Peard Leslie M, Pope John C, Dmochowski Roger
Division of Pediatric Urology, Monroe Carell Jr. Children's Hospital at Vanderbilt, Nashville, TN, USA.
Department of Urology, Vanderbilt University Medical Center, Nashville, TN, USA.
Expert Rev Neurother. 2023 Jul-Dec;23(9):763-774. doi: 10.1080/14737175.2023.2246661. Epub 2023 Aug 15.
Neurogenic detrusor overactivity (NDO) results in involuntary detrusor contractions during bladder filling or storage risking transmission of pressure to the upper urinary tracts and/or significant incontinence. The goals of bladder management in children with NDO prioritize the preservation of renal function, prevention of UTIs, and optimizing quality of life. First-line measures include intermittent catheterization and anticholinergic medication. However, when conservative measures fail, surgical intervention may be indicated. Historically, the next step was major reconstructive surgery to create a low-pressure urinary reservoir. The introduction of intravesical botulinum neurotoxin A (BoNT/A) for use in children in 2002 offered a less invasive option for management. However, its exact role is still evolving.
This article summarizes the mechanism of action of BoNT/A for management of NDO and evaluates the current literature defining common practice and clinical efficacy in children with NDO. The findings of the recently completed phase III trial for intravesical onabotulinumtoxinA in children are discussed in detail.
As the first BoNT/A approved for use in children with NDO, onabotulinumtoxinA appears to be a safe and less invasive alternative to major reconstructive surgery. However, data defining appropriate patient selection and its role as a long-term treatment option continue to develop.
神经源性逼尿肌过度活动（NDO）会导致膀胱充盈或储尿期间逼尿肌不自主收缩，存在将压力传导至上尿路和/或导致严重尿失禁的风险。NDO患儿膀胱管理的目标优先考虑保护肾功能、预防尿路感染以及优化生活质量。一线措施包括间歇性导尿和抗胆碱能药物治疗。然而，当保守措施失败时，可能需要进行手术干预。从历史上看，下一步是进行大型重建手术以创建一个低压储尿器。2002年膀胱内注射肉毒杆菌神经毒素A（BoNT/A）用于儿童，为治疗提供了一种侵入性较小的选择。然而，其确切作用仍在不断演变。
本文总结了BoNT/A治疗NDO的作用机制，并评估了现有文献中关于NDO患儿的常见治疗方法及临床疗效。详细讨论了最近完成的膀胱内注射A型肉毒毒素治疗儿童的III期试验结果。
作为首个被批准用于NDO患儿的BoNT/A，A型肉毒毒素似乎是大型重建手术的一种安全且侵入性较小的替代方法。然而，关于合适的患者选择及其作为长期治疗选择的作用的数据仍在不断发展。
