Functional Outcomes in Pediatric 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结局。这项研究的主要目的是在几个重要层面上检查这些患者的功能结局。首先，将在一个大型队列中测量健康相关生活质量（HRQOL），该队列已通过儿科肝移植研究（SPLIT）医学结局研究进行前瞻性随访。SPLIT是一个由38个中心组成的行业资助的联盟，成立于1995年，旨在跟踪联合器官共享网络（UNOS）数据库未捕获的医疗结果。该数据库现在包括1308名接受首次肝移植的儿童。来自一个经过充分验证的，儿科仪器测量HRQOL在800这些儿童的反应将进行比较，以规范的人口。移植前后收集的医学变量和人口统计学信息将用于开发一个模型，该模型预测HRQOL，并可作为干预研究的基础，以改善移植后的HRQOL。其次，将在按年龄定义的队列的子集中进行智力、入学准备、学业成绩和行为的纵向研究。大约150名儿童将在5-6岁和两年后接受研究。成套测试将包括智商、阅读和算术技能、适应行为（日常生活技能）、组织能力和异常行为筛查。具体来说，本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将随着移植间隔的增加而改善，但不等于正常人群。认知发育同样会延迟，预计与移植时的年龄、移植时的病情和医疗并发症以及持续的医疗残疾相关。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，家庭和临床医生肝移植后儿童的功能期望。

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.