PDF(Pubmed)
Abstract：
Onabotulinum Toxin-A (BTX-A) is a second-line treatment for neurogenic bladder (NB). It requires repeated injections over time, which is a possible limit for long-term adherence, especially in children, as general anesthesia is required. Almost 50% of adults discontinue therapy; few data on pediatric patients are present. The aim of this study is to share our long-term experience of BTX-A adherence in children. This study is a retrospective review of 230 refractory NB patients treated with BTX-A. The inclusion criteria were ≥3 treatments and the first injection performed ≥10 years before the study endpoint. Fifty-four patients were included. Mean follow-up was 10.2 years; mean treatment number was 6.4 for each patient. During follow-up, 7% did not need BTX-A anymore; 76% discontinued therapy, with a prevalence of acquired NB (64% acquired vs. 34% congenital; p = 0.03); sex-based and urodynamic findings did not influence the discontinuation rate (p = 0.6, p = 0.2, respectively). Considering those who withdrew from the therapy, 43% were lost to follow-up/died after a mean of 7.5 years (although 33% still experienced clinical efficacy); 33% changed therapy after a mean of 5.8 years (with reduced efficacy in 22%, persistent efficacy in 11%). BTX-A is a safe and effective therapy for pediatric patients. The treatment abandonment rate is higher for children than for adults; no specific reasons were highlighted. It is necessary to evaluate any age-specific factors to explain these data.
摘要：
Onabotulinum毒素A（BTX-A）是神经源性膀胱（NB）的二线治疗方法。它需要随着时间的推移重复注射，这是长期坚持的可能限制，尤其是在儿童中，因为需要全身麻醉。几乎50％的成年人停止治疗；关于儿科患者的数据很少。这项研究的目的是分享我们对儿童BTX-A依从性的长期经验。本研究是对230例用BTX-A治疗的难治性NB患者的回顾性分析。纳入标准为≥3次治疗，首次注射在研究终点前≥10年。包括54名患者。平均随访时间为10.2年；每位患者的平均治疗次数为6.4。随访期间,7%的人不再需要BTX-A；76%的人停止治疗，获得性NB的患病率(64%获得性与34％先天性；p=0.03）；基于性别和尿动力学的发现不影响停药率（分别为p=0.6，p=0.2）。考虑到那些退出治疗的人，43％的患者失去随访/平均7.5年后死亡（尽管33％仍然有临床疗效）；33％的患者在平均5.8年后改变了治疗（22％的疗效降低，11%的持续疗效)。BTX-A是儿科患者安全有效的治疗方法。儿童的治疗放弃率高于成人；没有强调具体原因。有必要评估任何特定年龄的因素来解释这些数据。
