Functional Outcomes in Pediatric Liver Transplantation

小儿肝移植的功能结果

• 批准号: 6867134
• 负责人: Estella M. Alonso
• 金额: $ 53.79万
• 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/6867134
• 关键词: academic achievement; adolescence (12-20); behavioral /social science research tag; child (0-11); clinical research; developmental disease /disorder; functional ability; human subject; intelligence; liver transplantation; longitudinal human study; outcomes research; patient oriented research; psychometrics; quality of life

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。拟议的研究的主要目标是检查这些患者的功能结果。首先，与健康相关的生活质量（HRQOL）将在大型队列中进行测量，该研究通过小儿肝移植（分裂）医疗结果研究进行了前瞻性遵循。 Split是一个由行业资助的财团组成的，成立于1995年，旨在遵循联合器官共享网络（UNOS）数据库未捕获的医疗结果。该数据库现在包括1308名接受过第一次肝移植的儿童。将这些儿童中有800名测量HRQOL的小儿仪器的反应与规范人群进行比较。移植前后收集的医疗变量和人口统计信息将用于开发一个模型，该模型可以预测HRQOL，并可以作为干预研究的基础，以改善移植后的HRQOL。其次，将在智力能力，学校准备，学术成就和行为的纵向研究中进行，以年龄定义的队列的子集。大约有150名儿童将在5-6岁和两年后进行研究。测试电池将包括智力商，阅读和算术技能的度量，适应性行为（日常生活技能），组织技能以及对异常行为的筛查。具体而言，这项研究将检验以下假设：肝移植后儿童的功能结果比正常儿童低。预计HRQOL会随着移植的间隔而改善，但不会等于规范人群。认知发展也将被延迟，并有望与移植时的年龄，疾病的敏锐度和医疗并发症在移植和持续的医疗残疾相关。这项多中心协作研究将得到具有成果和生活质量研究和发展评估的专业知识的顾问的支持。预计这项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，家庭和临床医生肝移植后儿童的功能期望。