A procedure called POEM significantly improved treatment outcomes among patients with achalasia — a rare swallowing disorder — compared with a standard therapy, according to a study published in JAMA.
“This randomized clinical trial showed POEM superior to pneumatic dilation and will greatly enhance its acceptance as a go-to therapy for achalasia patients,” said co-author Peter Kahrilas, MD, ’84 GME, the Gilbert H. Marquardt Professor of Medicine in the Division of Gastroenterology and Hepatology.
John Pandolfino, MD, ’97 GME, ’01 GME, ’05 MSCI, the Hans Popper Professor and chief of Gastroenterology and Hepatology, was also a co-author of the study that investigated the effectiveness of peroral endoscopic myotomy, or POEM.
Achalasia is a rare condition in which damage to the nerves of the esophagus leads to difficulty swallowing, and other symptoms such as chest pain, heartburn and weight loss.
There is no cure for the underlying disease, but treatment for achalasia can help alleviate symptoms by relaxing the lower esophageal sphincter (LES) muscle — either with drugs or mechanical treatments — to improve the passage of food and liquid.
The most commonly performed treatment is pneumatic dilation, in which an endoscopically guided balloon is placed over the LES and inflated with air. A surgical option called laparoscopic Heller myotomy, in which tiny cuts into the muscle weaken the LES, is a more permanent solution, but the procedure is invasive and carries the risk of severe complications.
In 2009, the POEM procedure was introduced as a new treatment option. The procedure allows surgical myotomy to be performed endoscopically through the mouth, as opposed to through the abdominal wall in a laparoscopic technique. “Consequently, patients recover much more quickly, have a potentially permanent solution to their achalasia, and have no skin wounds to heal or scar,” Kahrilas said.
Up until now, there has been a lack of large clinical trials comparing POEM with current treatment options; the current study is the first randomized clinical trial evaluating the use of POEM as an initial treatment for achalasia.
An international team of investigators randomized 133 patients with achalasia to receive either POEM or pneumatic dilation, the standard treatment. All participants had not received any prior treatment.
The primary outcome was the treatment success rate, defined as a reduction in the patient’s Eckardt score (a grading system for symptoms of achalasia) to less than or equal to three, and the absence of severe complications or need for re-treatment.
At the two-year follow-up, the treatment success rate was significantly higher in the POEM group, at 92 percent, compared to 54 percent in the pneumatic dilation group. There were no serious adverse events with POEM, although there were two in the pneumatic dilation group.
The development of reflux esophagitis, however, was found to be more frequent in the group that received POEM, as was the use of proton pump inhibitors, medications used to relieve symptoms of acid reflux.
The study’s findings support the consideration of POEM as an initial treatment option for patients with achalasia, the authors wrote.
“Going forward, we are working at Northwestern to tailor the POEM technique to the unique needs of individual patients, as defined by novel diagnostics that we have also developed at Northwestern,” Kahrilas said.
The study, led by investigators at the University of Amsterdam in the Netherlands, was supported by Fonds NutsOhra (FNO grant 1202-022) and an ESGE Research Grant.