Optimizing Measures for Clinical Trials in Covert Hepatic Encephalopathy

隐性肝性脑病临床试验的优化措施

基本信息

批准号：
10132309
负责人：
Elliot Tapper
金额：
$ 19.4万
依托单位：
UNIVERSITY OF MICHIGAN AT ANN ARBOR
依托单位国家：
美国
项目类别：
财政年份：
2019
资助国家：
美国
起止时间：
2019-07-01 至 2023-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10132309
关键词：
Abdominal Muscles Address Adrenergic beta-Antagonists Adverse event Albumins American Ammonia Area Ascites Bilirubin Blood Brain Calibration Calories Catabolic Process Cessation of life Chronic Cirrhosis Clinical Clinical Trials Clinical Trials Design Code Coma Complication Computers Confusion Data Data Analyses Development Diagnosis Diet Dietary Intervention Disorientation Electronic Mail Enrollment Executive Dysfunction Exercise Exposure to Fall prevention Feasibility Studies Focus Groups Goals Gold Grant Hand Hemorrhage Hepatic Encephalopathy Hospitalization Inpatients Intervention Interview Investigation Knowledge Lethargies Malnutrition Master&apos s Degree Measures Mentorship Methodology Methods Modeling Muscle Muscle function Nutritional Online Systems Outcome Outpatients Pathway interactions Patient Outcomes Assessments Patient Selection Patients Personal Satisfaction Pharmaceutical Preparations Physical Function Portal Hypertension Positioning Attribute Prospective cohort Proteins Publishing Randomized Controlled Trials Research Research Personnel Retrospective cohort Risk Risk Factors Severities Structure Talents Testing Text Messaging Time Training Training Programs Voice Work abdominal CT arm authority base career chronic liver disease clinical investigation clinical predictors cognitive change cognitive function cohort cost daily functioning disability executive function exercise intervention falls grasp health care service utilization health related quality of life high risk improved inattention insight modifiable risk mortality neurocognitive test neuropsychiatry novel nutrition patient oriented point of care predictive modeling prevent prospective randomized trial resistance exercise response risk prediction risk stratification sarcopenia success therapeutic target therapy design tool vehicular accident wasting

项目摘要

Optimizing Measures for Clinical Trials in Covert Hepatic Encephalopathy PI: Elliot B. Tapper, MD Among all complications of cirrhosis, none is more devastating than hepatic encephalopathy (HE). Although it will afflict up to 40% of patients with cirrhosis, HE has few dedicated researchers. A spectrum of neuropsychiatric disability, HE ranges in severity from covert (deficits in executive function) to overt (confusion, even coma). Even covert HE – the earliest stage – is independently associated with repeated hospitalization, falls, motor vehicle accidents and death. Unfortunately, strategies to prevent the development HE are limited. Furthermore, investigations of potential therapies are complicated by limited knowledge of both the clinical predictors of HE and impact on patient well-being and daily functions. This grant proposes to close these gaps. The training program aims to prepare the applicant for a career vested in identifying risk factors for HE, and in designing interventions to prevent HE and to improve patient well-being. A master's degree in clinical investigation with targeted additional courses will provide training in clinical trial design, calibration of patient reported measures for clinical trials, and longitudinal data analysis to develop clinical predictive models. The mentorship team assembled is diverse and talented, including Dr. Anna Lok (a clinical trialist and content expert), Dr. Jasmohan Bajaj (an authority on HE and clinical trials), Dr. Noelle Carlozzi (a neuropsychologist with expertise in mixed-methodology), and Dr. Susan Murphy (a trialist with a focus on interventions for frail patients). In research aim 1, we will determine the most important domains of health related quality of life (HRQOL) for patients with covert HE. In Aim 2, we will analyze a cohort of 300 cirrhosis patients without HE followed for up to 4 years to determine the modifiable risk factors for HE and to develop predictive models for HE and falls. In Aim 3, we will launch a pilot intervention aimed at the improvement of HRQOL and prevention of falls in patients with covert HE using a web-based platform that co-ordinates a combination of text-message, email and interactive-voice response to deliver structured encouragement to meet nutritional and exercise goals aimed at improving sarcopenia. By the conclusion of the K23, the applicant will be positioned for academic success with a niche in an important complication of cirrhosis and providing necessary preliminary data for an R01 supporting a larger, randomized trial.

优化秘密肝性脑病临床试验的措施 PI：Elliot B. Tapper，医学博士在肝硬化的所有并发症中，没有一个比肝病（He）更具毁灭性的。虽然是他的肝硬化患者中最多会折磨40％的患者，他很少有专门的研究人员。一部分神经精神上的残疾，他的严重程度从掩盖（执行功能的缺陷）到明显（混乱，甚至昏迷）。即使是最早的阶段 - 甚至秘密 - 与反复住院相关，瀑布，汽车事故和死亡。不幸的是，防止他受到限制的发展策略。此外，对潜在疗法的研究因临床的有限了解而变得复杂 HE的预测因素以及对患者福祉和日常功能的影响。这项赠款提案要缩小这些差距。该培训计划旨在为申请人做好准备，以归属于确定HE的风险因素的职业设计干预措施以防止他并改善患者福祉。临床硕士学位针对有针对性的其他课程的调查将提供临床试验设计，校准患者的培训报道了用于临床试验的测量和纵向数据分析以开发临床预测模型。 Menorship Team组装成多样而有才华，包括Anna Lok博士（临床试验者和内容专家），Jasmohan Bajaj博士（HE和临床试验的机构），Noelle Carlozzi博士（神经心理学家具有混合方法学专业知识）和苏珊·墨菲（Susan Murphy）患者）。在研究目标1中，我们将确定与健康相关的生活质量的最重要领域（HRQOL）患有覆盖的患者。在AIM 2中，我们将分析300名没有HE的肝硬化患者的队列紧随其后的是4年，以确定HE的可修改风险因素，并为他和跌倒。在AIM 3中，我们将启动旨在改善HRQOL和预防的飞行员干预措施他使用基于网络的平台的患者的跌倒患者，该平台协调了文本消息的组合，电子邮件和交互式声音响应，以提供结构化的鼓励以满足营养和锻炼旨在改善肌肉减少症的目标。根据K23的结论，申请人将被定位为在肝硬化的重要并发症中，利基市场的学术成就，并提供必要的初步 R01支持更大的随机试验的数据。