Uçar Murat, Akgül Ahsen Karagözlü, Parlak Ayşe, Yücel Cem, Kılıç Nizamettin, Balkan Emin
Section of Pediatric Urology, Department of Urology, Health Sciences University, Tepecik Education and Research Hospital, 35180, İzmir, Turkey.
Section of Pediatric Urology, Department of Pediatric Surgery, Health Sciences University, Van Education and Research Hospital, Van, Turkey.
Int Urol Nephrol. 2018 Aug;50(8):1367-1373. doi: 10.1007/s11255-018-1926-6. Epub 2018 Jul 2.
We aimed to evaluate the efficacy and safety of intravesical onabotulinum toxin A (onaBoNTA) injections for the treatment of children diagnosed with refractory overactive bladder (OAB) by using non-invasive methods.
A total of 31 pediatric patients with a mean age of 10.2 years received intravesical onaBoNTA injection at the dose of 10 U/kg (max: 200 U). Twenty-one patients who failed to respond to the first injection, received second injection 6 months after the first one. The patients were retrospectively evaluated after the 1st and the 2nd injections by means of standardized questionnaire forms and voiding diary records.
In the 6-month follow-up, 10 patients (32.2%) were determined to have full response after the first injection. The number of patients with partial response and no response were found to be 15 (48.4%) and 6 (19.4%), respectively. The mean value of Dysfunctional Voiding and Incontinence Symptom Score (DVISS) of the patients with full response was 8.5 before the injection, which decreased to 1 at 6-month follow-up and to 0.5 at 12-month follow-up (p < 0.001). Twenty-one patients without full response after the first injection received a second injection 6 months after the first one. Full response was achieved in 9 (42.9%) of the 21 patients who had a second injection, but only partial response was achieved in 9 (42.9%). Three (14.3%) of the remaining patients did not respond to the second injection either. 1-year follow-up evaluations revealed that the rates of the full response, partial response, and no response were 61.3, 29, and 9.7%, respectively.
Based on our results, onaBoNTA therapy is an effective and reliable second-line off-label therapy in the management of patients with non-neurogenic OAB that is refractory to medical therapy. Asking the patients/guardians to fill out a standardized questionnaire form before and after the therapy enables easy and non-invasive assessment of the response to the therapy.
我们旨在通过非侵入性方法评估膀胱内注射A型肉毒杆菌毒素（onaBoNTA）治疗难治性膀胱过度活动症（OAB）患儿的疗效和安全性。
共有31例平均年龄为10.2岁的儿科患者接受了剂量为10 U/kg（最大剂量：200 U）的膀胱内onaBoNTA注射。21例首次注射无反应的患者在首次注射6个月后接受了第二次注射。通过标准化问卷形式和排尿日记记录对患者在第一次和第二次注射后进行回顾性评估。
在6个月的随访中，10例患者（32.2%）在首次注射后被确定为完全缓解。部分缓解和无反应的患者人数分别为15例（48.4%）和6例（19.4%）。完全缓解患者的排尿功能障碍和尿失禁症状评分（DVISS）平均值在注射前为8.5，在6个月随访时降至1，在12个月随访时降至0.5（p < 0.001）。21例首次注射后未完全缓解的患者在首次注射6个月后接受了第二次注射。21例接受第二次注射的患者中有9例（42.9%）实现了完全缓解，但也有9例（42.9%）仅为部分缓解。其余3例（14.3%）患者对第二次注射也无反应。1年的随访评估显示，完全缓解、部分缓解和无反应的发生率分别为61.3%、29%和9.7%。
基于我们的研究结果，onaBoNTA治疗是治疗药物难治性非神经源性OAB患者的一种有效且可靠的二线非标签治疗方法。要求患者/监护人在治疗前后填写标准化问卷形式，能够轻松且非侵入性地评估治疗反应。
