肉毒杆菌毒素 A（肉毒素）：治疗儿童和青年成年轻度神经原性膀胱功能障碍的合理选择。
Onabotulinumtoxin A (Botox): A reasonable alternative for refractory neurogenic bladder dysfunction in children and young adults.
机构信息
Department of Urology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Division of Urologic Surgery, Beth Israel Deaconess Medical Center, Boston, USA.
出版信息
Neurourol Urodyn. 2021 Nov;40(8):1981-1988. doi: 10.1002/nau.24778. Epub 2021 Sep 6.
AIMS
We aimed to describe the effectiveness of Onabotulinumtoxin A (Botox) in children with neurogenic bladder (NGB) unresponsive to medical therapy to determine urodynamic parameters predictive of success.
METHODS
Children receiving Botox for refractory NGB, between 2008 and 2019, from a single academic center, were included in this study. Botox success was defined as improvement of incontinence and/or urodynamic parameters.
RESULTS
Of 34 patients who received Botox, 13 (38.2%) had a positive response from their first injection, with improvement in capacity by a median of 35% of expected capacity for age compared to only a 9% increase in those who did not respond clinically. When patients were divided into groups by baseline urodynamic parameters, high-pressure (Pdetmax > 20 cm H O) patients had significantly greater improvement in compliance compared with low-pressure patients (p = 0.017). Low compliance patients (<10 ml/cm H O) had a dramatic improvement of 3.08 ml/cm H O in their compliance compared with minimal change in the high compliance group (p = 0.003). Finally, low-capacity (<50% of expected CC) patients had significant improvement in capacity and compliance when compared with high-capacity patients (p = 0.004 and p = 0.036, respectively). Improvement in detrusor overactivity (DO) was noted in both the clinical responders and non-responders.
CONCLUSION
In our series, 38% had clinical success with intradetrusor Botox injections for refractory neurogenic bladder. When successful, improvement in capacity and compliance, DO, and/or incontinence was consistent with prior literature. While we could not determine which parameters predicted success, subdividing patients into categories based on baseline urodynamic parameters identified who would benefit from Botox treatment based on differential improvements in capacity and compliance. At least 1 injection of Botox should be considered for a subset of children with refractory NGB, before undertaking more invasive treatments.
目的
我们旨在描述在对药物治疗无反应的神经源性膀胱（NGB）患儿中使用肉毒毒素 A（Botox）的疗效，以确定预测成功的尿动力学参数。
方法
本研究纳入了 2008 年至 2019 年期间在单一学术中心接受 Botox 治疗难治性 NGB 的患儿。Botox 治疗成功的定义为改善失禁和/或尿动力学参数。
结果
34 例接受 Botox 治疗的患儿中，有 13 例（38.2%）首次注射后有阳性反应，与无临床反应的患儿相比，容量平均增加了预期年龄的 35%，而仅增加了 9%。当根据基线尿动力学参数将患者分组时，高压（Pdetmax>20cmH2O）患者的顺应性改善明显大于低压患者（p=0.017）。低顺应性患者（<10ml/cmH2O）的顺应性有显著改善，增加了 3.08ml/cmH2O，而高顺应性组变化很小（p=0.003）。最后，与高容量患者相比，低容量（<50%预期 CC）患者的容量和顺应性有显著改善（分别为 p=0.004 和 p=0.036）。在临床反应者和无反应者中均观察到逼尿肌过度活动（DO）改善。
结论
在我们的系列研究中，38%接受逼尿肌内 Botox 注射治疗难治性神经源性膀胱的患儿有临床疗效。当成功时，容量和顺应性、DO 和/或失禁的改善与既往文献一致。虽然我们无法确定哪些参数预测成功，但根据基线尿动力学参数将患者分为不同类别，确定了哪些患者将受益于 Botox 治疗，因为容量和顺应性的改善程度不同。在进行更具侵袭性的治疗之前，至少应考虑对一部分难治性 NGB 患儿进行 Botox 注射治疗。
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