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新型β3肾上腺素能受体激动剂维贝格隆对膀胱过度活动症儿童及青少年下尿路功能疗效的尿动力学评估

Urodynamic evaluation of the efficacy of vibegron, a new β3-adrenergic receptor agonist, on lower urinary tract function in children and adolescents with overactive bladder.

作者信息

Kitta Takeya, Chiba Hiroki, Kon Masafumi, Higuchi Madoka, Kusakabe Naohisa, Ouchi Mifuka, Togo Mio, Abe-Takahashi Yui, Tsukiyama Mayuko, Shinohara Nobuo

机构信息

Department of Renal and Genitourinary Surgery, Graduate School of Medicine, Hokkaido University, Sapporo, Japan.

Department of Renal and Genitourinary Surgery, Graduate School of Medicine, Hokkaido University, Sapporo, Japan.

出版信息

J Pediatr Urol. 2022 Oct;18(5):563-569. doi: 10.1016/j.jpurol.2022.07.012. Epub 2022 Jul 30.

DOI:10.1016/j.jpurol.2022.07.012
PMID:35965225
Abstract

INTRODUCTION

Idiopathic overactive bladder (OAB) is defined as an urgency symptom with or without urge incontinence, which is not due to known neurological abnormalities. Since children present with variable symptoms, pediatric nonneurogenic idiopathic OAB is a condition that is difficult to diagnose and treat. Although there are few reports on bladder function in pediatric patients compared to adult patients, it can be useful for diagnosis. Antimuscarinic therapy is the pharmacological mainstay of OAB management. However, antimuscarinic use is limited by side effects and Insufficient effects. Vibegron, a new drug with a different mechanism of action (β3-adrenoreceptor agonist), was recently introduced for treating OAB in adults but has not been studied in the pediatric population.

OBJECTIVE

This study aimed to determine the efficacy and tolerability of vibegron in children and adolescents with idiopathic OAB.

STUDY DESIGN

We conducted a retrospective study enrolling pediatric patients with OAB whose symptoms did not improve with behavioral therapy or pharmaceutical therapy. Efficacy and tolerability were assessed via a question, and patients underwent video-urodynamic testing before and during treatment with once-daily 50 mg vibegron. Statistical differences were evaluated using Wilcoxon matched-pairs signed-rank tests.

RESULTS

Out of the 17 patients that were recruited, full study with two urodynamic studies were confirmed by 11 patients. OAB symptoms improved in 14 (82.4%) patients, and 3 patients discontinued treatment because of ineffectiveness. No patients discontinued treatment because of intolerance to vibegron. The median (IQR) first desire to void (133 [82-185]-161 [123-227] mL), bladder capacity (158 [136-238]-204 [150-257] mL), and bladder compliance (18.1 [9.1-76.7]-34.0 [30.0-82.3] mL/cm HO) improved significantly post treatment compared to before treatment. Detrusor overactivity disappeared in one of the eight patients with this condition. The parameters of voiding function did not change significantly after the administration of vibegron.

DISCUSSION

Treatment with vibegron significantly improved clinical and urodynamic parameters of pediatric OAB with no adverse effects. Little information is available regarding the feasibility of switching drugs when patients discontinue prior pharmacological therapy because of insufficient efficacy or poor tolerability in children. Vibegron may be a promising OAB treatment option with a better balance of efficacy and tolerability.

CONCLUSIONS

Vibegron is an alternative agent for pediatric patients with idiopathic OAB for improving both subjective symptoms and lower urinary tract function. Future prospective randomized studies with larger sample sizes must be conducted to validate the results of the present study.

摘要

引言

特发性膀胱过度活动症(OAB)被定义为伴有或不伴有急迫性尿失禁的尿急症状,且并非由已知的神经异常引起。由于儿童表现出的症状各不相同,小儿非神经源性特发性OAB是一种难以诊断和治疗的疾病。尽管与成年患者相比,关于小儿患者膀胱功能的报道较少,但这对诊断可能有用。抗毒蕈碱疗法是OAB治疗的主要药物手段。然而,抗毒蕈碱药物的使用受到副作用和疗效不足的限制。维贝格隆是一种作用机制不同的新药(β3肾上腺素能受体激动剂),最近被引入用于治疗成人OAB,但尚未在儿科人群中进行研究。

目的

本研究旨在确定维贝格隆对患有特发性OAB的儿童和青少年的疗效和耐受性。

研究设计

我们进行了一项回顾性研究,纳入了行为疗法或药物疗法后症状未改善的小儿OAB患者。通过一个问题评估疗效和耐受性,患者在每日一次服用50mg维贝格隆治疗前和治疗期间接受视频尿动力学检测。使用Wilcoxon配对符号秩检验评估统计学差异。

结果

在招募的17例患者中,11例患者完成了两项尿动力学研究的完整研究。14例(82.4%)患者的OAB症状得到改善,3例患者因无效而停止治疗。没有患者因对维贝格隆不耐受而停止治疗。与治疗前相比,治疗后首次排尿欲望的中位数(IQR)(133[82 - 185] - 161[123 - 227]mL)、膀胱容量(158[136 - 238] - 204[150 - 257]mL)和膀胱顺应性(18.1[9.1 - 76.7] - 34.0[30.0 - 82.3]mL/cm H₂O)显著改善。8例患有逼尿肌过度活动症的患者中有1例的逼尿肌过度活动消失。服用维贝格隆后排尿功能参数没有显著变化。

讨论

维贝格隆治疗显著改善了小儿OAB的临床和尿动力学参数,且无不良反应。关于儿童患者因先前药物治疗疗效不足或耐受性差而停药时换药的可行性,目前信息较少。维贝格隆可能是一种有前景的OAB治疗选择,在疗效和耐受性方面有更好的平衡。

结论

维贝格隆是患有特发性OAB的小儿患者改善主观症状和下尿路功能的替代药物。必须进行未来更大样本量的前瞻性随机研究以验证本研究结果。

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