Clinical Center: Childhood Liver Disease Research and Education Network (ChiLDREN

临床中心：儿童肝病研究和教育网络（ChiLDREN

• 批准号: 8119739
• 负责人: PETER Frank WHITINGTON
• 金额: $ 35万
• 依托单位: LURIE CHILDREN'S HOSPITAL OF CHICAGO
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-15 至 2014-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8119739
• 关键词: Accounting; Alagille Syndrome; Bile fluid; Biliary Atresia; Child; Childhood; Cholestasis; Chronic; Clinical; Clinical Research; Defect; Disease; Disease Progression; Education; Elements; Failure; Functional disorder; Genetic; Goals; Human Resources; Individual; Infant; Injury; Kinetics; Liver; Liver diseases; Morbidity - disease rate; Operative Surgical Procedures; Participant; Performance; Plant Roots; Procedures; Progressive intrahepatic cholestasis; Public Health; Research; Resources; Response Elements; Staging; Techniques; Therapeutic; Time; Tissues; Transcriptional Regulation; Transplantation; Treatment Effectiveness; United States; Work; bile salts; design; end stage disease; expectation; infancy; insight; liver transplantation; meetings; mortality; novel; operation; patient population; research study; response; stable isotope; success

DESCRIPTION (provided by applicant): Liver disease is a major cause of infant and childhood morbidity and mortality. The diseases comprising "pediatric liver diseases" are individually rare, which has hindered the study of their causes/ pathophysiologies. As a result of this basic defect in understanding effective therapeutic strategies are lacking for most of them. This in turn results in many children with them progressing to end-stage liver disease necessitating orthotopic liver transplantation. Pediatric liver transplants comprise approximately 10% of all liver transplants performed, and the indications for most of them lie among the diseases to be studied in the Childhood Liver Disease Research and Education Network (ChiLDREN), the major objective of which is to "combine the expertise and resources of the [Biliary Atresia Research Consortium] BARC and [Cholestatic Liver Consortium] CLiC clinical centers ... to study pediatric liver diseases". An essential element of this objective is the combined expertise of the participants, which serves to guide the study of the individual diseases included. A major objective of this application is to participate in ChiLDREN as an expert in genetically determined cholestasis (in particular progressive familial intrahepatic cholestasis) and in clinical aspects of infantile cholestatic liver disease in general. The other element of the major objective of ChiLDREN is the resources to be contributed by the clinical centers, the most important being the subjects with liver disease. Our center has been a leading contributor of subjects to both BARC and CLiC and expects to continue as such in ChiLDREN. By participating in these ways we expect to contribute substantially to the performance of ChiLDREN in achieving its goal of successfully eliminating pediatric liver disease as a major cause of infant and childhood morbidity and mortality. The specific aims at our center include: a) to participate fully as a clinical center in ChiLDREN; and b) to contribute to the understanding of the pathophysiology of progressive familial intrahepatic cholestasis by designing and implementing studies to be carried out by the membership of ChiLDREN. Relevance: Chronic pediatric liver disease, although rare, is a devastating condition that has high public health impact. Children with biliary atresia or any of the five genetic cholestatic liver diseases studied by ChiLDREN account for the majority of pediatric liver transplantations performed in the United States.

描述（由申请人提供）： 肝病是婴儿和儿童发病率和死亡率的主要原因。包括“小儿肝病”的疾病是个别罕见的，这阻碍了其原因/病理生理学的研究。由于理解大多数有效的治疗策略的基本缺陷，因此缺乏这种缺陷。反过来，这导致许多患有他们的儿童发展到需要原位肝移植的终末期肝病。 Pediatric liver transplants comprise approximately 10% of all liver transplants performed, and the indications for most of them lie among the diseases to be studied in the Childhood Liver Disease Research and Education Network (ChiLDREN), the major objective of which is to "combine the expertise and resources of the [Biliary Atresia Research Consortium] BARC and [Cholestatic Liver Consortium] CLiC clinical centers ... to study pediatric liver疾病”。该目标的一个基本要素是参与者的综合专业知识，该专业知识是指导所包括的个体疾病的研究。该应用的一个主要目的是作为遗传确定的胆汁淤积（尤其是进行性家族性肝内胆汁淤积）和婴儿胆汁淤积性肝脏疾病的临床方面参与儿童。儿童主要目标的另一个要素是由临床中心贡献的资源，最重要的是患有肝病的受试者。我们的中心一直是BARC和CLIC受试者的主要贡献者，并希望在儿童中继续这样做。通过参与这些方式，我们期望为儿童的表现做出基本贡献，以成功地消除儿科肝病作为婴儿和儿童发病率和死亡率的主要原因。针对我们中心的具体目标包括：a）充分参加儿童临床中心； b）通过设计和实施儿童的成员资格进行研究和实施研究，从而有助于理解进行性家族性肝内胆汁淤积的病理生理学。 相关性：慢性小儿肝病虽然很少见，但却是一种具有较高公共卫生影响的毁灭性疾病。患有胆道闭锁的儿童或儿童研究的五种遗传胆汁淤积性肝疾病中的儿童是美国进行的大多数儿科肝移植。