Department of Rehabilitation Medicine, The Third Affiliated Hospital, Sun Yat-sen University, 600 Tianhe Road, Guangzhou, 510630, Guangdong Province, China.
Trials. 2021 Mar 31;22(1):238. doi: 10.1186/s13063-021-05195-8.
Cricopharyngeal dysfunction (CPD) occurs in various neurological disorders, especially stroke. The treatment approaches of CPD include swallowing training, cricopharyngeal dilation, botulinum toxin (BTX) injection, and cricopharyngeal myotomy. BTX injection into the cricopharyngeal muscle is effective and safe for some patients with dysphagia, with a success rate between 43 and 100% (mean = 76%). However, well-designed randomized controlled clinical trials are needed to verify its efficacy and safety for the treatment of CPD. The objective of this study is to explore the efficacy and safety of BTX for neurogenic cricopharyngeal achalasia, when administering an injection into the cricopharyngeal muscle guided by a novel precise positioning method, that combines ultrasound, catheter balloon, and electromyography (BECURE).
BECURE is a single-center randomized, placebo controlled, double-blinded, superiority clinical trial. To detect a significant difference between the 2 groups, a sample size of 44 patients is estimated. The intervention is BTX versus placebo, with 1:1 randomization. The randomization sequence from 1 to 44 was generated using the Statistical Package for Social Sciences. The study is divided into two phases. In the first phase, patients will be injected with BTX or the placebo. In the second phase, patients who received a placebo injection and those who did not respond to the first BTX injection will receive an injection of BTX. The primary outcome is the score of the Functional Oral Intake Scale (FOIS). The secondary outcomes are as follows: upper esophageal sphincter (UES) residual pressure, UES resting pressure, duration of UES relaxation, velopharyngeal and laryngopharyngeal peak pressure, UES opening, pharyngeal construction ratio, residue of bolus in the epiglottis valley or piriform sinus, and penetration and aspiration.
Dysphagia is a common complication of stroke. There is lack of high-quality evidence for the efficacy of BTX in treating neurogenic CPD. This study will clarify whether BTX injection into the cricopharyngeal muscle can be effective and safe for patients with stroke and CPD.
Chinese Clinical Trial Register ( ChiCTR1900025562 ). Registered on September 1, 2019.
环咽肌功能障碍（CPD）发生于多种神经疾病，特别是中风。CPD 的治疗方法包括吞咽训练、环咽肌扩张、肉毒毒素（BTX）注射和环咽肌切开术。BTX 注射到环咽肌对一些吞咽困难的患者是有效且安全的，成功率在 43%到 100%之间（平均值为 76%）。然而，需要进行精心设计的随机对照临床试验来验证其治疗 CPD 的疗效和安全性。本研究旨在探索使用一种新的精确定位方法（结合超声、导管球囊和肌电图的 BECURE）将 BTX 注射到环咽肌治疗神经源性环咽肌失弛缓的疗效和安全性。
BECURE 是一项单中心、随机、安慰剂对照、双盲、优效性临床试验。估计需要 44 例患者的样本量，以检测两组之间的显著差异。干预措施为 BTX 与安慰剂，采用 1:1 随机分组。使用社会科学统计软件包生成 1 到 44 的随机序列。该研究分为两个阶段。在第一阶段，患者将接受 BTX 或安慰剂注射。在第二阶段，接受安慰剂注射且对首次 BTX 注射无反应的患者将接受 BTX 注射。主要结局是功能性口服摄入量表（FOIS）评分。次要结局如下：上食管括约肌（UES）残余压、UES 静息压、UES 松弛持续时间、软腭和咽缩肌峰值压、UES 开放度、咽腔构建比、会厌谷或梨状隐窝内食团残留、渗透和吸入。
吞咽困难是中风的常见并发症。BTX 治疗神经源性 CPD 的疗效缺乏高质量证据。本研究将阐明 BTX 注射到环咽肌是否对中风和 CPD 患者有效且安全。
中国临床试验注册中心（ChiCTR1900025562）。注册于 2019 年 9 月 1 日。
