Predicting post-transplant mortality and global functional health based on pre-transplant functional status in liver transplantation

基于肝移植中移植前功能状态预测移植后死亡率和整体功能健康

• 批准号: 10221569
• 负责人: Jennifer C. Lai
• 金额: $ 66.72万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-06-15 至 2023-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10221569
• 关键词: Acute; Address; Ascites; Caregivers; Cessation of life; Characteristics; Cirrhosis; Clinical; Collaborations; Data; Decision Making; Elderly; Ensure; Extrahepatic; Future; Geriatrics; Goals; Habilitation; Hand Strength; Health; Hemorrhage; Hepatic; Icterus; Infrastructure; Intercept; Investigation; Knowledge; Liver; Liver diseases; Longitudinal Studies; Malnutrition; Measures; Modeling; Muscular Atrophy; Outcome; Outpatients; Patient Care; Patients; Performance; Peritonitis; Physical Function; Portal Hypertension; Preparation; Quality of life; Recovery of Function; Research; Risk; Standardization; Testing; Time; Transplant Recipients; Transplantation; Vulnerable Populations; Waiting Lists; Work; adverse outcome; balance testing; base; clinical decision-making; disability; experience; frailty; functional disability; functional status; health related quality of life; impaired functional status; improved; indexing; instrument; liver transplantation; mortality; mortality risk; post-transplant; prognostic model; shared decision making; stressor; tool; transplant centers

PROJECT SUMMARY The decision to proceed with liver transplantation in a patient with end-stage liver disease depends not just on the risk of death without transplant but the risk of adverse outcomes after it. The transplant clinician's assess- ment of a cirrhotic patient's global functional health – which we have conceptualized as his or her vulnerability to health stressors – is a critical factor (oftentimes the sole factor) in this decision. Yet at the current time, no standardized, objective criteria for poor global functional health exist to define who is “too frail for transplant”. Rather, assessment of functional status in transplant is subjective and is applied to decision-making ad hoc, resulting in unequal transplant access and potential denial of otherwise suitable candidates. To facilitate transplant decision-making, a precise understanding of how pre-transplant functional status impacts post- transplant outcomes is needed to inform prediction of who will not regain excellent global functional health after transplant. We have demonstrated that tools to quantify frailty and functional status in older adults have proven valuable to measure global functional health in cirrhotic patients and have developed an objective Liver Frailty Index, consisting of a composite of performance-based tests (grip strength, chairs stands, and balance testing), to capture longitudinal changes in functional status specifically for use in the pre- and post-transplant settings. Building logically upon this work, we propose to determine the impact of pre-transplant functional status on 1-year post-transplant mortality and global functional health and develop/validate clinical prediction rules for these outcomes that incorporate pre-transplant functional status. To accomplish these goals, we will leverage our existing Multi-center Functional Assessment in Liver Transplantation Study, consisting of 5 US liver transplant centers (UCSF, Johns Hopkins, Columbia, Baylor, and Duke) with a track record of collaboration and high-impact research to obtain data on a minimum of 1,300 liver transplant recipients with assessments of functional status pre-transplantation and assessments of global functional health (including the Liver Frailty Index, disability, and quality of life) 1-year post-transplantation. These data will be used to develop and validate clinical prediction rules that incorporate both pre-transplant functional status, patient and donor characteristics to predict death, functional status, disability, and quality of life 1-year after transplantation. This project will positively impact the field by expanding our ability to measure the benefit of transplant both by how long a recipient will live as well as by how well a recipient will live after liver transplantation. Importantly, this project will facilitate clinical decision-making for patients and their clinicians through the precise understanding of how functional status impacts outcomes and what patients can expect after liver transplantation with respect to functional recovery. Given that functional status is modifiable in cirrhotic patients, our data will also support future investigations to develop effective strategies to improve pre-transplant functional status with the goal of reducing mortality and optimizing post-transplant functional health.

项目摘要 终末期肝病患者进行肝移植的决定不仅取决于 死亡的风险无需移植，但后期不良后果的风险。移植临床评估 - 肝硬化患者的全球功能健康 - 我们已将其概念化为他或她的脆弱性 对于健康压力源而言，这是该决定中的关键因素（通常是唯一的因素）。然而目前，没有 对于全球功能健康差的标准化，客观标准，存在定义谁“太虚弱，无法进行移植”。 相反，对移植中功能状态的评估是主观的，并应用于决策临时， 导致不平等的移植访问和对其他合适候选者的潜在拒绝。促进 移植决策，对移植前功能状况如何影响的精确理解 - 需要移植结果来告知谁不会在 移植。我们已经证明，已证明了量化老年人脆弱和功能状况的工具已证明 值得衡量肝硬化患者全球功能健康的有价值的东西，并发展了客观的肝脏脆弱 索引，由基于性能的测试组成（握力强度，椅子和平衡 测试），捕获专门用于移植前后功能状态的纵向变化 设置。在这项工作的逻辑上，我们建议确定移植前功能的影响 转移后死亡率和全球功能健康和发展/验证临床预测的1年状态 这些结果的规则包含了移植前功能状态。为了实现这些目标，我们将 利用我们现有的多中心功能评估在肝移植研究中，由5 US组成 肝移植中心（UCSF，Johns Hopkins，Columbia，Baylor和Duke）的往绩记录 协作和高影响力研究以获取至少1300个肝移植接受者的数据 评估功能状态前移植和全球功能健康评估的评估（包括 肝脏脆弱指数，残疾和生活质量）转移后1年。这些数据将用于开发 并验证临床预测规则，该规则既包含移植前功能状态，患者和捐助者 预测移植后1年死亡，功能状况，残疾和生活质量的特征。这 项目将通过扩大我们衡量移植的能力的能力来对该领域产生积极影响 长期以来，接收者将生活在肝脏移植后的接收者生活状况。重要的是，这个 项目将通过确切的理解来促进患者及其临床医生的临床决策 功能状态如何影响结局以及患者在肝移植后与尊重的期望 进行功能恢复。鉴于肝硬化患者的功能状态是可修改的，我们的数据也将支持 未来投资以制定有效策略以改善移植前功能状况的目标 降低死亡率并优化移植后功能健康。