BACKGROUND Complete inability to belch due to retrograde cricopharyngeus dysfunction (R-CPD) may lead to chronic gas-related gastrointestinal complaints. We aimed to validate high-resolution manometric (HRM) diagnostic criteria, and prospectively evaluate the feasibility and efficacy of cricopharyngeal botulinum toxin injection (CBTI) via flexible endoscopy. METHODS Consecutive manometrically diagnosed R-CPD patients were included. Asymptomatic volunteers were also included for diagnostic validation. R-CPD patients underwent CBTI (treatment group) or deferred/declined treatment (control group). Outcomes included ability to belch, clinical symptoms and quality of life measured using self-report questionnaires. RESULTS Sixty-five subjects were included (52 treatment group, 7 controls, 6 asymptomatic volunteers). All R-CPD patients had inability to belch since childhood. During HRM with carbonated drink provocative testing, all R-CPD patients demonstrated characteristic esophageal pressurization patterns associated with failure of upper esophageal sphincter relaxation; these findings were never seen in asymptomatic volunteers. At 3-months, 92% patients who received CBTI were able to belch (compared to 0 controls; P<0.001), and experienced improved clinical symptoms (global symptom score improved from 7.3 ± 1.7 to 1.8 ± 2.3, whereas in controls was static 7.5 ± 2.1 to 7.7 ± 1.8; P<0.0001 for comparison). Quality of life significantly improved in the treatment group but not controls (P=0.0002). At 3-months, 43/51 (84%) of the treatment group reported being 'satisfied' or 'very satisfied' with therapeutic outcome. CONCLUSION HRM with carbonated drink provocation demonstrates pathognomonic signs of R-CPD that were not seen in health. Flexible endoscopic cricopharyngeal botulinum toxin injection is highly effective for symptomatic relief compared to no treatment.

中文翻译：

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内窥镜下肉毒杆菌毒素注射治疗逆行环咽功能障碍、无法打嗝综合征的前瞻性对照研究。


背景 由于逆行环咽功能障碍 （R-CPD） 而完全无法打嗝可能导致慢性气体相关的胃肠道不适。我们旨在验证高分辨率测压 （HRM） 诊断标准，并前瞻性评价通过软式内窥镜进行环咽肉毒杆菌毒素注射 （CBTI） 的可行性和有效性。方法 纳入连续测压诊断的 R-CPD 患者。无症状志愿者也被纳入诊断验证。R-CPD 患者接受 CBTI （治疗组） 或延迟/拒绝治疗 （对照组）。结局包括打嗝能力、临床症状和使用自我报告问卷测量的生活质量。结果 共纳入 65 例受试者 （52 例治疗组，7 例对照组，6 例无症状志愿者）。所有 R-CPD 患者自幼无法打嗝。在碳酸饮料激发试验的 HRM 期间，所有 R-CPD 患者都表现出与食管上括约肌松弛失败相关的特征性食管加压模式;这些发现从未在无症状志愿者中发现。在 3 个月时，92% 接受 CBTI 的患者能够打嗝（相比之下，对照组为 0;P<0.001），临床症状有所改善（总体症状评分从 7.3 ± 1.7 提高到 1.8 ± 2.3，而对照组为静态 7.5 ± 2.1 提高到 7.7 ± 1.8;P<0.0001 进行比较）。治疗组生活质量显著改善，对照组未见 （P=0.0002）。在 3 个月时，治疗组中有 43/51 （84%） 报告对治疗结果“满意”或“非常满意”。结论 HRM 伴碳酸饮料激发表现出 R-CPD 的特异性体征，这些体征在健康中未见。 与不治疗相比，软式内窥镜环咽肉毒杆菌毒素注射对缓解症状非常有效。