Functional Outcomes in Pediatric Liver Transplantation

小儿肝移植的功能结果

• 批准号: 7212223
• 负责人: Estella M. Alonso
• 金额: $ 44.26万
• 依托单位: LURIE CHILDREN'S HOSPITAL OF CHICAGO
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-04-01 至 2010-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7212223
• 关键词: Academic achievement; Acute Liver Failure; Adaptive Behaviors; Address; Adult; Affect; Age; Age-Years; Behavior; Biliary Atresia; Child; Childhood; Clinical; Cognitive; Collaborations; Contracts; Daily; Data; Data Collection; Data Element; Data Quality; Databases; Development; Developmental Delay Disorders; Ensure; Family; Foundations; Funding; Goals; Growth; Industry; Intelligence quotient; Intervention; Intervention Studies; Investigation; Life; Link; Living Wills; Long-Term Survivors; Longitudinal Studies; Measures; Medical; Modeling; Morbidity - disease rate; Neurologic; North America; Numbers; Operative Surgical Procedures; Outcome; Outcome Study; Outcomes Research; Patients; Performance; Population; Population Growth; Prevalence; Provider; Psychometrics; Quality of life; Range; Rate; Readiness; Reading; Registries; Renal function; Research; Risk Factors; Schools; Score; Screening procedure; System; Testing; Time; Transplant Recipients; Transplantation; United Network for Organ Sharing; cognitive function; cohort; data management; demographics; disability; expectation; follow-up; functional outcomes; health related quality of life; improved; instrument; liver transplantation; medical complication; response; size; skills; social

DESCRIPTION (provided by the applicant): Our broad, long-term goals are to improve outcomes for children who undergo liver transplantation. The primary objectives of the proposed study are to examine functional outcome of these patients on several important levels. First, health-related quality of life (HRQOL) will be measured in a large cohort that has been followed prospectively through the Studies of Pediatric Liver Transplantation (SPLIT) medical outcomes study. SPLIT is an industry-funded consortium of 38 centers established in 1995 to follow medical outcomes not captured by the United Network for Organ Sharing (UNOS) database. The database now includes 1308 children who have received their first liver transplant. Responses from a well-validated, pediatric instrument measuring HRQOL in 800 of these children will be compared to a normative population. Medical variables and demographic information collected before and following transplantation will be used to develop a model, which predicts HRQOL and which could serve as the foundation for intervention studies to improve HRQOL after transplant. Second, a longitudinal study of intellectual ability, school readiness, academic achievement and behavior will be conducted in a sub-set of the cohort defined by age. Approximately, 150 children will be studied at 5-6 years of age and two years later. Testing batteries will include measures of intellectual quotient, reading and arithmetic skills, adaptive behavior (daily-living skills), organizational skills, and screening for abnormal behavior. Specifically, this study will test the hypothesis that functional outcome is lower for children after liver transplant than for normal children. It is expected that HRQOL will improve with interval from transplant, but will not be equal to a normative population. Cognitive development will likewise be delayed and is expected to correlate with age at transplant, acuity of illness and medical complications at the time of transplant and ongoing medical disability. This multi-center collaborative study will be supported by consultants with expertise in outcomes and quality of life research and developmental assessment. It is expected that information gained in this study will allow clinicians to identify interventions that optimize outcomes and better inform patients, families and clinicians about functional expectations for children after liver transplantation.

描述（由申请人提供）：我们广泛的长期目标是改善接受肝移植的儿童的预后。拟议研究的主要目标是在几个重要层面上检查这些患者的功能结果。首先，将在一个大型队列中测量与健康相关的生活质量 (HRQOL)，并通过儿科肝移植研究 (SPLIT) 医疗结果研究对这些队列进行前瞻性跟踪。 SPLIT 是一个由 38 个中心组成的行业资助联盟，成立于 1995 年，旨在跟踪联合器官共享网络 (UNOS) 数据库未记录的医疗结果。该数据库目前包含 1308 名接受第一次肝移植的儿童。经充分验证的儿科仪器测量 800 名儿童的 HRQOL，其反应将与正常人群进行比较。移植前后收集的医学变量和人口统计信息将用于开发一个模型，该模型可预测 HRQOL，并可作为改善移植后 HRQOL 的干预研究的基础。其次，将在按年龄定义的队列子集中对智力、入学准备、学业成绩和行为进行纵向研究。大约 150 名 5-6 岁和两年后的儿童将接受研究。测试电池将包括智商、阅读和算术技能、适应行为（日常生活技能）、组织技能以及异常行为筛查的测量。具体来说，这项研究将检验肝移植后儿童的功能结果低于正常儿童的假设。预计 HRQOL 将随着移植间隔时间的延长而改善，但不会等于正常人群。认知发育同样会被延迟，并且预计与移植时的年龄、疾病的严重程度和移植时的医疗并发症以及持续的医疗残疾有关。这项多中心合作研究将得到在结果和生活质量研究和发展评估方面具有专业知识的顾问的支持。预计本研究中获得的信息将使临床医生能够确定优化结果的干预措施，并更好地告知患者、家属和临床医生对肝移植后儿童的功能期望。