Cleveland Clinic Lerner College of Medicine, Case Western Reserve University , Cleveland, OH , USA.
Curr Med Res Opin. 2014 Mar;30(3):509-26. doi: 10.1185/03007995.2013.860021. Epub 2013 Nov 25.
There is currently a lack of formal guidance for assessing treatment response and non-response in patients with overactive bladder (OAB). Such guidance would be useful for both clinical practice and the design of clinical trials. Our purpose was to review and assess definitions of treatment response and non-response used in patients with OAB.
We conducted a systematic review of articles published between January 1, 2005 and August 8, 2013 using PubMed. Search terms included (overactive bladder) AND ('treatment response' OR responder OR success OR satisfied OR goal OR refractory OR nonresponder OR fail OR persistent OR dissatisfied). Limits were 'humans' and 'English'. Studies conducted in subjects with neurogenic detrusor overactivity, conditions other than OAB, or OAB symptoms following lower urinary tract/pelvic surgery were excluded; case reports and letters were also excluded.
The literature search returned 423 articles, of which 75 met the inclusion criteria and defined a specific threshold by which treatment response or non-response was determined for patients receiving behavioral therapy and/or treatment with an antimuscarinic, β3-agonist, botulinum toxin, or neural stimulation. One published abstract from congress proceedings and three additional articles that were not identified by the search were included; thus, a total of 79 records were included. A wide variety of symptom-based definitions and patient-reported outcomes (PROs) were used. Symptom-based definitions frequently used a threshold of 50-100% improvement in general or specific symptoms; urgency urinary incontinence (UUI) was often used in studies with incontinent patients. Definitions based on PROs frequently used measures of satisfaction, general improvement, or goal achievement. Studies of patients with refractory OAB often referred to a failure to respond to ≥1 other therapy, or to poor efficacy or unacceptable tolerability, without further specification. Limitations of this review are that only English language articles were included and that only the PubMed database was used for the literature search.
There is considerable heterogeneity in the definitions of treatment response and non-response in trials of patients with OAB; some standardization would be beneficial. However, there is also heterogeneity among patients of what constitutes treatment success or failure, and conceptualizations of treatment response and non-response in both clinical trials and clinical practice must take patient characteristics into account. For patients with UUI, it is recommended that the criteria for treatment response include this symptom, as measured by change in the absolute number of UUI episodes or achievement of continence, given its impact on patients' lives and associated bother. PROs provide important information that confirm symptom-based measures by demonstrating that observed changes in symptoms are meaningful to the patient. In clinical practice, measures of treatment satisfaction and goal achievement can be highly useful.
目前，对于评估膀胱过度活动症（OAB）患者的治疗反应和无反应，缺乏正式的指导。这种指导对于临床实践和临床试验的设计都将是有用的。我们的目的是回顾和评估用于 OAB 患者的治疗反应和无反应的定义。
我们使用 PubMed 进行了一项 2005 年 1 月 1 日至 2013 年 8 月 8 日期间发表的文章的系统综述。搜索词包括（膀胱过度活动症）和（“治疗反应”或应答者或成功或满意或目标或难治性或无应答者或失败或持续或不满意）。限制条件为“人类”和“英语”。排除了在神经源性逼尿肌过度活动、OAB 以外的疾病或下尿路/盆腔手术后出现 OAB 症状的受试者进行的研究；病例报告和信件也被排除在外。
文献检索共返回 423 篇文章，其中 75 篇符合纳入标准，并为接受行为治疗和/或抗毒蕈碱、β3-激动剂、肉毒杆菌毒素或神经刺激治疗的患者确定了特定的反应或无反应阈值。一篇来自会议论文的已发表摘要和另外三篇未通过搜索确定的文章也被纳入；因此，共纳入 79 份记录。使用了各种基于症状的定义和患者报告的结果（PROs）。基于症状的定义通常使用一般或特定症状改善 50-100%的阈值；在有尿失禁患者的研究中，常使用急迫性尿失禁（UUI）。基于 PRO 的定义通常使用满意度、总体改善或目标达成的衡量标准。对于难治性 OAB 患者的研究，通常是指对≥1 种其他治疗方法无反应，或疗效不佳或不能耐受，而没有进一步的具体说明。本综述的局限性在于，仅纳入了英语文章，并且仅使用 PubMed 数据库进行文献检索。
在 OAB 患者的临床试验中，治疗反应和无反应的定义存在很大的异质性；一些标准化将是有益的。然而，对于哪些构成治疗成功或失败，以及临床试验和临床实践中治疗反应和无反应的概念化，患者之间也存在异质性，必须考虑到患者的特征。对于有 UUI 的患者，建议将该症状的治疗反应标准包括在内，如通过 UUI 发作次数的绝对减少或达到无尿失禁来衡量，因为它对患者的生活和相关困扰有影响。PROs 提供了重要的信息，通过证明观察到的症状变化对患者有意义，从而证实了基于症状的措施。在临床实践中，治疗满意度和目标达成的衡量标准非常有用。
