Comparative Effectiveness of Biofeedback, Sacral Nerve Stimulation, and Injectable Bulking Agents for Treatment of Fecal Incontinence

生物反馈、骶神经刺激和注射填充剂治疗大便失禁的效果比较

• 批准号: 9092194
• 负责人: ADIL E. BHARUCHA
• 金额: $ 38.51万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-06-20 至 2018-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9092194
• 关键词: Address; Adherence; Admission activity; Adult; Adverse event; Advertisements; Advocate; Agreement; Anal canal; Anus; Authorship; Biofeedback; Characteristics; Clinic; Clinical; Clinical Data; Clinical Research; Clinical Trials Data Monitoring Committees; Colorectal Surgery; Consent Forms; Data; Data Collection; Data Coordinating Center; Data Quality; Development; Diabetes Mellitus; Diarrhea; Digestive System Disorders; Discipline of Nursing; Educational workshop; Electric Stimulation; Electronics; Equipment and Supplies; Fecal Incontinence; Feces; Female; Frequencies; Funding; Goals; Grant; Individual; Industry; Injectable; Injection of therapeutic agent; Institutes; Institutional Review Boards; International; Intervention; Kidney Diseases; Knowledge; Lead; Manuals; Measures; Mediation; Methods; Modality; Monitor; National Institute of Diabetes and Digestive and Kidney Diseases; North Carolina; Outcome; Outcome Study; Participant; Patients; Pelvic floor structure; Physiological; Physiology; Procedures; Process; Protocols documentation; Publications; Quality of life; Randomized; Randomized Controlled Trials; Recording of previous events; Recruitment Activity; Reporting; Research Personnel; Research Priority; Resistance; Sacral nerve; Safety; Sensory Thresholds; Severities; Site; Statistical Data Interpretation; Surveys; Symptoms; System; Technology; TimeLine; Training; Treatment Cost; Treatment Protocols; Universities; alternative treatment; base; comparative effectiveness; compare effectiveness; control trial; cost; cost effective; data management; design; diaries; follow-up; implantable device; inclusion criteria; incontinence symptom; male; meetings; neurophysiology; predict clinical outcome; predicting response; premature; pressure; public health relevance; quality assurance; rectal; response; secondary outcome; sphincter ani muscle structure; success; tocol; treatment choice; treatment response; trial comparing; trial design; volunteer; web portal; web site; willingness

 DESCRIPTION (provided by applicant): Fecal incontinence occurs at least weekly in 3% of U.S. adults and has a significant impact on quality of life. Biofeedback, sacral nerve electrical stimulation (SNS), and anal injection of dextranomer, an inert bulking agent, have been advocated, but absence of studies directly comparing them, and lack of understanding their pathophysiological basis are critical barriers to progress. Our central hypothesis is that biofeedback is more effective, safer, and less costly than dextranomer injection or SNS. We propose a two-step process to address this significant knowledge gap: Step 1 is a U34 planning grant to finalize treatment protocols and confirm access to sufficient numbers of eligible patients, while Step 2 is a U01 multicenter randomized controlled trial (RCT) designed to compare these three treatments using uniform inclusion criteria, design, and endpoints. Specific aims of U34 Planning Years 1 and 2: (1) Finalize procedure manuals. (2) Develop detailed plans for a study management website, electronic symptom diary, data quality checks, and statistical analysis. (3) Develop the safety monitoring plan; NIDDK will appoint Data Safety Monitoring Board. (4) Assess feasibility of recruitment strategy by surveys at all sites. (5) Negotiate agreements with industry for equipment, supplies, technical support. (6) Develop IRB applications and begin ClinicalTrials.gov registration. (7) Recruit, train, and certify biofeedback interventionists. (8) Finalize plans for project governance. Specific aims of U01: (1) Compare effectiveness, safety, and cost of these three treatments in 387 male and female patients with moderate to severe FI who have failed best-practice conservative treatment. The primary measure of efficacy will be >75% reduction in FI episodes compared to baseline. Safety will be measured by number of adverse events, and cost will be measured from both the personal and societal perspectives. Secondary endpoints include validated FI severity and quality of life scales. Outcomes will be assessed at 6, 12, and 24 months. (2) Identify baseline patient characteristics that predict response to each intervention. (3) Identify the mechanistic basis for treatment success for each intervention by comparing pre to post anal sphincter pressures, rectal sensory thresholds, rectal compliance, neurophysiological parameters, and stool consistency. (4) Evaluate treatment combinations. Non-responders by 6 months will be invited to receive one of the other two interventions as additional therapy. (5) Assess predictors of response to conservative treatment and durability of benefits. The study will be conducted by University of North Carolina, Mayo Clinic, Colon and Rectal Surgery Associates of Minneapolis, Georgia Regents University, and a data coordinating center (DCC) at RTI International in North Carolina. Expected outcomes include identifying the best treatment for moderate to severe fecal incontinence that is mechanistically based, durable, and safe. The impact of our project will include new understanding of the comparative effectiveness and safety of three commonly used treatment modalities, a scientific basis for each treatment, and identification of factors that predict clinical outcome.

 描述（由适用提供）：粪便失禁至少每周在3％的美国成年人中发生，并对生活质量产生重大影响。已经提倡生物反性，s骨神经电刺激（SNS）和右旋烷体的肛门注射（一种惰性散装剂），但缺乏直接比较它们的研究，并且缺乏了解其病理生理学基础是进步的关键障碍。我们的核心假设是，比葡萄糖剂注射或社交媒体更有效，安全，更加安全，更昂贵。我们提出了一个两步过程，以解决这一重要的知识差距：步骤1是U34计划赠款，以最终确定治疗方案并确认访问足够数量的合格患者，而步骤2是U01多中心随机对照试验（RCT），旨在比较使用统一的包含标准，设计和Endpoints和Endpoints进行这三种处理方法。 U34计划1和2：（1）最终确定程序手册的具体目标。 （2）为研究管理网站，电子症状日记，数据质量检查和统计分析制定详细计划。 （3）制定安全监控计划； NIDDK将任命数据安全监控委员会。 （4）通过所有站点的调查评估招聘策略的可行性。 （5）与行业的设备，供应和技术支持协商协议。 （6）开发IRB应用并开始临床。 （7）招募，培训和认证生物反馈 干预主义者。 （8）最终确定项目治理计划。 U01的具体目的：（1）比较387名中度至重度FI的男性和女性患者的这三种治疗方法的有效性，安全性和成本。与基线相比，FI发作的主要测量效率将降低75％。安全将通过不良事件的数量来衡量，并且成本将从个人和社会意见的角度衡量。次要终点包括经过验证的FI严重性和生活质量尺度。结果将在6、12和24个月进行评估。 （2）确定基线患者特征，以预测每种干预的反应。 （3）通过比较肛门后压力，直肠感觉阈值，直肠依从性，神经生理学参数和粪便一致性来确定每种干预措施的机械基础。 （4）评估治疗组合。到6个月的无反应者将邀请其他两种干预措施之一作为其他治疗。 （5）评估对保守治疗和福利持久性的反应预测指标。这项研究将由北卡罗来纳大学，梅奥诊所，结肠和直肠外科助理进行，乔治亚大学摄政大学以及北卡罗来纳州RTI International的数据协调中心（DCC）。预期的结果包括确定对中度至重度粪便尿失禁的最佳治疗方法，该治疗是基于机械的，耐用且安全的。我们项目的影响将包括对三种常用治疗方式的比较有效性和安全性的新理解，每种治疗的科学基础以及鉴定预测临床结果的因素。