Peroral endoscopic myotomy (POEM) had a success rate exceeding 90%, versus just about 50% for standard balloon dilation in what investigators say is, to their knowledge, the first-ever randomized trial to evaluate POEM as a first-line modality for this esophageal motility disorder.
Reflux esophagitis was the major downside of POEM, according to investigators, who reported the complication in 41% of patients at a 2-year follow-up, as compared to just 7% of patients undergoing the standard balloon dilation.
Nevertheless, there were no serious adverse events among 63 POEM-treated patients, while one patient out of 63 undergoing pneumatic dilation had a perforation that required endoscopic closure and hospitalization, according to senior study author Albert J. Bredenoord, MD, PhD, of Amsterdam University Medical Center.
“These findings support consideration of POEM as an initial treatment option for patients with achalasia,” Dr. Bredenoord and coinvestigators said in a report on the study appearing in.
While endoscopic pneumatic dilation is the usual treatment for achalasia, POEM has become more commonly used following case series showing high rates of efficacy, according to the authors.
The POEM procedure also offers advantages over laparoscopic Heller myotomy, which is invasive and associated with severe complications, including a transmural perforation rate of 4%-10%, they said in their report.
Their randomized trial included 133 adults with newly diagnosed achalasia enrolled at one of six hospitals in Germany, Hong Kong, Italy, Netherlands, and the United States.
Patients were randomly assigned to undergo 1-2 pneumatic dilations performed by an endoscopist who had performed at least 20 such procedures in the past, or to a POEM procedure likewise performed by an expert who had already done more than 20 such procedures.
At baseline, patients’ Eckardt symptom scores ranged from 6 to 9 on a scale with 0 indicating the lowest severity, to 12 indicating the highest. The median Eckardt scores were 8 in the POEM group and 7 in the pneumatic dilation group.
Treatment success, defined as an Eckardt score under 3 and no severe complications or retreatment at 2 years, was achieved by 58 of 63 patients (92%) in the POEM group, compared with 34 of 63 patients (54%) in the pneumatic dilation group (P less than .001), investigators reported.
Reflux esophagitis was observed in 22 of 54 POEM-treated patients (41%) who underwent endoscopy at a 2-year evaluation, compared with only 2 of 29 patients (7%) who had received the balloon dilation procedure (P = .002). In line with that finding, both reflux symptoms and daily proton pump inhibitor use were more common in the POEM group, investigators said.
However, there were no differences between POEM and pneumatic dilation groups in quality of life and other secondary endpoints, including median barium column height and median integrated relaxation pressure, they reported.
Two serious adverse events related to treatment were seen, according to investigators, including one perforation requiring an endoscopic closure plus antibiotics and hospitalization for 13 days, and one hospital admission for a night because of severe chest pain with no signs of perforation.
“Although POEM is more invasive and requires more technical endoscopic skills, the risk of severe complications was not higher than with pneumatic dilation, especially when performed by experienced endoscopists,” Dr. Bredenoord and coauthors said in their report.
However, these results do not imply that the traditional dilation procedure should be abandoned, they said, as POEM is more invasive, more involved, and more likely to result in reflux esophagitis.
“It seems reasonable to offer both options to treatment-naive patients with achalasia and counsel them to select treatment based on the patient’s characteristics, personal preference, comorbidity, and disease subtype,” they said.
Funding for the study came from Fonds NutsOhra and the European Society of Gastrointestinal Endoscopy. Dr. Bredenoord reported disclosures related to Norgine, Laborie, Medtronic, Diversatek, Nutricia, Regeneron, Celgene, Bayer, and Dr. Falk Pharma.
SOURCE: Ponds FA et al. JAMA. 2019;322(2):134-44. .