A population-based cohort to study outcomes in end-stage liver disease patients

基于人群的队列研究终末期肝病患者的结果

• 批准号: 8633209
• 负责人: David Seth Goldberg
• 金额: $ 15.51万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-09-14 至 2018-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8633209
• 关键词: Advisory Committees; Age; Algorithms; Area; Biometry; Caring; Case Mixes; Cessation of life; Clinical; Cohort Studies; Comorbidity; Complex; Data; Data Analyses; Data Set; Data Sources; Databases; Deterioration; Disease; Disease Outcome; Educational process of instructing; Environment; Epidemiology; Evaluation; Fostering; Future; Gender; Geographic Factor; Goals; Health Services; Hepatology; Hospitalization; ImProv; Improve Access; Intervention; Knowledge; Lead; Link; Liver; Liver diseases; Medicaid; Mentorship; Methods; Organ; Organ Transplantation; Outcome; Outcome Study; Outcomes Research; Patients; Pattern; Pennsylvania; Physicians; Policies; Population; Population Decreases; Public Health; Registries; Research; Research Personnel; Resources; Scientist; Severities; Specialist; Staging; Structure; System; Testing; Transplantation; United Network for Organ Sharing; Universities; Variant; Work; administrative database; adverse outcome; career; cohort; data sharing; density; geographic difference; improved; liver transplantation; low socioeconomic status; male; medical schools; medical specialties; mortality; neglect; novel; patient population; population based; prevent; public health relevance

Project Summary/Abstract Although liver transplantation is lifesaving for patients with end-stage liver disease (ESLD), the scarcity of cadaveric livers constrains its public health impact. Each year, approximately 6,000 patients undergo liver transplantation in the U.S., while roughly 2,000 are removed from the waitlist due to death or clinical deterioration.25 These 2,000 patients are a small fraction of the estimated 30,000 people that die each year from complications of ESLD26, suggesting the scarcity of livers may cause a potentially large number of ESLD patients to never be waitlisted. Indeed, the few available studies have estimated that less than 5% of potential transplant candidates are ever waitlisted.27,28 There are very limited data on factors that influence who among the population of patients with ESLD is listed for transplantation, and almost none of the data are population- based. Some factors, such as gender and cause of ESLD can be studied in local settings. However, the absence of nationally representative population-based data has prevented an in-depth evaluation of how systemic factors, such as geographic variations in supply and demand influence listing practices for ESLD patients. Thus, a population-based approach to studying ESLD is needed to bridge these unmet knowledge gaps. This proposal aims to establish such a population-based dataset of ESLD, and to use this new resource to test the central hypothesis that traditional research, focusing only on patients listed for liver transplantation, fails to capture the true impact of listing practices and allocation policies. The work could shift the paradigm by which future ESLD and transplant outcomes research is conducted to encompass population-level impacts of policies and practices. This work will: a) employ two novel algorithms to generate a national cohort of ESLD patients to be used for present and future ESLD outcomes research1,10~ b) identify potentially modifiable factors (e.g. listing practices) that can improve access to transplantation and outcomes among all potential transplant candidates~ and c) teach the applicant to use several new epidemiologic and demographic methods to foster his goal of become a leading physician-scientist focused on improving outcomes for ESLD patients. The specific aims are to: 1) identify patient and geographic factors associated with listing for transplantation among ESLD patients~ 2) evaluate the total impact of local mismatches between liver supply and demand~ and 3) examine how rates of listing for liver transplantation influence adverse patient outcomes among both waitlisted patients and all local ESLD patients. The research structure at the University of Pennsylvania and the Clinical Center for Epidemiology and Biostatistics, supplemented by the didactic coursework offered through the Perelman School of Medicine at the University of Pennsylvania offer the optimal environment within which the candidate will complete this work and foster his career as a clinician and independent investigator. Dr. Goldberg has assembled a mentorship team with expertise in epidemiologic, health services, and hepatology research, and an Advisory Committee with a longstanding track record of research and mentorship.

项目总结/摘要 虽然肝移植可以挽救终末期肝病（ESLD）患者的生命，但 尸体肝脏限制了其对公共卫生的影响。 每年约有6,000名患者接受肝脏移植。 移植在美国，而大约2,000人因死亡或临床原因从等待名单中删除 25这2,000名患者只是每年估计死亡的30,000人中的一小部分。 来自ESLD的并发症26，这表明肝脏的稀缺可能会导致潜在的大量ESLD 病人永远不会被列入名单。 事实上，现有的少数研究估计， 移植候选人永远被列入等候名单。27，28关于影响谁的因素的数据非常有限 ESLD患者人群被列为移植对象，几乎没有数据是人群- 基于.一些因素，如性别和ESLD的原因可以在当地环境中进行研究。 但 由于缺乏具有全国代表性的人口数据，无法深入评价 系统性因素，如供应和需求的地理差异，影响了ESLD的上市实践 患者因此，需要一种基于人群的方法来研究ESLD，以弥合这些未满足的知识 差距。 本建议旨在建立这样一个基于人口的ESLD数据集，并利用这一新资源 为了验证中心假设，传统的研究，只关注肝移植的患者， 未能反映上市做法和分配政策的真正影响。这项工作可以改变范式， 未来的ESLD和移植结果研究将涵盖以下人群水平的影响： 政策和做法。 这项工作将：a）采用两种新的算法来生成一个全国性的ESLD队列 用于当前和未来ESLD结局研究的患者1，10~ B）确定潜在的可改变因素 (e.g.列出的做法），可以改善获得移植和所有潜在的移植结果 c）教申请人使用几种新的流行病学和人口统计学方法来培养 他的目标是成为一名领先的医生科学家，专注于改善ESLD患者的预后。 的 具体目标是：1）确定与移植名单相关的患者和地理因素， ESLD患者~ 2）评估肝脏供需之间局部不匹配的总体影响~和3） 研究肝移植的登记率如何影响两个等待名单中的不良患者结局 患者和所有本地ESLD患者。宾夕法尼亚大学的研究结构和临床 流行病学和生物统计学中心，辅以通过提供的教学课程， 宾夕法尼亚大学佩雷尔曼医学院提供了最佳环境， 候选人将完成这项工作，并促进他的职业生涯作为一个临床医生和独立调查员。 博士 戈德堡组建了一个具有流行病学、卫生服务和肝病学专业知识的导师团队 研究，以及一个咨询委员会，具有长期的研究和指导记录。