Peroral endoscopic myotomy is the superior initial treatment strategy for achalasia, according to study results published in The Lancet Gastroenterology & Hepatology.
For the multi-center, randomized controlled trial, researchers enrolled patients (N=133) with newly diagnosed achalasia from 2012 to 2015 at 6 hospitals in the Netherlands, Germany, Italy, the United States, and Hong Kong. Patients were randomly assigned 1:1 to receive peroral endoscopic myotomy (n=67) or pneumatic dilation (n=66). For this analysis, 5-year outcomes were assessed. Treatment success was defined as Eckardt score less than or equal to 3 with no severe treatment-related complications or need for retreatment.
The peroral endoscopic myotomy and pneumatic dilation cohorts comprised patients with a median age of 47 (IQR, 37-56) and 50 (IQR, 32-62) years; 52% and 61% were men; they had a BMI of 23.2 (IQR, 3.7) and 23.4 (IQR, 4.1) kg/m2; 66% and 59% had achalasia subtype II; Eckardt scores at baseline were 8 (IQR, 6-9) and 7 (IQR, 6-9); and achalasia-specific quality of life (achalasia-DSQoL) scores were 8 (IQR, 6-11) and 8 (IQR, 6-10) points, respectively.
Similar to outcomes at 3 months and 1 and 2 years, peroral endoscopic myotomy was superior to pneumatic dilation, with 5-year treatment success rates of 81% and 40% (P <.0001), respectively. Patients in the peroral endoscopic myotomy and pneumatic dilation cohorts had treatment failure due to recurrence of symptoms (18% vs 40%), initial treatment failure (2% vs 19%), and adverse events (0% vs 2%), respectively.
By the 5-year follow-up, 13% in the peroral endoscopic myotomy group had been retreated, with 11% receiving pneumatic dilation and 2% laparoscopic Heller myotomy. For the pneumatic dilation group, 48% had been retreated, with 41% undergoing additional pneumatic dilation, 14% peroral endoscopic myotomy after the additional pneumatic dilation, 8% peroral endoscopic myotomy without additional pneumatic dilation, and 2% laparoscopic Heller myotomy.
For secondary outcomes, the 2 intervention groups had similar Eckardt scores, except that the pneumatic dilation cohort had significantly lower regurgitation subscores than the peroral endoscopy myotomy group (P =.025). The pneumatic dilation group also had a smaller Barium column diameter at 5 cm (median, 1.9 vs 2.8 cm; P =.0009) and fewer used proton-pump inhibitors (13% vs 46%; P =.0082), but they had a lower Gastroesophageal Reflux Disease Questionnaire (GERDQ) score (median, 6 vs 7; P =.0081) compared with the peroral endoscopic myotomy cohort, respectively.
In the posthoc sensitivity analysis, peroral endoscopic myotomy remained favored over pneumatic dilation. An additional analysis stratified by age found that patients younger than 40 years of age had a higher peroral endoscopic myotomy failure rate (44%) compared with patients aged 40 years or older (9%; P =.0033). No similar pattern was observed in the pneumatic dilation cohort (73% vs 54%; P =.21), respectively.
The secondary outcomes in this study may have been biased, as evaluating only treatment successes would have included selection bias but evaluating all patients would also be biased owing to the large amount of cross-over among patients who experienced treatment failure.
“Peroral endoscopic myotomy results in a significantly greater long-term treatment success than a single series pneumatic dilation (30-35 mm) in treatment-naive patients with achalasia,” the study authors noted. “The incidence of reflux symptoms and proton-pump inhibitor use remains high 5 years after peroral endoscopic myotomy. Based on this study, peroral endoscopic myotomy should be proposed as an initial treatment option for patients with achalasia.”
Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Kuipers T, Ponds FA, Fockens P, Bastiaansen BAJ, et al. Peroral endoscopic myotomy versus pneumatic dilation in treatment-naive patients with achalasia: 5-year follow-up of a randomised controlled trial. Lancet Gastroenterol Hepatol. Published online October 4, 2022. doi:10.1016/S2468-1253(22)00300-4