Effect of Peroral Endoscopic Myotomy vs Pneumatic Dilation on Symptom Severity and Treatment Outcomes Among Treatment-Naive Patients With Achalasia: A Randomized Clinical Trial.
经口内镜下肌切开术与气压扩张术对未治疗的失弛缓症患者症状严重程度和治疗结果的影响: 一项随机临床试验。

摘要

Importance: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.
Objective: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.
Interventions: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.
Main Outcomes and Measures: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.
Results: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.
Conclusions and Relevance: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.
Trial Registration:Netherlands Trial Register number: NTR3593.

译文

重要性: 病例系列表明经口内镜下肌切开术 (POEM) 治疗失弛缓症患者的良好效果。缺乏将 POEM 与气压扩张术 (achalasia 患者的标准治疗方法) 进行比较的数据。
目的: 比较 POEM 与气动扩张作为治疗初治患者的疗效。
设计、背景和参与者: 这项随机多中心临床试验在荷兰、德国、意大利、香港和美国的 6 家医院进行。包括新诊断为失弛缓症且 Eckardt 评分大于 3 的未接受治疗的成年患者。该研究在 2012年9月至 2015年7月间进行,随访时间为首次治疗后 2 年,最终随访日期为 2017年11月22日。
干预: 随机接受 POEM (n =-67) 或用 30mm 和 35mm 气球 (n =-66) 进行气动扩张,根据医院进行分层。
主要结果和措施: 主要结果是 2 年随访的治疗成功 (定义为 Eckardt 评分 ≤ 3,没有严重并发症或再治疗)。在没有治疗失败的患者中,总共检查了 14 个次要终点,包括通过高分辨率测压法对食管下括约肌进行的综合松弛压力、定时食管钡剂图上的钡剂柱高度、以及反流性食管炎的存在。
结果: 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,气动扩张组 66 名) 和 126 (95%) 完成了研究。治疗成功的主要结果发生在 POEM 组 63 名患者中的 58 名 (92%),而气动扩张组 63 名患者中的 34 名 (54%), 差异为 38% ([95% CI,22%-52%]; p   < 。 001)。在 14 个预先设定的次要终点中,10 个终点没有显示出组间的显著差异。组间的平均综合松弛压力没有显著差异 (POEM 组为 9.9毫米汞柱,气动扩张组为 12.6毫米汞柱; 差异,2.7毫米汞柱 [95% CI, -2.1 到 7.5]; p   =  。 07) 或钡中柱高度(诗歌组为 2.3厘米,气动扩张组为 0厘米; 差异,2.3厘米 [95% CI,1.0-3.6]; p =)。反流性食管炎在 POEM 组比气动扩张组更常见 (54 [41%] 中的 22 vs 29 [7%] 中的 2; 差异,34% [95% CI, 12%-49%]; p   =  。 002)。气动扩张后发生 2 次严重不良事件,包括 1 次穿孔,而 POEM 术后未发生严重不良事件。
结论和相关性: 在未接受治疗的失弛缓症患者中,与气动扩张相比,POEM 治疗 2 年的治疗成功率明显更高。这些发现支持将 POEM 作为失弛缓症患者的初始治疗选择。
试用注册: 荷兰试用注册号码: ntr3593。

achalasia

儿科 食管动力障碍性疾病 疾病
概述  :  

贲门失弛缓症是一种食管动力障碍性疾病,主要表现为吞咽困难、胸痛、反流、呕吐和体重下降,其病理生理改变主要是下食管括约肌(lower esophageal sphincter,LES)松弛障碍和食管体部无效收缩。 发病机制一、神经机制下食管括约肌抑制性神经元改变:位于食管和胃连接部的LES形成生理性高压带,以防止胃内容物反流至食管;吞咽时LES短暂舒张,允许食团进入胃内。迷走神经背核(包括腹侧的兴奋性神经元和尾部的抑制性神经元)发出的运动纤维支配LES肌间神经丛神经元,调节LES舒

achalasia   英 /,ækə'leiziə/  美 /,ækə'leiziə/

释    义   n. [内科] 失弛缓性,弛缓不能

例    句   Achalasia surgery should preferably be performed by surgeons with special knowledge, trainingand experience in the management of gastroesophageal swallowing disorders. 贲门失弛缓症手术最好的表演与特殊的知识,培训和胃吞咽障碍的管理经验的外科医生。

短    语   esophageal achalasia 食管失弛症

primary achalasia 原发性贲门失弛缓症

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