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打破恶性循环:阿柏西普用于治疗难治性功能性排尿障碍患儿

Breaking the vicious circle: Onabotulinum toxin A in children with therapy-refractory dysfunctional voiding.

作者信息

't Hoen L A, van den Hoek J, Wolffenbuttel K P, van der Toorn F, Scheepe J R

机构信息

Department of Pediatric Urology, Sophia Children's Hospital, Erasmus Medical Center, Rotterdam, The Netherlands.

出版信息

J Pediatr Urol. 2015 Jun;11(3):119.e1-6. doi: 10.1016/j.jpurol.2014.10.006. Epub 2015 Feb 25.

DOI:10.1016/j.jpurol.2014.10.006
PMID:25794865
Abstract

INTRODUCTION

An increased activity of the external urethral sphincter or pelvic floor muscles during voluntary voiding leads to dysfunctional voiding. Frequently reported symptoms are urinary incontinence, urinary tract infections and high post-void residuals. Dysfunctional voiding is a common problem in school-aged children and despite various treatment options, 10-40% of the children remain therapy-refractory.

OBJECTIVE

The aim of this study is to evaluate the effectiveness of Onabotulinum toxin-A (BTX-A) injections in the external urethral sphincter in children with therapy-refractory dysfunctional voiding.

PATIENTS AND METHODS

Patients with therapy-refractory dysfunctional voiding who have received BTX-A injections in the external urethral sphincter from 2010 to 2013 were analysed. Children with known neuropsychiatric disorders were excluded. All children had abnormal flow patterns and increased pelvic floor tone during uroflowmetry/EMG studies. They had received at least five sessions of urotherapy and two sessions of pelvic floor physical therapy prior to treatment. A total of 100 IU of BTX-A was injected in the external urethral sphincter at the 3, 9 and 12 o'clock positions. Our main outcome measures were urinary incontinence, recurrent urinary tract infections and post-void residual.

RESULTS

A total of twenty patients, of whom 16 girls, with a median age of 9 years (range 5-14) were treated with BTX-A. The median follow-up was 13 months (range 5-34). Post-void residual decreased by 75% after BTX-A, from a median of 47.5 ml (16.3-88.5 ml) to 0 ml (0.0-28.0 ml) (p = 0.001) Six patients had a post-void residual < 20 ml prior to treatment. After BTX-A sixteen patients had a post-void residual <20 ml (Figure). No significant changes in uroflowmetry results was seen. Sixteen children are no longer daily incontinent, of whom 9 became completely dry (p = 0.0001). Eleven patients suffered from recurrent urinary tract infections prior to treatment. After BTX-A five children remained infection free, while the other six experienced only one urinary tract infection during follow-up (p = 0.003). Fourteen patients received additional urotherapy after BTX-A. Repeat injections were necessary in four patients after initial satisfactory results, with repeated good clinical responses. Two children showed no improvement after first BTX-A injection. No serious adverse events were reported.

DISCUSSION

The results in this homogenous group of patients confirm the conclusions of previous studies in opting BTX-A in the external urethral sphincter to be a viable treatment option for the therapy-refractory group of patients with dysfunctional voiding. What is new, is that in most of our patients post-injection urotherapy was used to amplify the BTX-A effect. During our long-term follow-up the satisfactory results were sustained, similar to the results of the long-term follow-up presented by Vricella et al. [1]. The retrospective character and relative small sample size are limitations of this study.

CONCLUSIONS

This study shows safe and persistent satisfactory results during our average 13-month follow-up in 90% of our patients with therapy-refractory dysfunctional voiding. A prospective study using validated and standardized measurements will be performed to affirm our results and evaluate the exact role of post-injection urotherapy.

摘要

引言

在自主排尿期间,尿道外括约肌或盆底肌肉活动增强会导致排尿功能障碍。常见症状包括尿失禁、尿路感染和排尿后残余尿量增多。排尿功能障碍在学龄儿童中是一个常见问题,尽管有多种治疗选择,但仍有10% - 40%的儿童治疗效果不佳。

目的

本研究旨在评估注射A型肉毒杆菌毒素(BTX - A)至尿道外括约肌对治疗效果不佳的排尿功能障碍儿童的有效性。

患者与方法

分析2010年至2013年期间接受BTX - A注射至尿道外括约肌治疗且治疗效果不佳的排尿功能障碍患者。排除已知患有神经精神疾病的儿童。所有儿童在尿流率/肌电图检查中均有异常的尿流模式且盆底肌张力增加。在治疗前,他们均接受了至少五节排尿治疗课程以及两节盆底物理治疗课程。在尿道外括约肌的3点、9点和12点位置共注射100 IU的BTX - A。我们的主要观察指标为尿失禁、复发性尿路感染和排尿后残余尿量。

结果

共有20名患者接受了BTX - A治疗,其中16名女孩,中位年龄为9岁(范围5 - 14岁)。中位随访时间为13个月(范围5 - 34个月)。注射BTX - A后,排尿后残余尿量减少了75%,从中位值47.5 ml(16.3 - 88.5 ml)降至0 ml(0.0 - 28.0 ml)(p = 0.001)。治疗前有6名患者排尿后残余尿量<20 ml。注射BTX - A后,有16名患者排尿后残余尿量<20 ml(图)。尿流率结果未见显著变化。16名儿童不再每日尿失禁,其中9名完全不再尿床(p = 0.0001)。治疗前有11名患者患有复发性尿路感染。注射BTX - A后,5名儿童未再感染,另外6名儿童在随访期间仅经历了一次尿路感染(p = 0.003)。14名患者在注射BTX - A后接受了额外的排尿治疗。4名患者在初次获得满意效果后需要重复注射,且重复注射后临床反应良好。2名儿童在首次注射BTX - A后未见改善。未报告严重不良事件。

讨论

该同质患者组的结果证实了先前研究的结论,即选择向尿道外括约肌注射BTX - A是治疗效果不佳的排尿功能障碍患者的一种可行治疗选择。新的发现是,在我们的大多数患者中,注射后进行排尿治疗以增强BTX - A的效果。在我们的长期随访中,满意的结果得以维持,与Vricella等人[1]所呈现的长期随访结果相似。本研究的局限性在于其回顾性特点和相对较小的样本量。

结论

本研究表明,在我们对治疗效果不佳的排尿功能障碍患者平均13个月的随访中,90%的患者取得了安全且持久的满意结果。将进行一项使用经过验证和标准化测量的前瞻性研究,以证实我们的结果并评估注射后排尿治疗的确切作用。

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