Department of Gastroenterology and Hepatology, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands.
Department of Gastroenterology, Evangelisches Krankenhaus Düsseldorf, Düsseldorf, Germany.
JAMA. 2019 Jul 9;322(2):134-144. doi: 10.1001/jama.2019.8859.
Case series suggest favorable results of peroral endoscopic myotomy (POEM) for treatment of patients with achalasia. Data comparing POEM with pneumatic dilation, the standard treatment for patients with achalasia, are lacking.
To compare the effects of POEM vs pneumatic dilation as initial treatment of treatment-naive patients with achalasia.
DESIGN, SETTING, AND PARTICIPANTS: This randomized multicenter clinical trial was conducted at 6 hospitals in the Netherlands, Germany, Italy, Hong Kong, and the United States. Adult patients with newly diagnosed achalasia and an Eckardt score greater than 3 who had not undergone previous treatment were included. The study was conducted between September 2012 and July 2015, the duration of follow-up was 2 years after the initial treatment, and the final date of follow-up was November 22, 2017.
Randomization to receive POEM (n = 67) or pneumatic dilation with a 30-mm and a 35-mm balloon (n = 66), with stratification according to hospital.
The primary outcome was treatment success (defined as an Eckardt score ≤3 and the absence of severe complications or re-treatment) at the 2-year follow-up. A total of 14 secondary end points were examined among patients without treatment failure, including integrated relaxation pressure of the lower esophageal sphincter via high-resolution manometry, barium column height on timed barium esophagogram, and presence of reflux esophagitis.
Of the 133 randomized patients, 130 (mean age, 48.6 years; 73 [56%] men) underwent treatment (64 in the POEM group and 66 in the pneumatic dilation group) and 126 (95%) completed the study. The primary outcome of treatment success occurred in 58 of 63 patients (92%) in the POEM group vs 34 of 63 (54%) in the pneumatic dilation group, a difference of 38% ([95% CI, 22%-52%]; P < .001). Of the 14 prespecified secondary end points, no significant difference between groups was demonstrated in 10 end points. There was no significant between-group difference in median integrated relaxation pressure (9.9 mm Hg in the POEM group vs 12.6 mm Hg in the pneumatic dilation group; difference, 2.7 mm Hg [95% CI, -2.1 to 7.5]; P = .07) or median barium column height (2.3 cm in the POEM group vs 0 cm in the pneumatic dilation group; difference, 2.3 cm [95% CI, 1.0-3.6]; P = .05). Reflux esophagitis occurred more often in the POEM group than in the pneumatic dilation group (22 of 54 [41%] vs 2 of 29 [7%]; difference, 34% [95% CI, 12%-49%]; P = .002). Two serious adverse events, including 1 perforation, occurred after pneumatic dilation, while no serious adverse events occurred after POEM.
Among treatment-naive patients with achalasia, treatment with POEM compared with pneumatic dilation resulted in a significantly higher treatment success rate at 2 years. These findings support consideration of POEM as an initial treatment option for patients with achalasia.
Netherlands Trial Register number: NTR3593.
病例系列研究表明，经口内镜下肌切开术（POEM）治疗贲门失弛缓症患者效果良好。缺乏比较 POEM 与气囊扩张术（贲门失弛缓症的标准治疗方法）的相关数据。
比较 POEM 与气囊扩张术作为未经治疗的贲门失弛缓症患者初始治疗的效果。
设计、地点和参与者：这是一项在荷兰、德国、意大利、中国香港和美国的 6 家医院进行的随机多中心临床试验。纳入新诊断为贲门失弛缓症且 Eckardt 评分大于 3 分且未接受过治疗的成年患者。该研究于 2012 年 9 月至 2015 年 7 月进行，初始治疗后随访 2 年，最终随访日期为 2017 年 11 月 22 日。
随机分为 POEM 组（n = 67）或气囊扩张组（30mm 和 35mm 球囊，n = 66），根据医院分层。
主要结局是 2 年随访时的治疗成功率（定义为 Eckardt 评分≤3 且无严重并发症或再次治疗）。在无治疗失败的患者中，共检查了 14 个次要终点，包括高分辨率测压法下食管下括约肌整合松弛压、钡餐食管造影时钡柱高度和反流性食管炎的存在。
在 133 名随机患者中，130 名（平均年龄 48.6 岁；73 名[56%]男性）接受了治疗（POEM 组 64 名，气囊扩张组 66 名），126 名（95%）完成了研究。POEM 组 63 例患者中 58 例（92%）达到治疗成功的主要结局，而气囊扩张组 63 例患者中 34 例（54%），差异为 38%（[95%CI，22%-52%]；P < .001）。在 14 个预先指定的次要终点中，10 个终点在两组间无显著差异。两组间中位食管下括约肌整合松弛压（POEM 组 9.9mmHg，气囊扩张组 12.6mmHg；差异 2.7mmHg[95%CI，-2.1 至 7.5]；P = .07）或中位钡柱高度（POEM 组 2.3cm，气囊扩张组 0cm；差异 2.3cm[95%CI，1.0-3.6]；P = .05）均无显著差异。POEM 组反流性食管炎发生率高于气囊扩张组（22 例[41%]比 2 例[7%]；差异 34%[95%CI，12%-49%]；P = .002）。气囊扩张后发生 2 例严重不良事件，包括 1 例穿孔，而 POEM 后无严重不良事件发生。
在未经治疗的贲门失弛缓症患者中，与气囊扩张术相比，POEM 治疗 2 年后的治疗成功率显著提高。这些发现支持将 POEM 作为贲门失弛缓症患者的初始治疗选择。
荷兰临床试验注册中心编号：NTR3593。
