Predicting urinary continence status with sacral neuromodulation and botulinum toxin treatments

通过骶神经调节和肉毒杆菌毒素治疗预测尿失禁状态

基本信息

批准号：
10457243
负责人：
James Arthur Hokanson
金额：
$ 13.72万
依托单位：
MEDICAL COLLEGE OF WISCONSIN
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-09-01 至 2024-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10457243
关键词：
Address Adult Adverse event Aftercare Animals Anti-Cholinergics Benign Bladder Bladder Control Botox Botulinum Toxins Catheters Characteristics Clinical Clinical Trials Counseling Data Diagnostic Functional disorder Goals Gold Health Care Costs Healthcare Systems Human Individual Knowledge Lead Learning Literature Machine Learning Measurement Medical History Mentors Methods Modeling Modernization Multi-Institutional Clinical Trial National Institute of Diabetes and Digestive and Kidney Diseases Nocturia Overactive Bladder Patient Care Patients Pattern Performance Pharmaceutical Preparations Phenotype Physicians Population Probability Process Questionnaires Refractory Regimen Research Residual volume Resistance Sampling Selection for Treatments Symptoms Testing Therapeutic Human Experimentation Training Treatment outcome Urge Incontinence Urinary Incontinence Urinary Retention Urinary tract infection Urodynamics Urology Woman alpha Toxin base comparative efficacy cost design diagnostic tool effective therapy expectation experience feature selection improved individual patient infection risk innovation lower urinary tract symptoms mathematical model micturition urgency neuroregulation novel novel therapeutics patient response personalized medicine predictive modeling primary outcome programs response shared decision making signal processing success treatment response trial comparing

项目摘要

PROJECT SUMMARY Lower Urinary Tract Symptoms (LUTS) include urinary urgency, incontinence, and nocturia, afflict roughly 50% of the adult population, and have substantial, wide-ranging impact. Many gaps remain in our understanding of these symptoms, leading to inadequate treatment. Urgency urinary incontinence, one form of LUTS, was the focus of two recent multi-site clinical trials (Anticholinergic vs. Botox Comparison Study or ABC and Refractory Overactive Bladder: Sacral NEuromodulation vs. BoTulinum Toxin Assessment or ROSETTA). These trials compared the efficacy of urgency urinary incontinence therapies, 100 Units (U) Onabotulinum Toxin A (Botox) versus an anticholinergic medication (ABC) and 200U Botox versus sacral neuromodulation (SNM)(ROSETTA). These studies compared whether on average 100U Botox was superior to a standard anticholinergic regimen (ABC) and whether 200U Botox was superior to SNM (ROSETTA). The objective of this K01 proposal is to use the same clinical trial data to create predictive models that accurately estimate the likelihood that a patient will respond to Botox and SNM treatments, as well as the likelihood of developing urinary retention or urinary tract infections (risks associated with Botox treatment). In addition to medical histories and questionnaires some of the predictive models will incorporate diagnostic measurements, both standard and novel, from tests of bladder function (urodynamics). This approach will allow for the formal assessment of the utility of urodynamics in guiding treatment decisions, something that is currently debated in the field. This project will result in innovative models which can be used to: 1) predict whether a patient will respond to Botox and SNM treatments and the likelihood of adverse events (Botox only); 2) determine the utility of urodynamics data in predicting patient response to treatment and 3) identify patients that are resistant to treatment, so as to guide future research. These models will offer a patient specific, data-driven approach to treatment that will greatly benefit patient care and help reduce costs to the health care system. This project will also provide training in predictive modeling and clinical benign urology under the guidance of an experienced mentoring team. The candidate’s long-term objective is to lead a strong independent research program to design new therapies for patients with (LUTS) informed by both animal and human studies, and to match patients with suitable therapies based on individual patient characteristics (personalized medicine).

项目摘要较低的尿路症状（LUTS）包括尿液紧急，尿失禁和夜尿，大约50％成年人口，并具有重大的广泛影响。我们对这些符号，导致治疗不足。紧急尿失禁是一种形式的LUTS，是两个最近的多站点临床试验的重点（抗胆碱能与肉毒杆菌毒素比较研究或ABC和难治性过度膀胱：s骨神经调节与肉毒杆菌毒素评估或rosetta）。这些试验比较了紧迫性尿失禁疗法，100个单位（U）Onabotulinum毒素A（肉毒杆菌毒素）与抗胆碱能药物（ABC）和200U肉毒杆菌毒素与s骨神经调节（SNM）（Rosetta）。这些研究比较100U肉毒杆菌毒素是否优于标准抗胆碱能方案（ABC）和 200U肉毒杆菌毒素是否优于SNM（Rosetta）。该K01提案的目的是使用相同临床试验数据以创建预测模型，以准确估计患者应对的可能性肉毒杆菌毒素和SNM治疗，以及发展尿位retention留或尿路感染的可能性（与肉毒杆菌治疗有关的风险）。除了病史和问卷外预测模型将从膀胱测试中纳入标准和新颖的诊断测量值功能（尿动力学）。这种方法将允许对尿动力学效用的正式评估指导治疗决策，目前在该领域进行了辩论。该项目将产生创新的模型，可用于：1）预测患者是否会回应肉毒杆菌和SNM治疗以及不良事件的可能性（仅肉毒杆菌毒素）； 2）确定效用预测患者对治疗的反应时的尿动力学数据和3）确定对患者的抗性患者治疗，以指导未来的研究。这些模型将为患者提供特定于患者的数据驱动方法将极大地受益于患者护理并帮助降低医疗保健系统的成本。该项目还将在预测建模和临床良性泌尿外科方面提供培训。一支经验丰富的心理团队。候选人的长期目标是领导一项强大的独立研究针对动物和人类研究告知的患者（LUTS）设计新疗法的计划，以及根据个体患者特征（个性化医学）将患者与适当的疗法相匹配。