEM with pneumatic dilation, the standard treatment for patients with achalasia, are lacking. Objective: To compare the effects of POEM vs pneumatic dilation as initial treatment of treatment-naive patients with achalasia. Design, Setting, and Participants: This randomized multicenter clinical trial was conducted at 6 hospitals in the Netherlands, Germany, Italy, Hong Kong, and the United States. Adult patients with newly diagnosed achalasia and an Eckardt score greater than 3 who had not undergone previous treatment were included. The study was conducted between September 2012 and July 2015, the duration of follow-up was 2 years after the initial treatment, and the final date of follow-up was November 22, 2017. Interventions: Randomization to receive POEM (n = 67) or pneumatic dilation with a 30-mm and a 35-mm balloon (n = 66), with stratification according to hospital. Main Outcomes and Measures: The primary outcome was treatment success (defined as an Eckardt score ≤3 and the absence of severe complications or re-treatment) at the 2-year follow-up. A total of 14 secondary end points were examined among patients without treatment failure, including integrated relaxation pressure of the lower esophageal sphincter via high-resolution manometry, barium column height on timed barium esophagogram, and presence of reflux esophagitis. Results: Of the 133 randomized patients, 130 (mean age, 48.6 years; 73 [56%] men) underwent treatment (64 in the POEM group and 66 in the pneumatic dilation group) and 126 (95%) completed the study. The primary outcome of treatment success occurred in 58 of 63 patients (92%) in the POEM group vs 34 of 63 (54%) in the pneumatic dilation group, a difference of 38% ([95% CI, 22%-52%]; P <.001). Of the 14 prespecified secondary end points, no significant difference between groups was demonstrated in 10 end points. There was no significant between-group difference in median integrated relaxation pressure (9.9 mm Hg in the POEM group vs 12.6 mm Hg in the pneumatic dilation group; difference, 2.7 mm Hg [95% CI, -2.1 to 7.5]; P =.07) or median barium column height (2.3 cm in the POEM group vs 0 cm in the pneumatic dilation group; difference, 2.3 cm [95% CI, 1.0-3.6]; P =.05). Reflux esophagitis occurred more often in the POEM group than in the pneumatic dilation group (22 of 54 [41%] vs 2 of 29 [7%]; difference, 34% [95% CI, 12%-49%]; P =.002). Two serious adverse events, including 1 perforation, occurred after pneumatic dilation, while no serious adverse events occurred after POEM. Conclusions and Relevance: Among treatment-naive patients with achalasia, treatment with POEM compared with pneumatic dilation resulted in a significantly higher treatment success rate at 2 years. These findings support consideration of POEM as an initial treatment option for patients with achalasia. Trial Registration: Netherlands Trial Register number: NTR3593.

AB - Importance: Case series suggest favorable results of peroral endoscopic myotomy (POEM) for treatment of patients with achalasia. Data comparing POEM with pneumatic dilation, the standard treatment for patients with achalasia, are lacking. Objective: To compare the effects of POEM vs pneumatic dilation as initial treatment of treatment-naive patients with achalasia. Design, Setting, and Participants: This randomized multicenter clinical trial was conducted at 6 hospitals in the Netherlands, Germany, Italy, Hong Kong, and the United States. Adult patients with newly diagnosed achalasia and an Eckardt score greater than 3 who had not undergone previous treatment were included. The study was conducted between September 2012 and July 2015, the duration of follow-up was 2 years after the initial treatment, and the final date of follow-up was November 22, 2017. Interventions: Randomization to receive POEM (n = 67) or pneumatic dilation with a 30-mm and a 35-mm balloon (n = 66), with stratification according to hospital. Main Outcomes and Measures: The primary outcome was treatment success (defined as an Eckardt score ≤3 and the absence of severe complications or re-treatment) at the 2-year follow-up. A total of 14 secondary end points were examined among patients without treatment failure, including integrated relaxation pressure of the lower esophageal sphincter via high-resolution manometry, barium column height on timed barium esophagogram, and presence of reflux esophagitis. Results: Of the 133 randomized patients, 130 (mean age, 48.6 years; 73 [56%] men) underwent treatment (64 in the POEM group and 66 in the pneumatic dilation group) and 126 (95%) completed the study. The primary outcome of treatment success occurred in 58 of 63 patients (92%) in the POEM group vs 34 of 63 (54%) in the pneumatic dilation group, a difference of 38% ([95% CI, 22%-52%]; P <.001). Of the 14 prespecified secondary end points, no significant difference between groups was demonstrated in 10 end points. There was no significant between-group difference in median integrated relaxation pressure (9.9 mm Hg in the POEM group vs 12.6 mm Hg in the pneumatic dilation group; difference, 2.7 mm Hg [95% CI, -2.1 to 7.5]; P =.07) or median barium column height (2.3 cm in the POEM group vs 0 cm in the pneumatic dilation group; difference, 2.3 cm [95% CI, 1.0-3.6]; P =.05). Reflux esophagitis occurred more often in the POEM group than in the pneumatic dilation group (22 of 54 [41%] vs 2 of 29 [7%]; difference, 34% [95% CI, 12%-49%]; P =.002). Two serious adverse events, including 1 perforation, occurred after pneumatic dilation, while no serious adverse events occurred after POEM. Conclusions and Relevance: Among treatment-naive patients with achalasia, treatment with POEM compared with pneumatic dilation resulted in a significantly higher treatment success rate at 2 years. These findings support consideration of POEM as an initial treatment option for patients with achalasia. Trial Registration: Netherlands Trial Register number: NTR3593.

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