Continuation of the Childhood Liver Disease Research Network Seattle Clinical Cen

儿童肝病研究网络西雅图临床中心的延续

• 批准号: 8910693
• 负责人: KAREN F MURRAY
• 金额: $ 27.19万
• 依托单位: SEATTLE CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-10 至 2019-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8910693
• 关键词: Affect; Alagille Syndrome; Alaska; Alaska Native; American; American Indians; Biliary Atresia; Biological; Blood Platelet Disorders; Blood Platelets; Caring; Catchment Area; Child; Child Care; Childhood; Clinical; Collection; Cystic Fibrosis; Data; Databases; Development; Disease; Enrollment; Family; Fibrosis; Functional disorder; Funding; Future; Genetic; Hawaii; Health; Hemorrhage; Hepatology; Idaho; Individual; Investigation; Knowledge; Liver; Liver diseases; Logic; Medical; Metabolic Diseases; Montana; Morbidity - disease rate; Natural History; Operative Surgical Procedures; Oregon; Pacific Northwest; Pathogenesis; Patient Care; Patients; Pediatric Hospitals; Pediatrics; Phenotype; Population; Positioning Attribute; Productivity; Progressive intrahepatic cholestasis; Protein C Inhibitor; Questionnaires; Recruitment Activity; Relative (related person); Research; Risk; Role; Specimen; Syndrome; Techniques; Testing; Thrombelastography; Time; United States; Washington; Wyoming; alpha 1-Antitrypsin Deficiency; base; chronic liver disease; experience; hypercholesterolemia; improved; liver transplantation; mortality; novel; pediatric patients; programs; research study

DESCRIPTION (provided by applicant): Biliary atresia and the other childhood cholestatic liver diseases are significant causes of chronic liver disease in children, and the leading causes for liver transplantation in pediatrics. The initial funding periods for the proceeding consortiums leading to Childhood Liver Disease Research Network (ChiLDReN) have resulted in unprecedented collections of well phenotyped subjects and banked data and biological specimens. Although ongoing recruitment of subjects with these rare conditions is needed to allow full attainment of many of the individual study Aims, the collection of subjects, data, and biospecimens is now sufficient to support meaningful investigation into the pathogenesis of these diseases and inform the investigation of novel therapies. This next funding period will continue and expand the currently ongoing research of natural history, genetics, and treatment, by continuing to completion the ongoing studies. Furthermore, new therapies will be trialed in the hopes of improving the care that can be provided to children afflicted with these diseases. Usage of the subject data, biospecimens, and engaged subject populations and their families for advancement of knowledge of these diseases and treatment of affected children will also be a priority for this coming funding period. The Seattle Clinical Center has the experience, expertise, and proven track record to continue participation in ChiLDReN, and has the expected patients over time to support the new consortium trials. Dr. Murray and her Clinical Center team has also proposed a study of bleeding risk and platelet function in Alagille syndrome subjects in comparison to subjects with alpha-1-antitrypsin deficiency and progressive familial intrahepatic cholestasis, that will fill a gap in our knowledge about this condition and significantly impact children with Alagille. This study will use a Pediatric Bleeding Questionnaire to assess bleeding risk in these children, and then analyze their platelet function utilizing the Platelet Functional Analyzer-100 and thromboelastography. The results from analysis of these ChiLDReN subjects will be evaluated in conjunction with collected consortium data on these subjects to verify that children with Alagille syndrome have an increased bleeding risk unique to their syndrome and that this is in part due to platelet dysfunction.

描述（由申请人提供）：胆道闭锁和其他儿童胆汁淤积性肝病是儿童慢性肝病的重要原因，也是儿科肝移植的主要原因。上述联营集团的初始供资期 导致儿童肝病研究网络（ChiLDReN）已经导致前所未有的良好表型受试者和库存数据和生物标本的收集。尽管需要持续招募患有这些罕见疾病的受试者以完全实现许多个体研究目的，但目前收集的受试者、数据和生物标本足以支持对这些疾病的发病机制进行有意义的研究，并为研究新疗法提供信息。下一个资助期将继续并扩大目前正在进行的自然史，遗传学和治疗研究，继续完成正在进行的研究。此外，将试验新的治疗方法，希望能改善为患有这些疾病的儿童提供的护理。使用受试者数据、生物标本和参与的受试者人群及其家属来提高对这些疾病的认识和治疗受影响的儿童也将是即将到来的资助期的优先事项。西雅图临床中心有经验，专业知识， 和良好的跟踪记录，继续参与ChiLDReN，并有预期的患者随着时间的推移，以支持新的财团试验。Murray博士和她的临床中心团队还提出了一项关于Alagille综合征受试者出血风险和血小板功能的研究，与α-1-抗胰蛋白酶缺乏症和进行性家族性肝内胆汁淤积症受试者进行比较，这将填补我们对这种情况的知识空白，并显著影响Alagille儿童。本研究将使用儿科出血问卷评估这些儿童的出血风险，然后使用血小板功能分析仪-100和血栓弹力图分析他们的血小板功能。这些ChiLDReN受试者的分析结果将结合收集的这些受试者的联盟数据进行评价，以验证Alagille综合征儿童的出血风险增加，这是其综合征特有的，部分原因是血小板功能障碍。