Yuki B. Werner, M.D., Bengt Hakanson, M.D., Jan Martinek, M.D., Alessandro Repici, M.D., Burkhard H.A. von Rahden, M.D., Albert J. Bredenoord, M.D., Raf Bisschops, M.D., Helmut Messmann, M.D.,Marius C. Vollberg, M.Sc., Tania Noder, R.N., Jan F. Kersten, M.Sc.,Oliver Mann, M.D., Jakob Izbicki, M.D., Alexander Pazdro, M.D., Uberto Fumagalli, M.D., Riccardo Rosati, M.D., Christoph-Thomas Germer, M.D., Marlies P. Schijven, M.D., Alice Emmermann, M.D., Daniel von Renteln, M.D., Paul Fockens, M.D., Guy Boeckxstaens, M.D., and Thomas Rösch, M.D.
Pneumatic dilation and laparoscopic Heller’s myotomy (LHM) are established treatments for idiopathic achalasia. Peroral endoscopic myotomy (POEM) is a less invasive therapy with promising early study results.
In a multicenter, randomized trial, we compared POEM with LHM plus Dor’s fundoplica-tion in patients with symptomatic achalasia. The primary end point was clinical success, defined as an Eckardt symptom score of 3 or less (range, 0 to 12, with higher scores indi-cating more severe symptoms of achalasia) without the use of additional treatments, at the 2-year follow-up; a noninferiority margin of −12.5 percentage points was used in the pri-mary analysis. Secondary end points included adverse events, esophageal function, Gas-trointestinal Quality of Life Index score (range, 0 to 144, with higher scores indicating better function), and gastroesophageal reflux.
A total of 221 patients were randomly assigned to undergo either POEM (112 patients) or LHM plus Dor’s fundoplication (109 patients). Clinical success at the 2-year follow-up was observed in 83.0% of patients in the POEM group and 81.7% of patients in the LHM group (difference, 1.4 percentage points; 95% confidence interval [CI], −8.7 to 11.4; P = 0.007 for noninferiority). Serious adverse events occurred in 2.7% of patients in the POEM group and 7.3% of patients in the LHM group. Improvement in esophageal func-tion from baseline to 24 months, as assessed by measurement of the integrated relax-ation pressure of the lower esophageal sphincter, did not differ significantly between the treatment groups (difference, −0.75 mm Hg; 95% CI, −2.26 to 0.76), nor did improve-ment in the score on the Gastrointestinal Quality of Life Index (difference, 0.14 points; 95% CI, −4.01 to 4.28). At 3 months, 57% of patients in the POEM group and 20% of patients in the LHM group had reflux esophagitis, as assessed by endoscopy; at 24 months, the corresponding percentages were 44% and 29%.
In this randomized trial, POEM was noninferior to LHM plus Dor’s fundoplication in controlling symptoms of achalasia at 2 years. Gastroesophageal reflux was more common among patients who underwent POEM than among those who underwent LHM. (Funded by the European Clinical Research Infrastructure Network and others; ClinicalTrials.gov number, NCT01601678.)