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大原因，是儿科肝移植的主要原因。诉讼联盟的初始资金期限 导致儿童肝病研究网络（儿童）导致了良好的表型受试者以及银行数据和生物标本的前所未有的收集。尽管需要持续招募具有这些罕见条件的受试者，以充分实现许多个人研究的目的，但现在的受试者，数据和生物质量的收集足以支持对这些疾病发病机理的有意义的研究并为新疗法的研究提供了信息。下一个资金期限将继续并通过继续完成正在进行的研究来继续并扩大对自然史，遗传学和治疗的正在进行的研究。此外，将对新疗法进行试用，以期改善可以提供给患有这些疾病的儿童提供的护理。使用这些疾病知识的知识以及受影响儿童的治疗的主题数据，生物测量和敬业的受试者及其家人的使用也将是即将到来的资金期限的优先事项。西雅图临床中心具有经验，专业知识， 并经过验证的记录，可以继续参加儿童，并且随着时间的推移，有预期的患者支持新的财团试验。默里博士和她的临床中心团队还提出了一项研究阿拉吉尔综合征受试者出血风险和血小板功能的研究，与患有α-1-抗抗蛋白酶缺乏症的受试者和渐进式家族性肝内胆汁淤积相比，这将在我们的知识中填补这一疾病的空白，并对儿童产生严重影响。这项研究将使用小儿出血问卷来评估这些儿童的出血风险，然后利用血小板功能分析仪100和血栓射击分析其血小板功能。对这些儿童受试者分析的结果将与有关这些受试者的收集的联盟数据一起评估，以验证阿拉吉尔综合征的儿童的出血风险增加了其综合征所特有的出血风险，这部分是由于血小板功能障碍所致。