麻豆传媒

Peroral endoscopic myotomy (POEM) appears to be a viable alternative to pneumatic dilation for first-line treatment of esophageal achalasia, according to an international randomized study.

Albert J. Bredenoord, MD, PhD, of Amsterdam University Medical Center in the Netherlands, and associates reported a significantly higher 2-year treatment success rate in patients given POEM compared with dilation, at 92% versus 54%, respectively (38% difference, 95% CI 22-52%, P<0.001).

As noted in the team's study online in , success among the 133 randomized patients was defined as an Eckardt score of 3 or less and the absence of severe complications or re-treatment. No significant inter-group difference emerged for 10 of 14 secondary endpoints.

"These findings support consideration of POEM as an initial treatment option for patients with achalasia," the researchers wrote.

Study Details

During the period of September 2012 to July 2015, the trial enrolled 130 previously untreated patients from six centers in the Netherlands, Germany, Italy, Hong Kong, and the U.S. Patients had a mean age of 48.6 years, 56% were men, and participants were followed for 2 years after initial treatment.

After randomization, 64 patients received POEM and 66 had dilation, with 126 patients (95%) completing the trial. There were no significant differences between groups in either of the following:

• Median integrated esophageal relaxation pressure: 9.9 mm Hg in the POEM group and 12.6 mm Hg in the dilation group, for a difference of 2.7 mm Hg (95% CI -2.1 to 7.5, P=0.07)

• Median barium column height: 2.3 cm in the POEM arm vs 0 cm in the dilation arm, for a difference of 2.3 cm (95% CI 1.0-3.6, P=0.05)

Reflux esophagitis, however, was more frequent after POEM than dilation: 22 of 54 patients (41%) compared with 2 of 29 patients (7%), respectively (34% difference, 95% CI 12-49%, P=0.002).

POEM was also associated with greater use of proton pump inhibitors, and 8% of patients in the POEM group had reflux of severe grade.

Two serious adverse events, including one perforation, occurred after pneumatic dilation, while there were no serious adverse events after POEM.

"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," the authors pointed out.

They also noted that previous have reported a similar therapeutic efficacy with POEM, although the new study appears to be the first randomized controlled trial to evaluate POEM as first-line treatment.

Study limitations, the authors said, including the unblinded design and lack of a strict intention-to-treat analysis, which resulted in exclusion of randomized patients who were never treated or followed up. Furthermore, the start time for follow-up was treatment initiation rather than randomization, and the follow-up time differed between the treatment groups (24 months for POEM vs 24.5 months for the dilation group). And no conclusions can be drawn for the longer-term treatment success of POEM, especially since achalasia is a lifelong chronic disease, the team added.

In an , Brian C. Jacobson, MD, MPH, and David R. Lichtenstein, MD, of Boston University School of Medicine, said the findings suggest that POEM is more effective than pneumatic dilation for symptom control, at least within a 2-year timeframe. They cautioned, however, that "the higher incidence of clinically significant [gastroesophageal reflux disorder] and invasive nature of POEM should be accounted for in any discussion with patients regarding treatment options."

Jacobson and Lichtenstein noted that the current trial's low rate of success with pneumatic dilation relative to that of earlier studies can be largely explained by a protocol in which dilation was limited to one or two procedures with 30-mm and 35-mm balloons. reported success rates of 85-90% at 2- to 5-year follow-up and allowed sequential dilation from 30-40 mm at several-week intervals until adequate symptom response was achieved.

The editorialists said that looking ahead, POEM's acceptance as a first-line therapy "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."