BCM/TCH CHOLESTATIC LIVER DISEASE CONSORTIUM
BCM/TCH 胆汁淤积性肝病联盟
基本信息
- 批准号:10632146
- 负责人:
- 金额:$ 36万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2014
- 资助国家:美国
- 起止时间:2014-09-10 至 2024-05-31
- 项目状态:已结题
- 来源:
- 关键词:AcetylcysteineAddressAdjuvant TherapyAlagille SyndromeAmericanBacterial GenesBile Acid Biosynthesis PathwayBile AcidsBile fluidBiliary AtresiaBiological MarkersChildChild HealthChildhoodCholestasisClinicalClinical DataClinical ResearchClinical TrialsCystic FibrosisDefectDevelopmentDiagnosisDiseaseDuct (organ) structureEnrollmentEtiologyEvidence based treatmentFecesFibrosisFundingGene Expression ProfileGenomicsHepatologyHourImpairmentIndividualInfantInflammatory Bowel DiseasesInterventionIntravenousInvestigationLeadLeadershipLifeLiverLiver diseasesLongitudinal StudiesMarketingMedicineMetagenomicsMethodsMitochondriaMorbidity - disease rateObservational StudyOutcomeOutcome MeasurePathogenesisPatientsPatternPediatric HospitalsPerformancePhasePilot ProjectsProgressive intrahepatic cholestasisProtocols documentationResearchResearch ActivityResearch PersonnelRoleSafetySamplingSclerosing CholangitisSerumSeverity of illnessTestingTexasTranslational ResearchUnited StatesValidationWhole-Genome Shotgun Sequencingalpha 1-Antitrypsin Deficiencyclinical centerclinical investigationclinical phenotypecollegedata repositorydisease phenotypedisease prognosisfeasibility trialfecal microbiomefollow-upgenome sequencinggut microbiomeimprovedimproved outcomeliver transplantationlongitudinal analysismetropolitanmicrobial signaturemicrobiome analysismicrobiome researchmicrobiome signaturemortalitynovelopen labelpilot trialpopulation basedprimary outcomeprimary sclerosing cholangitisprospectiveresearch studysecondary outcomestool sampletrial designwhole genome
项目摘要
Project Summary
Cholestatic liver diseases including Alagille syndrome, alpha-1 antitrypsin deficiency, bile acid synthesis
defects, biliary atresia, cystic fibrosis, mitochondrial hepatopathies, progressive familial intrahepatic cholestasis
and primary sclerosing cholangitis, lead to significant morbidity and mortality in childhood, frequently
necessitating liver transplantation. No single North American clinical center sees a large enough number of
patients with these disorders to permit a rigorous approach to addressing unresolved questions including
etiology and pathogenesis, optimal methods of diagnosis and treatment, and factors that influence disease
severity and prognosis. This competitive renewal proposal from the Baylor College of Medicine and Texas
Children's Hospital (BCM/TCH) seeks to continue ongoing research activities in the Childhood Liver Disease
Research Network (ChiLDReN). This application for renewal funding includes a strong commitment to
continuing the on-going research efforts and two new proposals, a novel pilot and feasibility clinical trial applied
to biliary atresia and a translational protocol focused on the pathogenesis of sclerosing cholangitis. The clinical
center at BCM/TCH includes an outstanding group of clinician investigators with an extensive track record in
synergistic translational and clinical research relevant to pediatric liver diseases. Performance to date in the
on-going studies of ChiLDReN has been exemplary and has taken full advantage of a predominant market
share of the population base of the Houston metropolitan region (5th largest in the United States) and the large
referral patterns to TCH as a quaternary center for Pediatric Hepatology and Liver Transplantation. A pilot and
feasibility trial of intravenous NAC will be conducted as an open label investigation in infants undergoing
hepatoportoenterstomy for biliary atresia (IND 135796 and NCT03499249). The primary outcome measure will
focus on biomarkers of bile flow. Promising findings in this pilot study could be expanded to a larger scale
multi-center investigation as part of ChiLDReN. The proposed translational protocol will use metagenomic
whole genome sequencing to assess the bacterial fecal microbiome in children with sclerosing cholangitis and
correlate microbial signatures and bacterial gene expression patterns with clinical phenotypes characterized in
the newly developed prospective observational study of primary sclerosing cholangitis in children.
