Factors Associated with Therapeutic Efficacy of Intravesical OnabotulinumtoxinA Injection for Overactive Bladder Syndrome

ObjectivesTo analyze the predictors of therapeutic efficacy after intravesical botulinum toxin A injection for overactive bladder syndrome (OAB) refractory to antimuscarinic therapy.MethodsAll consecutively OAB patients, who visited the urologic outpatient clinics of a medical center and refractory to antimuscarinic treatment, were prospectively enrolled. All enrolled patients received intravesical injection of 100 U onabotulinumtoxinA (Botox). The Global Response Assessment (GRA) score ≥ 2 at 3 months after Botox injection was defined as a successful treatment, otherwise failed.ResultsOverall, 89 patients received intravesical injection. Eighty patients, including 42 men and 38 women, had received follow-up at 3 months. The overall success rate was 63.8%. The global response assessment, urgency severity score, urgency, urgency urinary incontinence and frequency episodes, and functional bladder capacity improved after treatment. However, post-void residual volume (PVR) increased, and voiding efficiency (VE) decreased after treatment. Female gender (odds ratio = 3.75) was the only independent factor associated with the success. Female gender (coefficient = 0.74), low baseline overactive bladder symptoms score (coefficient = -0.12) and the presence of OAB-wet (coefficient = 0.79) were independent factors associated with therapeutic efficacy (i.e., GRA score). VE (odds ratio = 0.062) was the only predictor for a large PVR at 3 months. The optimum cutoff value of VE was ConclusionsThe therapeutic effects of Botox can persist till 6 months after treatment. Female gender, low overactive bladder symptoms score and OAB-wet are associated better therapeutic efficacy, and low baseline VE is associated with large PVR. These findings can serve as an initial guide or assist in consultation regarding the treatment of OAB patients with Botox injection.Trial RegistrationClinicalTrials.gov NCT01657409

**研究目的**：分析经抗胆碱能治疗难治的膀胱过度活动症（overactive bladder syndrome, OAB）患者接受膀胱内A型肉毒毒素注射后的治疗疗效预测因素。**研究方法**：本研究前瞻性纳入某医学中心泌尿外科门诊就诊的、经抗胆碱能治疗无效的连续性OAB患者。所有入组患者均接受膀胱内注射100U的注射用A型肉毒毒素（onabotulinumtoxinA，商品名Botox）。以注射后3个月时的整体应答评估（Global Response Assessment, GRA）评分≥2作为治疗成功的判定标准，反之则判定为治疗失败。**研究结果**：本研究共纳入89例接受膀胱内注射的患者，其中80例（男42例、女38例）完成3个月随访。总体治疗成功率为63.8%。治疗后，患者的整体应答评估评分、尿急严重程度评分、尿急症状、急迫性尿失禁发作频次及功能性膀胱容量均得到显著改善。但治疗后患者的排尿后残余尿量（post-void residual volume, PVR）升高，排尿效率（voiding efficiency, VE）下降。女性性别（优势比=3.75）是与治疗成功相关的唯一独立影响因素。女性性别（系数=0.74）、较低的基线膀胱过度活动症症状评分（系数=-0.12）以及合并OAB-wet（急迫性尿失禁型膀胱过度活动症，系数=0.79）是与治疗疗效（即GRA评分）相关的独立影响因素。排尿效率（VE，优势比=0.062）是3个月时出现高残余尿量的唯一预测因素。原文此处疑似缺失排尿效率最优截断值的相关描述。**研究结论**：A型肉毒毒素的治疗疗效可维持至治疗后6个月。女性性别、较低的基线膀胱过度活动症症状评分以及OAB-wet表型与更佳的治疗疗效相关，而基线低排尿效率与3个月时的高残余尿量相关。上述研究结果可为OAB患者接受A型肉毒毒素注射治疗的临床诊疗提供初步参考依据，或有助于相关诊疗咨询的开展。**试验注册**：ClinicalTrials.gov NCT01657409