Peroral Endoscopic Myotomy an Upfront Option for Achalasia

By Megan Brooks

July 10, 2019

NEW YORK (Reuters Health) - For the swallowing disorder achalasia, peroral endoscopic myotomy (POEM) provided better symptom control than pneumatic dilation but was associated with more reflux esophagitis in a multicenter randomized controlled trial.

"These findings support consideration of POEM as an initial treatment option for patients with achalasia," researchers write in a paper online today in JAMA.

Achalasia is a rare esophageal motor disorder that makes it difficult to swallow. It affects roughly 10 of every 100,000 adults. Case series have suggested favorable results with POEM but no study, until now, has compared POEM with pneumatic dilation, the standard treatment for patients with achalasia, they note.

Dr. Arjan Bredenoord from Amsterdam University Medical Center in the Netherlands and colleagues at six hospitals in the Netherlands, Germany, Italy, Hong Kong and the United States enrolled adults (mean age, 48.6; 56% men) with newly diagnosed achalasia and Eckardt score >3 who had not undergone previous treatment.

Sixty-seven were randomly allocated to POEM and 66 to pneumatic dilation with a 30-mm and a 35-mm balloon. Sixty-four and 66 patients, respectively, underwent assigned treatment.

At two years of follow-up, the treatment success rate - defined as durable symptom relief (Eckardt score 3 or lower) and no serious adverse event or need for re-treatment - was 92% in the POEM group and 54% in the pneumatic dilation group (P<0.001).

There were no serious procedure-related adverse events with POEM and a single perforation (rate, 1.5%) that was treated with endoscopic closure occurred following pneumatic dilation. Reflux esophagitis occurred significantly more often with POEM than in pneumatic-dilation group (41% vs. 7%; P=0002) and is the "major disadvantage" of POEM, the researchers write. POEM is also more time-consuming and significantly more invasive.

Commenting on the findings in email to Reuters Health, Dr. Bredenoord said, "The hypothesis was that POEM would be more effective than pneumatic dilation, so this was expected. The high rate of reflux esophagitis was not expected beforehand. Patients should realize there is a high chance they will need to take anti-reflux medication after POEM for years."

In their paper, the researchers say the higher medium-term efficacy of POEM found in the study does not mean that pneumatic dilation should be abandoned.

"In my view," Dr. Bredenoord told Reuters Health, "there are now three good treatment options" for patients with achalasia: pneumatic dilation, Heller myotomy and POEM.

"The decision on which treatment option for which patient should be based on patients' preference and centers' expertise; each treatment has specific advantages and disadvantages and the patient should decide how they balance that," he said. "Some patients may choose a less invasive treatment that gives a higher chance of symptom recurrence while others may go for an invasive surgical procedure that gives a very small chance of recurrence right away."

Dr. Bredenoord also noted that POEM is being performed "more often every year in Europe, USA and Asia but is still often not reimbursed by healthcare systems and/or health-insurance companies."

He added, "I sincerely hope that our study will change this and will make POEM accessible for all achalasia patients, so they can be informed about it and decide themselves if they think this treatment option fits their demand. In my view, our study definitely pulls the technique of POEM out of the experimental phase into the regular treatment environment."

In a JAMA editorial, Drs. Brian Jacobson and David Lichtenstein, gastroenterologists at Boston Medical Center, say "the degree to which this novel technique is embraced as a first-line therapy for patients with achalasia may depend not only on longer follow-up to assess the durability of response, but also, in part, on whether the reimbursement value of this procedure is acknowledged to be more similar to a surgical intervention than a minimally invasive endoscopic intervention."

The study did not have commercial funding.


JAMA 2019.