Fecal Incontinence Treatment Study (FIT Study)

大便失禁治疗研究（FIT 研究）

• 批准号: 10907221
• 负责人: ADIL E. BHARUCHA
• 金额: $ 137.72万
• 依托单位: MAYO CLINIC ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-18 至 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10907221
• 关键词: Admission activity; Adult; Adverse event; Agreement; Algorithms; Anus; Anxiety; Belief; Biofeedback; Characteristics; Clinic; Clinical; Clinical Research; Colorectal Surgery; Combined Modality Therapy; Common Terminology Criteria for Adverse Events; Cost Analysis; Cost Effectiveness Analysis; Data Coordinating Center; Diabetes Mellitus; Digestive System Disorders; Educational workshop; Electric Stimulation; Enrollment; Esthesia; Evoked Potentials; Expectancy; Fecal Incontinence; Feces; Frequencies; Geographic Locations; Hospitalization; Infection; Injections; Intervention; Kidney Diseases; Knowledge; Liquid substance; Magnetism; Measures; Medical; Medical Care Costs; Mental Depression; Methods; NIH Program Announcements; Neural Conduction; Nursing Homes; Outcome; Outcome Measure; Participant; Pathway interactions; Patients; Pelvic Pain; Pelvic floor structure; Persons; Physiological; Physiology; Policies; Pragmatic clinical trial; Prediction of Response to Therapy; Predictive Factor; Principal Investigator; Quality of life; Quality-Adjusted Life Years; Questionnaires; Randomized; Randomized, Controlled Trials; Recommendation; Recording of previous events; Rectum; Reporting; Research; Sacral nerve; Safety; Self Efficacy; Severities; Site; Solid; Sphincter; Telephone; Time; Universities; alternative treatment; cell motility; clinical research site; cohort; comparative cost effectiveness; comparative efficacy; compare effectiveness; cost; cost comparison; cost effectiveness; cost estimate; cost per quality-adjusted life year; design; efficacy evaluation; experience; follow-up; improved; inclusion criteria; neural stimulation; neurophysiology; predict responsiveness; premature; pressure; programs; psychological distress; psychological outcomes; psychosocial; recruit; rectal; response; safety assessment; secondary analysis; secondary outcome; sex; sphincter ani muscle structure; symposium; treatment comparison; treatment group; treatment responders; treatment response; treatment site; ultrasound; willingness

ABSTRACT This is an initial application for a U01 in response to PAR-15-067. This program announcement grew out of an NIDDK Workshop held in 2013 to identify pathways for promoting research on fecal incontinence (FI). Conference attendees identified gaps in knowledge and the best approaches to progress, and all identified Biofeedback (BIO), Sacral Neural Stimulation (SNS), and Injections of bulking agents (INJ) in the perianal space as treatments supported by controlled trials, but with no clear indication of which is most effective, safest, and least costly. Outcome measures have been variable, and inclusion criteria have also differed. The recommendation was to compare all 3 using the same paradigm. Aims: (1) Compare all 3 treatments on the basis of efficacy, safety, and cost, as well as secondary outcome measures. (2) Identify physiological, clinical, and psychosocial predictors of who will respond to each treatment. (3) Allow patients who do not respond to the randomly assigned treatment to select a second treatment at 3 months. (4) Compare treatment combinations to see which appears best (uncontrolled). (5) Assess the effects of enhanced Medical Management (MM) on efficacy, safety, and cost (uncontrolled). (6) Compare the cost-effectiveness of BIO, SNS, and INJ for FI throughout the two years. Methods: Select 567 patients appropriate to all treatments including >2 per week solid or liquid FI. A treatment responder is someone with >75% reduction in FI frequency. Primary assessment is 3 months after the beginning of randomized treatment. If patients are responders, they will continue to be studied for 24 months, but if they fail to respond they will be offered alternative treatment and followed. Secondary outcomes include the rate of responding with a 50% decrease in FI, continence, changes on the Bristol Stool Severity scale, FI Quality of Life, and validated severity scales (FI Severity Scale, St. Mark’s FI Severity Scale). Psychological outcomes include the PROMIS Anxiety-7, Depression-8, and Self-Efficacy scales. Costs effectiveness analysis will be based on Quality Adjusted Life Years. Moderators will include (1) physiological assessments (anal ultrasound, anorectal motility, and magnetic evoked potentials); (2) a priori beliefs in treatments (Credibility/Expectancy Questionnaire), quality of life (general and FI specific), sex, and type of FI (passive, urge). The 4 clinical site principal investigators (PIs) are Adil Bharucha (Mayo Clinic), Satish Rao (University of Augusta in Georgia), Ann Lowry (Colon and Rectal Surgery in Minneapolis), and William Whitehead (Chapel Hill, NC) – all senior PIs. The PI of the data coordinating center (DCC) is Marie Gantz, also an experienced PI. This is a collaborative agreement with NIDDK, and Frank Hamilton will participate in program management. A successful outcome will guide treatment algorithms and reimbursement policies.

抽象的 这是响应 PAR-15-067 的 U01 的初始应用。该计划的公告源于 NIDDK 研讨会于 2013 年举办，旨在确定促进大便失禁 (FI) 研究的途径。 与会者确定了知识差距和取得进展的最佳方法，并确定了所有 生物反馈 (BIO)、骶神经刺激 (SNS) 和肛周填充剂注射 (INJ) 空间作为受对照试验支持的治疗方法，但没有明确表明哪种方法最有效， 最安全，成本最低。结果衡量标准各不相同，纳入标准也各不相同。这 建议使用相同的范例来比较所有 3 个。目的： (1) 比较所有 3 种治疗方法 功效、安全性和成本以及次要结果指标的基础。 (2) 识别生理、临床、 以及谁会对每种治疗产生反应的社会心理预测因素。 (3) 让没有反应的患者 随机分配的治疗选择在 3 个月时进行第二次治疗。 (4) 比较治疗 组合以查看哪种效果最好（不受控制）。 (5) 评估强化医疗的效果 关于功效、安全性和成本（不受控制）的管理 (MM)。 (6)比较BIO的成本效益， FI的SNS和INJ贯穿了两年。方法：选择适合所有治疗的 567 名患者 包括每周 >2 次固体或液体 FI。治疗有反应者是指 FI 降低 > 75% 的人 频率。初步评估是在随机治疗开始后 3 个月进行。如果患者是 回应者，他们将继续接受24个月的研究，但如果他们未能回应，他们将被提供 替代治疗并遵循。次要结果包括响应率下降 50% FI、失禁、布里斯托尔大便严重程度量表的变化、FI 生活质量和经过验证的严重程度量表 (FI 严重程度、圣马可 FI 严重程度）。心理结果包括 PROMIS Anxiety-7、 抑郁 8 级和自我效能量表。成本效益分析将基于调整生活质量 年。主持人将包括 (1) 生理评估（肛门超声、肛门直肠蠕动和磁力评估） 诱发电位）； (2) 对治疗的先验信念（可信度/期望问卷）、生活质量 （一般和 FI 特定）、性别和 FI 类型（被动、冲动）。 4 名临床中心主要研究者 (PI) 是 Adil Bharucha（梅奥诊所）、Satish Rao（乔治亚州奥古斯塔大学）、Ann Lowry（结肠和直肠） 明尼阿波利斯外科）和 William Whitehead（北卡罗来纳州教堂山）——都是高级 PI。数据的PI 协调中心（DCC）是 Marie Gantz，她也是一位经验丰富的 PI。这是一份合作协议 NIDDK 和 Frank Hamilton 将参与项目管理。成功的结果将指导 治疗算法和报销政策。