Childhood Liver Disease Research Network (ChiLDReN) Clinical Centers

儿童肝病研究网络 (ChiLDReN) 临床中心

• 批准号: 10197884
• 负责人: Estella M. Alonso
• 金额: $ 37.63万
• 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 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10197884
• 关键词: Adult; Alagille Syndrome; Ammonia; Antibiotics; Area; Automobile Driving; Bacterial DNA; Bacterial Translocation; Bile Acid Biosynthesis Pathway; Biliary Atresia; Biological; Blood - brain barrier anatomy; Child; Childhood; Cholestasis; Chronic; Cirrhosis; Clinical Data; Clinical Research; Clinical Trials; Cross-Over Trials; Defect; Disease; Disease Progression; Double-Blind Method; Employment; Enrollment; Etiology; Failure; Fatigue; Functional disorder; Gene Expression; Genetic; Goals; Growth; Growth and Development function; Hepatic Encephalopathy; Immunology; Impairment; Individual; Infant; Inflammatory; Insulin-Like Growth Factor I; Interleukin-6; Investigation; Lactulose; Lead; Leadership; Link; Liver; Liver diseases; Measurement; Measures; Mitochondria; Morbidity - disease rate; Natural History; Neurocognitive; Oral; Pathogenesis; Patient Outcomes Assessments; Patients; Pattern; Pediatrics; Performance; Permeability; Pharmaceutical Preparations; Pilot Projects; Placebos; Plant Roots; Plasma; Population; Portal Hypertension; Process; Progressive intrahepatic cholestasis; Public Health; Randomized; Reporting; Research; Research Personnel; Risk; Role; Safety; Sampling; School-Age Population; Schools; Secondary to; Serum; Site; Somatotropin; Specimen; Testing; United States; alpha 1-Antitrypsin Deficiency; biobank; burden of illness; clinical center; cohort; cytokine; design; driving skills; effective therapy; efficacy testing; epigenomics; expectation; experience; feasibility trial; frailty; health related quality of life; improved; inflammatory marker; liver cystic fibrosis; liver transplantation; meetings; member; mortality; neonatal hepatitis; neurocognitive test; neurotoxic; pediatric patients; prevent; primary sclerosing cholangitis; recruit; reduce symptoms; research study; response; rifaximin; sarcopenia; simulation; systemic inflammatory response; therapeutically effective; trial design

Project Summary/Abstract 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 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 Network (ChiLDReN). This network combines the efforts of several large and individually successful clinical research enterprises to recruit subjects and carry them through rigorous clinical studies and trials with the expectation of establishing well-characterized patient cohorts that can be followed through the natural history of their disease process and which can be accessed for trials of emerging therapies. In addition, the biological specimens linked to clinical data provide the fuel for studies of etiology (genetic and other) and the influences of gene expression and epigenomics on disease expression and progression, as well as response to therapy. We propose to participate in ChiLDReN as a center wherein investigators have substantial expertise in several of the key areas of investigation within the consortium as a whole. Our center has been one of the top contributors of subjects to studies undertaken by ChiLDReN over the term of its existence. We expect to continue 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 leading clinical center in ChiLDReN; b) To pilot a double-blind, randomized, placebo controlled, cross-over trial testing the efficacy of rifaximin therapy to reduce symptoms of minimal hepatic encephalopathy (MHE) and improve Health Related Quality of Life (HRQOL) in school aged patients in the ChiLDReN network with portal hypertension (PHTN); c) To characterize the impact of systemic inflammatory markers and measures of bacterial translocation on both PROs and the growth hormone axis in school age children with and without PHTN.

项目概要/摘要 肝病是婴儿和儿童发病和死亡的主要原因。 包括“小儿肝病”在内的疾病个别罕见，这阻碍了对其的研究 由于理解有效治疗的基本缺陷。 他们中的大多数人都缺乏策略，这反过来导致许多孩子进展到了。 需要进行原位肝移植的终末期肝病。 约占所有肝移植手术的 10%，并且大多数肝移植手术的适应症 它们属于儿童肝病研究网络要研究的疾病之一 （ChiLDReN）。这个网络结合了几个大的和单独成功的努力。 临床研究企业招募受试者并进行严格的临床研究 以及旨在建立特征良好的患者队列的试验，这些患者队列可以 跟踪他们疾病过程的自然史，并且可以访问 此外，与临床数据相关的生物样本提供了新疗法的试验。 病因学（遗传和其他）研究以及基因表达和影响的燃料 表观基因组学研究疾病的表达和进展，以及对治疗的反应。 提议作为中心参与ChiLDReN 研究人员拥有丰富的专业知识 我们中心在整个联盟内的几个关键领域进行了调查。 是ChiLDReN在本学期进行的研究中科目的最大贡献者之一 我们期望继续为该公司的业绩做出重大贡献。 ChiLDReN 实现了成功消除小儿肝病作为主要疾病的目标 我们中心的具体目标包括：a) 作为领先的 ChiLDReN 临床中心全面参与；b) 试点双盲、 随机、安慰剂对照、交叉试验测试利福昔明治疗的疗效 减轻轻微肝性脑病 (MHE) 的症状并改善健康相关 ChiLDReN 网络中学龄患者的生活质量 (HRQOL) 门户网站 高血压 (PHTN)；c) 表征全身炎症标志物的影响和 学龄期 PRO 和生长激素轴上细菌易位的测量 患有和不患有 PHTN 的儿童。