Emma Children's Hospital, Amsterdam UMC, University of Amsterdam and Vrije Universiteit Amsterdam, Department of Pediatric Surgery, Amsterdam Gastroenterology and Metabolism Research Institute and Amsterdam Reproduction and Development Research Institute, Amsterdam, Netherlands; Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Follow Me Aftercare Program, Department of Pediatrics, Amsterdam Reproduction and Development Research Institute, Amsterdam, Netherlands.
Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Follow Me Aftercare Program, Department of Pediatrics, Amsterdam Reproduction and Development Research Institute, Amsterdam, Netherlands.
J Pediatr Surg. 2021 Aug;56(8):1342-1348. doi: 10.1016/j.jpedsurg.2020.11.025. Epub 2020 Nov 27.
Patients with Hirschsprung disease may have obstructive symptoms after resection of the aganglionic segment. Botulinum toxin (BT) injections can help improve faecal passage by relaxing the internal anal sphincter. This study assess effect of BT injections and aims to identify factors associated with receiving BT injections and favourable response to the first BT injection.
A retrospective study was performed in a cohort of consecutive patients treated for Hirschsprung disease in our centre between 2003 and 2017. The indication for BT injections was obstructive defecation problems that were non-responsive to high-dose laxatives or rectal irrigation, or an episode of Hirschsprung-associated enterocolitis (HAEC). Effectiveness of BT injections was measured in terms of clinical improvement. Relationships between factors associated with receiving BT injections and with response to the first BT injection were tested with group comparison and logistic regression.
Forty-one out of 131 patients received BT injections (31%) with a median of two injections (range 1-11). All patients had obstructive defecation problems non-responsive to high-dose laxatives or rectal irrigation, two patient also had an episode of HAEC. Twenty-five out of 41 patients (61%) had clinical improvement after first injection. In 29 of the 41 patients (71%) spontaneous defecation or treatment with laxatives only was achieved. Adverse effects were seen in 12 out of 41 patients (29%) after 14 injections (16%), and consisted of anal pain, temporary loss of stools and dermatitis. Patients who received BT injections more often had long segment disease, more often required laxatives or rectal irrigation and had longer length of hospital stay, both after corrective surgery and in follow-up. None of the tested factors was associated with clinical improvement after first BT injection.
Our findings show that BT injections effectively treat obstructive defecation problems in the majority of patients with Hirschsprung disease with mild adverse effects.
Level III.
先天性巨结肠症患者在切除无神经节细胞段后可能出现梗阻症状。肉毒杆菌毒素（BT）注射可通过松弛内括约肌帮助改善粪便排泄。本研究评估 BT 注射的效果，并旨在确定与接受 BT 注射相关的因素以及对首次 BT 注射的良好反应。
对 2003 年至 2017 年间在我们中心接受先天性巨结肠症治疗的连续患者队列进行回顾性研究。BT 注射的适应证为对高剂量泻药或直肠灌洗无反应的梗阻性排便问题，或发生先天性巨结肠相关性结肠炎（HAEC）。BT 注射的有效性通过临床改善来衡量。采用组间比较和逻辑回归检验与接受 BT 注射相关的因素和首次 BT 注射反应之间的关系。
41 例患者（31%）接受了 BT 注射（共 41 例），中位数为 2 次（范围 1-11 次）。所有患者均有对高剂量泻药或直肠灌洗无反应的梗阻性排便问题，2 例患者还发生了 HAEC。首次注射后 25 例患者（61%）临床改善。在 41 例患者中，29 例（71%）自发排便或仅用泻药即可达到治疗效果。41 例患者中有 12 例（29%）在 14 次注射（16%）后出现不良反应，包括肛门疼痛、暂时性大便失禁和皮炎。接受 BT 注射的患者更常患有长段疾病，更常需要使用泻药或直肠灌洗，且在矫正手术后和随访期间住院时间更长。未发现测试的因素与首次 BT 注射后的临床改善相关。
我们的研究结果表明，BT 注射可有效治疗大多数先天性巨结肠症患者的梗阻性排便问题，且不良反应轻微。
III 级。
