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Clinical Center for Cholestatic Liver Disease in Children

Clinical Center for Cholestatic Liver Disease in Children
儿童胆汁淤积性肝病临床中心
批准号:
10631943
负责人:
Alexander Miethke
金额:
$80.1万
依托单位国家:
美国
项目类别:
财政年份:
2002
资助国家:
美国
项目状态:
已结题
起止时间:
2002-09-15 至 2024-05-31
关键词:
AcetylcysteineAcuteAlagille SyndromeAncillary StudyAntioxidantsBile AcidsBiliaryBiliary AtresiaBiological MarkersCaringChildChild CareChildhoodCholestasisClinicalClinical DataClinical ResearchClinical Research ProtocolsClinical TrialsCollectionCystic FibrosisDataData AnalysesData Coordinating CenterDefectDevelopmentDiseaseDisease OutcomeDrainage procedureEducationEnrollmentEpitheliumEtiologyFundingGeneticGoalsGrantIL8 geneInfantInfrastructureInjuryInstitutional Review BoardsInterventionIntestinesIntravenous ImmunoglobulinsKnowledgeLeadershipLiverLiver FibrosisLiver diseasesLogicLongitudinal StudiesMagnetic Resonance ImagingMaintenanceMatrilysinMeasuresMitochondriaMolecularNational Institute of Diabetes and Digestive and Kidney DiseasesNeonatalObservational StudyOutcomePaperPathogenesisPathologyPatientsPerformancePortal HypertensionPositioning AttributeProgressive intrahepatic cholestasisProspective StudiesProtocols documentationPruritusPublicationsQuality of lifeRNA purificationRadiology SpecialtyReportingResearchResearch InfrastructureResearch PersonnelRoleSafetySerumServicesSodiumSteroidsTissue SampleTissuesTranslational ResearchU-Series Cooperative AgreementsUnited States National Institutes of HealthVancomycinWorkalpha 1-Antitrypsin Deficiencybile acid transporterchronic liver diseaseclinical centerclinical developmentclinical research siteclinical translationclinical trial protocoldata submissiondesigndisease natural historyefficacy evaluationelastographyimprovedimproved outcomeinnovationintrahepaticliver cystic fibrosisliver stiffnessmemberopen labelprimary sclerosing cholangitisprospectiveprotocol developmentresponsesmall molecule inhibitorsupport networksymposiumtranslational study

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中文摘要
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项目总结/文摘
英文摘要
PROJECT SUMMARY/ABSTRACT We propose to remain a Member of the Childhood Liver Disease Research Network (ChiLDReN). As a Charter Member of the Network, our proposal represents a logical extension of the long-standing commitment of our Center to improve the care of children with chronic liver disease through innovative patient-based research. In the previous member of the Network, we worked collaboratively with investigators from other Centers and with the Data Coordinating Center to build a strong infrastructure to conduct patient-based studies on biliary atresia, cholestatic syndromes, mitochondrial hepatopathies and cystic fibrosis, with initial work to develop an observational and interventional protocol for children with primary sclerosing cholangitis (PSC). Our key contributions in the previous cycle included: 1) leadership and/or partnership in the development of network-wide clinical studies, with data analyses and publication of 11 original papers; 2) completion of the first two clinical trials (the steroid trial START and the IV-IG trial PRIME); 3) high enrollment and retention of subjects into prospective studies, including the new FORCE study; 4) timely processing of liver tissue by the staff of RNA Purification Service, Bile Acid Analysis Service, and Pathology Service to support Network studies; and 5) co-leading the organization of an NIH symposium on biliary atresia, with publication of its proceedings. We look forward to significantly contributing to the scientific goals and operational excellence of ChiLDReN through three aims. In Aim 1, we will enroll subjects into prospective ChiLDReN protocols to enable observational studies and translational science. This will be done by pursuing high enrollment rate of subjects into approved protocols, with the timely collection and submission of data and tissue samples to meet the enrollment goals established by the Network. In Aim 2, we will develop and implement research protocols for clinical trials. We are working with Network investigators to conduct a trial to determine the efficacy and safety of a small molecule inhibitor of the intestinal sodium-dependent bile acid transporter (IMAGINE-II trial) in children with cholestasis-associated pruritus. We are also developing a clinical trial to determine the efficacy of Vancomycin in children with PSC. And in Aim 3, we propose a new clinical trial focusing on the use of anti- oxidants to decrease biliary injury in infants who acquire biliary drainage after hepatoportoenterostomy for biliary atresia. We also propose an ancillary study to determine the role of matrix metalloproteinase-7 as a biomarker of hepatic fibrosis in biliary atresia. By pursuing the three aims, we will be well positioned to fully execute the new Network aims of pursuing translational science projects and conducting trials to improve the outcome of children with cholestatic liver diseases.
期刊论文(12)
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会议论文
DOI: 10.1126/scitranslmed.aan8462
发表时间: 2017-11-22
期刊: Science translational medicine
影响因子: 17.1
作者: [Lertudomphonwanit C, Mourya R, Fei L, Zhang Y, Gutta S, Yang L, Bove KE, Shivakumar P, Bezerra JA]
通讯作者: Bezerra JA
Exome Sequencing in Individuals with Isolated Biliary Atresia.
孤立性胆道闭锁个体的外显子组测序。
DOI: 10.1038/s41598-020-59379-4
发表时间: 2020
期刊: Scientific reports
影响因子: 4.6
作者: [Rajagopalan,Ramakrishnan, Tsai,EllenA, Grochowski,ChristopherM, Kelly,SusanM, Loomes,KathleenM, Spinner,NancyB, Devoto,Marcella]
通讯作者: Devoto,Marcella
DOI: 10.1002/hep.27401
发表时间: 2015-01
期刊: HEPATOLOGY
影响因子: 13.5
作者: [Heubi, James E., Setchell, Kenneth D. R., Jha, Pinky, Buckley, Donna, Zhang, Wujuan, Rosenthal, Philip, Potter, Carol, Horslen, Simon, Suskind, David]
通讯作者: Suskind, David
DOI: 10.1126/scitranslmed.3002069
发表时间: 2011-09-28
期刊: Science translational medicine
影响因子: 17.1
作者: [Saxena V, Shivakumar P, Sabla G, Mourya R, Chougnet C, Bezerra JA]
通讯作者: Bezerra JA
8
    The role of regulatory T cells in biliary atresia
    • 批准号:
      8529520
    • 项目类别:
    • 资助金额:
      $32.21万
    • 财政年份:
      2012
    • 负责人:
      Alexander Miethke
    • 依托单位:
    Control of hepatic T cell responses in biliary atresia
    • 批准号:
      10133058
    • 项目类别:
    • 资助金额:
      $41.29万
    • 财政年份:
      2012
    • 负责人:
      Alexander Miethke
    • 依托单位:
    The role of regulatory T cells in biliary atresia
    • 批准号:
      8400215
    • 项目类别:
    • 资助金额:
      $33.38万
    • 财政年份:
      2012
    • 负责人:
      Alexander Miethke
    • 依托单位:
    The role of regulatory T cells in biliary atresia
    • 批准号:
      8893971
    • 项目类别:
    • 资助金额:
      $33.38万
    • 财政年份:
      2012
    • 负责人:
      Alexander Miethke
    • 依托单位:
    海外基金