Establishment of a New Gold Standard for Achalasia Treatment
Achalasia is a rare and often misdiagnosed disease of the esophagus that can cause considerable difficulties in swallowing. This is due to a permanent cramping of the sphincter in the lower end of the esophagus so that food does not completely or only partially empties into the stomach. Conventional treatment methods include either repeated balloon dilatations performed during endoscopy under radiographic control (pneumatic dilatation) or minimally invasive (keyhole) surgery in which the sphincter is cut, known as laparoscopic Heller myotomy (LHM).
The peroral endoscopic myotomy (POEM) is a newly-developed treatment form in which the esophageal sphincter can be cut during endoscopy. What makes this technique extraordinary is that despite cutting the muscle barrier, hardly any bacteria from the esophagus enters into the abdominal cavity so that severe and life-threatening infection does not result.
To determine if the efficacy of the POEM treatment is not inferior to the conventional surgical therapy (LHM), a total of 221 patients diagnosed with achalasia in 8 centers in 6 European countries were evaluated and randomly assigned to one of the treatment forms (1:1) and monitored over 2 years. The accepted noninferiority margin of 12.5% was defined before the beginning of the study after consideration of various aspects. After 2 years, the success rates measured according to the Eckardt Symptom Score were almost identical (POEM 83.0% versus LHM 81.7%). The primary efficacy endpoint was reached (p=0.007) and additionally supported by the nearly identical improvements of esophageal function and quality of life in both groups. Acute complications occurred in 3 out of 112 (2.7%) patients in the POEM group and 8 out of 109 (7.3%) patients in the LHM group, without fatalities. Another observation was that the reflux rates at 3-month follow-up examinations were 57.0% after POEM and 20% after LHM. At the 2-year follow-up, reflux rates were 44% after POEM and 29% after LHM.
The introduction of the endoscopic technique POEM has brought about a world-wide boost to innovation in the areas of gastroenterology-endoscopy and visceral surgery. Until now, there has been a lack of randomized controlled studies with the established treatment methods that are of tremendous and practical importance. The published study in the December 5, 2019, issue of the New England Journal of Medicine demonstrates that the findings as a result of of POEM are comparable with the findings after surgery (LHM) in the treatment of achalasia. However, a higher development of reflux can be observed after POEM. The long-term consequences of reflux development resulting after both procedures can be estimated by means of the prospective, 5-year follow-up data in this study.
Aside from sufficient study planning and essential interdisciplinary collaborations with gastroenterologists and surgeons, patient recruitment for the study (treatment assigned randomly) and incidence of disease (orphan disease) also posed a challenge because of the different approaches (endoscopy versus surgery). Planning and implementation of this international-European work under the leadership of Prof. Dr. Thomas Rösch and Dr. Yuki Werner were made possible by the generous support of various foundations, especially those in Hamburg.
Contact for scientific information:
Prof. Dr. Thomas Rösch
Department of Interdisciplinary Endoscopy
Universitätsklinikum Hamburg-Eppendorf (UKE)
20246 Hamburg, Germany
phone: 0049-40- 7410-50098
Werner Y, Rösch Th, et al. Endoscopic or Surgical Myotomy in Patients with Idiopathic Achalasia, NEngl J Med 2019; 381:2219-29. DOI: 10.1056/NEJMoa1905380