Esophagus to Small Intestine
Lancet Gastroenterol Hepatol. 2022;7(12):1103–11
Peroral endoscopic myotomy versus pneumatic dilation in treatment-naive patients with achalasia: 5-year follow-up of a randomized controlled trial
Background: Two-year follow-up data from this randomized controlled trial showed that peroral endoscopic myotomy (POEM) is associated with a significantly higher efficacy than pneumatic dilation as initial treatment of therapy-naive patients with achalasia. Here, the authors report therapeutic success rates in patients treated with POEM compared with pneumatic dilation at the 5-year follow-up.
Methods: They did a multicenter, randomized controlled trial in 6 hospitals in the Netherlands, Germany, Italy, Hong Kong, and the USA. Adults aged 18–80 years with newly diagnosed symptomatic achalasia (based on an Eckardt score > 3) were eligible for inclusion. Patients were randomly assigned (1:1) to POEM or pneumatic dilation using web-based randomization with a random block size of 8 and stratification according to site. Randomization concealment for treatment type was double-blind until official study enrolment. Treatment was unmasked because of the different technical approach of each procedure. Patients in the pneumatic dilation group were dilated with a single series of 30–35-mm balloons. The need for subsequent dilations in the pneumatic dilation group, and the need for dilation after initial treatment in the POEM group, was considered treatment failure. The primary outcome was therapeutic success (Eckardt score ≤ 3 in the absence of severe treatment-related complications and no need for retreatment). Analysis of the primary outcome was by modified intention-to-treat, including all patients randomly assigned to a group, excluding those patients who did not receive treatment or were lost to follow-up. Safety was assessed in all included patients.
Findings: Between September 21, 2012, and July 20, 2015, 182 patients were assessed for eligibility, 133 of whom were included in the study and randomly assigned to POEM (n = 67) or pneumatic dilation (n = 66). Five-year follow-up data were available for 62 patients in the POEM group and 63 patients in the pneumatic dilation group. 50 (81%) patients in the POEM group had treatment success at 5 years, compared with 25 (40%) in the pneumatic dilation group, an adjusted absolute difference of 41% (95% confidence interval [CI]: 25–57; p < 0.0001). Reasons for failure were no initial effect of treatment (1 patient in the POEM group vs. 12 patients in the pneumatic dilation group) and recurrent symptoms causing treatment failure (11 patients in the POEM group [7 patients between 2 and 5 years] vs. 25 patients in the pneumatic dilation group [9 patients between 2 and 5 years]); 1 patient in the pneumatic dilation group had treatment failure due to an adverse event. Proton-pump inhibitor use (mostly daily) was significantly higher after POEM than after pneumatic dilation among patients still in clinical remission (23/50 patients [46%] vs. 3/24 patients [13%]; p = 0.008). Five-year follow-up endoscopy of patients still in clinical remission showed reflux esophagitis in 14 of 42 patients (33%) in the POEM group (12 [29%] grade A or B, 2 [5%] grade C or D) and 2 of 16 patients (13%) in the pneumatic dilation group (2 [13%] grade A or B, none grade C or D; p = 0.19). No intervention-related serious adverse events occurred between 2 and 5 years after treatment. The following non-intervention-related serious adverse events occurred between 2 and 5 years: a stroke (1 [2%]) in the POEM group; and death due to a melanoma (1 [2%]) and dementia (1 [2%]) in the pneumatic dilation group.
Interpretation: Based on this study, peroral endoscopic myotomy (POEM) should be proposed as an initial treatment option for patients with achalasia. Although this study has shown that POEM has greater long-term efficacy with a low risk of major treatment-related complications, this should not lead to abandonment of pneumatic dilation from clinical practice. Ideally, all treatment options should be discussed with treatment-naive patients with achalasia and a shared decision should be made.