麻豆传媒

In idiopathic achalasia patients a less invasive procedure proved equal to a surgical approach in controlling symptoms at 2 years but resulted in more cases of gastroesophageal reflux, according to an international randomized trial.

Thomas Rösch, MD, of University Hospital Hamburg-Eppendorf in Germany, and colleagues compared the efficacy of peroral endoscopic myotomy (POEM) with laparoscopic Heller's myotomy (LHM) plus Dor's fundoplication and found POEM to be non-inferior in the study's primary endpoint.

At 2-year follow-up, clinical success – defined as an of 3 or less with no additional treatments – was observed in 83.0% of POEM patients and 81.7% of LHM patients (95% confidence interval –8.7-11.4, P=0.007 for non-inferiority).

"Our results could assist in shared decision making in selecting the appropriate individual therapy for patients with primary achalasia," wrote the European-North American research team in the study online in the .

Study Details

From December 2012 to October 2015, a total of 221 patients at eight centers in six countries were randomly assigned in an open-label fashion to either POEM (112 patients) or LHM plus Dor's fundoplication (109 patients). The mean overall age of participants was 48.6, and 60.7% of the POEM patients and 55% of the LHM patients were male. Baseline esophageal function was similar in the two groups, with an integrated relaxation pressure of 26.8 and 26 mmHg, respectively.

Some patients in both the POEM and LHM arms had undergone previous therapies, including dilation (24.1% and 28.4%, respectively), botulinum toxin injection (6.2% and 7.3%), or both (4.5% and 0.9%).

Based on a validated questionnaire, the Eckardt score is calculated as the sum of symptom scores of four achalasia components: dysphagia, regurgitation, chest pain, and weight loss to a maximum of 12, with higher scores indicating worse symptoms. Baseline Eckardt scores were nearly identical in both arms: POEM 6.8, LHM 6.7.

Improvement in esophageal function from baseline to 24 months, as assessed by the integrated relaxation pressure of the lower esophageal sphincter, did not vary significantly between treatment arms: difference –0.75 mmHg (95% CI –2.26-0.76), the researchers reported. Nor was there a significant inter-arm difference in improvement in the Gastrointestinal Quality of Life Index score: difference 0.14 points (95% CI –4.0-4.28).

At 3 months, however, endoscopy-diagnosed reflux esophagitis was more prevalent in the POEM group: 57% vs 20% in the LHM group. At 24 months, the corresponding percentages were 44% and 29%, respectively.

In other trial outcomes, serious adverse events occurred in 2.7% of participants in the POEM arm vs 7.3% in the LHM arm, and no POEM patients required conversion via LHM.

Procedure time was shorter in the POEM group by 13.81 minutes (95% CI 6.26-21.36), but length of hospital stay did not differ significantly between the groups: difference of just 0.26 days (95% CI –0.12-0.63).

Asked for his perspective, David R. Lichtenstein, MD, of Boston University School of Medicine, who was not involved with the research, said: "This is an excellent study. Although the outcomes are not surprising, they are supportive of the non-surgical POEM approach to achalasia."

Also commenting, Amit Bhatt, MD, of the Cleveland Clinic, told 麻豆传媒 that while previous retrospective studies suggested that POEM might be as effective as traditional Heller's myotomy, "this trial now proves this with a well-designed multicenter, randomized non-inferiority study, showing similar clinical success at 2 years."

"This study proves POEM is not only an elegantly designed procedure but also an effective procedure compared with traditional surgery," Bhatt said.

Regarding the higher rate of acid reflux with POEM, Bhatt said the clinical significance is still to be determined. "Will we see an increase in Barrett's esophagus-related complications, for example? But procedural innovation is already on its way – POEM with fundoplication, which may be able to address the increased reflux seen with POEM," he added.

Earlier this year, another comparative study reported that POEM had a higher treatment success rate at 2 years than pneumatic dilatation, but like the current trial, it was found that POEM resulted in more reflux esophagitis and also showed a greater use of proton pump inhibitors in POEM recipients.

The study had several limitations, the authors said. Fewer than 50% of eligible patients participated in the trial, mainly because of failure to consent to randomization. In addition, the participating surgeons were more experienced in performing LHM plus Dor's fundoplication than the endoscopists were in performing POEM. Furthermore, the study did not analyze treatment effects on postoperative pain or the need for pain medications. Lastly, because of the unblinded design, patients and trial personnel were aware of their treatment group assignment, potentially constituting a source of bias since the trial's primary end point was based on patient-reported symptoms.

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