De Smedt William, Jansen Katrien, Bogaert Guy
Faculty of Medicine, University of Leuven, Belgium.
Department of Pediatric Neurology, University Hospitals Leuven, Belgium.
J Pediatr Urol. 2025 Jun;21(3):661-669. doi: 10.1016/j.jpurol.2025.01.015. Epub 2025 Jan 28.
Intravesical oxybutynin as treatment for neurogenic bladder in children is promising, especially for patients in whom oral oxybutynin fails due to systemic anticholinergic side effects. However, little is known about the long-term outcomes of intravesical oxybutynin in children.
To assess, in a single-centre retrospective observational cohort study, the long-term urodynamic and renal outcomes of intravesical oxybutynin treatment for neurogenic bladder in children due to congenital spinal anomalies.
122 children who started intravesical oxybutynin before the age of 18 were included. The retrospective analysis focused on their urodynamics, X-ray cystography, DMSA (99 Tm-dimercaptosuccinic acid) scintigraphy, 51Cr-EDTA (51Cr-ethylene-diamine-tetra-acetic-acid) clearance, and need for augmentation cystoplasty. Urodynamic parameters during follow-up with intravesical oxybutynin were compared to baseline urodynamics prior to starting intravesical treatment, either without any anticholinergic medication or while on oral oxybutynin.
Mean intravesical oxybutynin treatment duration was 7.8 years (SD 5.9). Compared to baseline urodynamics without anticholinergic medication (n = 53), the proportion of patients with normal range of age-adjusted bladder capacity increased significantly from 36 % to 81 % (n = 47; p < 0.001). Mean end-filling pressure decreased significantly from 49.6 cmHO (SD 37.4) to 26.1 cmHO (SD 20.4; n = 27; p = 0.017). Detrusor overactivity presence (>30 cmHO) reduced significantly from 50 % to 18 % (n = 34; p = 0.007). Vesicoureteral reflux (VUR) presence dropped significantly from 45 % to 18 % (n = 99; p < 0.001). Compared to baseline urodynamics under oral oxybutynin (n = 62), a significant decrease in median end-filling pressure was the only notable difference found (Md = 24.5 cmHO; n = 36). During follow-up with intravesical oxybutynin, 21 % (n = 26 out of 122) reported pyelonephritic episodes. At long-term, 29 % (n = 22 out of 77) had asymmetric kidney function distribution with renal scarring and 16 % (n = 20 out of 122) underwent augmentation cystoplasty. 97 % (n = 86 out of 89) maintained a GFR above 60 ml/1.73 m/min. Longer treatment duration was a significant predictor for better age-adjusted bladder capacity, less detrusor overactivity and less VUR.
These long-term urodynamic and renal outcome data in a large study population demonstrate the efficacy of intravesical oxybutynin in paediatric patients with neurogenic bladder. The positive impact of intravesical oxybutynin on VUR is of clinical relevance since it is associated with upper urinary tract deterioration, eventually leading to renal function impairment. The association of longer intravesical treatment duration with improved urodynamic outcomes highlights the importance of continuous patient compliance in achieving favourable results.
Intravesical oxybutynin treatment is effective for managing neurogenic bladder in children, with good long-term urodynamic and renal outcomes.
膀胱内注射奥昔布宁治疗儿童神经源性膀胱颇具前景,尤其适用于因全身性抗胆碱能副作用而口服奥昔布宁无效的患者。然而,关于膀胱内注射奥昔布宁在儿童中的长期疗效知之甚少。
在一项单中心回顾性观察队列研究中,评估膀胱内注射奥昔布宁治疗先天性脊柱异常所致儿童神经源性膀胱的长期尿动力学和肾脏结局。
纳入122例18岁前开始膀胱内注射奥昔布宁的儿童。回顾性分析聚焦于他们的尿动力学、膀胱造影X线检查、二巯基丁二酸(DMSA)肾闪烁显像、51铬-乙二胺四乙酸(51Cr-EDTA)清除率以及膀胱扩大术的需求。将膀胱内注射奥昔布宁随访期间的尿动力学参数与开始膀胱内治疗前的基线尿动力学参数进行比较,基线时未使用任何抗胆碱能药物或正在口服奥昔布宁。
膀胱内注射奥昔布宁的平均治疗时长为7.8年(标准差5.9)。与未使用抗胆碱能药物的基线尿动力学检查结果(n = 53)相比,年龄校正膀胱容量在正常范围内的患者比例从36%显著增至81%(n = 47;p < 0.001)。平均终末充盈压从49.6 cmH₂O(标准差37.4)显著降至26.1 cmH₂O(标准差20.4;n = 27;p = 0.017)。逼尿肌过度活动(>30 cmH₂O)的发生率从50%显著降至18%(n = 34;p = 0.007)。膀胱输尿管反流(VUR)的发生率从45%显著降至18%(n = 99;p < 0.001)。与口服奥昔布宁时基线尿动力学检查结果(n = 62)相比,唯一显著差异是中位终末充盈压显著降低(中位数 = 24.5 cmH₂O;n = 36)。在膀胱内注射奥昔布宁随访期间,21%(122例中的26例)报告有肾盂肾炎发作。长期来看,29%(77例中的22例)肾功能分布不对称伴肾瘢痕形成,16%(122例中的20例)接受了膀胱扩大术。97%(89例中的86例)的肾小球滤过率(GFR)维持在60 ml/1.73 m²/min以上。治疗时间越长,年龄校正膀胱容量越大、逼尿肌过度活动越少以及VUR越少的显著预测指标。
在大量研究人群中得出的这些长期尿动力学和肾脏结局数据证明了膀胱内注射奥昔布宁对儿童神经源性膀胱患者的疗效。膀胱内注射奥昔布宁对VUR的积极影响具有临床意义,因为它与上尿路恶化相关,最终导致肾功能损害。膀胱内治疗时间越长与尿动力学结局改善之间的关联凸显了患者持续依从性对取得良好效果的重要性。
膀胱内注射奥昔布宁治疗儿童神经源性膀胱有效,具有良好的长期尿动力学和肾脏结局。