Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.
Yale New Haven Children's Hospital, New Haven, Connecticut, USA.
Neurourol Urodyn. 2021 Jan;40(1):493-501. doi: 10.1002/nau.24588. Epub 2020 Dec 11.
This study evaluated whether one (or more) of three doses of onabotulinumtoxinA were safe and effective to treat neurogenic detrusor overactivity (NDO) in children.
This was a 48-week prospective, multicenter, randomized, double-blind study in children (aged 5-17 years) with NDO and urinary incontinence (UI) receiving one onabotulinumtoxinA treatment (50, 100, or 200 U; not to exceed 6 U/kg). Primary endpoint: change from baseline in daytime UI episodes. Secondary endpoints: change from baseline in urine volume at first morning catheterization, urodynamic measures, and positive response on the treatment benefit scale. Safety was also assessed.
There was a similar reduction in urinary incontinence from baseline to Week 6 for all doses (-1.3 episodes/day). Most patients reported positive responses on the treatment benefit scale (75.0%-80.5%). From baseline to Week 6, increases were observed in urine volume at first morning clean intermittent catheterization (50 U, 21.9 ml; 100 U, 34.9 ml; 200 U, 87.5 ml; p = 0.0055, 200 U vs. 50 U) and in maximum cystometric capacity (range 48.6-63.6 ml) and decreases in maximum detrusor pressure during the storage phase (50 U, -12.9; 100 U, -20.1; 200 U, -27.3 cmH O; p = 0.0157, 200 U vs. 50 U). The proportion of patients experiencing involuntary detrusor contractions dropped from baseline (50 U, 94.4%; 100 U, 88.1%; 200 U, 92.6%) to Week 6 (50 U, 61.8%; 100 U, 44.7%; 200 U, 46.4%). Safety was similar across doses; urinary tract infection was most frequent.
OnabotulinumtoxinA was well tolerated and effective for the treatment of NDO in children; 200 U showed greater efficacy in reducing bladder pressure and increasing bladder capacity.
本研究旨在评估单剂量（或多剂量）肉毒毒素 A 治疗儿童神经源性逼尿肌过度活动（NDO）的安全性和有效性。
这是一项为期 48 周的前瞻性、多中心、随机、双盲研究，纳入了患有 NDO 和尿失禁（UI）的儿童（年龄 5-17 岁），他们接受了一种肉毒毒素 A 治疗（50、100 或 200 U；不超过 6 U/kg）。主要终点：与基线相比，日间 UI 发作次数的变化。次要终点：首次晨尿导尿时尿量、尿动力学测量以及治疗益处量表上的阳性反应的变化。同时评估了安全性。
所有剂量组从基线到第 6 周的尿失禁均有类似的减少（-1.3 次/天）。大多数患者对治疗益处量表报告了积极的反应（75.0%-80.5%）。从基线到第 6 周，首次晨尿清洁间歇导尿时的尿量（50 U 增加 21.9 ml；100 U 增加 34.9 ml；200 U 增加 87.5 ml；p=0.0055，200 U 与 50 U 相比）和最大膀胱容量（范围 48.6-63.6 ml）增加，而储尿期最大逼尿肌压力降低（50 U 降低 12.9 cmH₂O；100 U 降低 20.1 cmH₂O；200 U 降低 27.3 cmH₂O；p=0.0157，200 U 与 50 U 相比）。出现不自主逼尿肌收缩的患者比例从基线时（50 U 为 94.4%；100 U 为 88.1%；200 U 为 92.6%）下降到第 6 周（50 U 为 61.8%；100 U 为 44.7%；200 U 为 46.4%）。各剂量组的安全性相似；尿路感染最常见。
肉毒毒素 A 治疗儿童 NDO 耐受性良好且有效；200 U 减少膀胱压力和增加膀胱容量的效果更显著。