项目摘要
胆汁淤积性肝病包括Alagille综合征、α-1抗胰蛋白酶缺乏、胆汁酸合成
缺陷、胆道闭锁、囊性纤维化、线粒体肝病、进行性家族性肝内胆汁淤积症
和原发性硬化性胆管炎,在儿童时期经常导致显著的发病率和死亡率
有必要进行肝脏移植。没有一家北美临床中心能看到足够多的
这些疾病的患者允许采取严格的方法来解决未解决的问题,包括
病因和发病机制,最佳诊断和治疗方法,以及影响疾病的因素
严重程度和预后。这项由贝勒医学院和德克萨斯州提出的竞争性续签提案
儿童医院(BCM/TCH)寻求继续进行儿童肝病的研究活动
研究网络(儿童)。这份续签资金申请包括对
继续正在进行的研究工作和两项新建议,应用了一项新的中试和可行性临床试验
到胆道闭锁和一项专注于硬化性胆管炎发病机制的翻译方案。临床部
BCM/TCH中心包括一批杰出的临床医生研究人员,他们在
与儿科肝病相关的协同式翻译和临床研究。到目前为止的表现
正在进行的儿童研究堪称典范,并充分利用了一个占主导地位的市场
休斯敦大都市区的人口基数(美国第五大)和
TCH作为儿科肝病和肝移植第四中心的转诊模式。一名飞行员和
静脉注射NAC的可行性试验将作为一项开放标签研究在接受治疗的婴儿中进行
胆道闭锁的肝门肠吻合术(IND 135796和NCT03499249)。主要结果衡量标准将是
关注胆汁流动的生物标志物。这项初步研究中有希望的发现可以扩大到更大的规模。
作为儿童的一部分,多中心调查。拟议的翻译协议将使用元基因组学
全基因组测序评估硬化性胆管炎和慢性胆管炎患儿的细菌粪便微生物群
微生物特征和细菌基因表达模式与临床表型的相关性
儿童原发性硬化性胆管炎最新前瞻性观察研究。
项目成果
期刊论文数量(1)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
Development of a Novel Tool to Assess the Impact of Itching in Pediatric Cholestasis.
- DOI:10.1007/s40271-017-0266-4
- 发表时间:2018-03
- 期刊:
- 影响因子:0
- 作者:Kamath BM;Abetz-Webb L;Kennedy C;Hepburn B;Gauthier M;Johnson N;Medendorp S;Dorenbaum A;Todorova L;Shneider BL
- 通讯作者:Shneider BL
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{{ truncateString('BENJAMIN L SHNEIDER', 18)}}的其他基金
Clinical Center for ChiLDREN: Pathogenesis, Biomarkers, and Antifibrotic Therapy
ChiLDREN 临床中心:发病机制、生物标志物和抗纤维化治疗
- 批准号:
9552403 - 财政年份:2014
- 资助金额:
$ 36万 - 项目类别:
Clinical Center for ChiLDREN: Pathogenesis, Biomarkers, and Antifibrotic Therapy
ChiLDREN 临床中心:发病机制、生物标志物和抗纤维化治疗
- 批准号:
9135724 - 财政年份:2014
- 资助金额:
$ 36万 - 项目类别:
Clinical Center for ChiLDREN: Pathogenesis, Biomarkers, and Antifibrotic Therapy
ChiLDREN 临床中心:发病机制、生物标志物和抗纤维化治疗
- 批准号:
8774339 - 财政年份:2014
- 资助金额:
$ 36万 - 项目类别:
Clinical Center for ChiLDREN: Pathogenesis, Biomarkers, and Antifibrotic Therapy
ChiLDREN 临床中心:发病机制、生物标志物和抗纤维化治疗
- 批准号:
9317473 - 财政年份:2014
- 资助金额:
$ 36万 - 项目类别:
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