UCSF is currently one of the ten original sites in the Biliary Atresia Research Consortium (BARC) and the Cholestatic Liver Consortium (CLiC) which is being continued, expanded and merged to form the Childhood Liver Disease Research and Education Network (ChiLDREN). The specific objectives of the newly formed ChiLDREN could include but are not limited to: 1) Completion of the steroid safety and efficacy clinical trial in biliary atresia. 2) Continuation of the BARC and CLiC prospective longitudinal studies of children to continue to provide data and biospecimens for ancillary studies aimed at discovering new diagnostics, etiologic and treatment options for children both pre and post liver transplantation. 3) Continuation of the prospective longitudinal study of cystic fibrosis liver disease (CFLD) aimed at identifying predictors of development of liver disease in children with CF as well as predictors of outcome in children with CFLD. 4) Identification and validation of non-invasive markers of liver disease in CF patients including but not limited to new imaging modalities. 5) Phase l/ll study of new agents to treat cholestasis in children. 6) Study of modifier genes in Alagille Syndrome and Alpha-1-antrypsin disease. 7) Expansion to include other diseases of cholestasis in children with industry and patient advocacy group support. 8) Continue to provide training opportunities for investigators in pediatric liver disease. 9) Continue to provide support for small pilot and demonstration projects within the Network.10) Continue to provide education about pediatric liver diseases to the scientific and lay communities through publications and the website. In addition, at UCSF we specifically plan to continue to investigate the role of HLA in biliary atresia in collaboration with Dr. Cara Mack at the University of Colarado. Further, in order to determine if rotavirus plays an etiologic role in biliary atresia, we plan to compare data from the former BARC database and new ChiLDREN database with CDC data for rotavirus activity in the United States. Dr. Bull plans to develop fast and reliable screening methods for diagnosis of PFIC. Relevance: Combining BARC and CLiC will enhance the capacity for unhindered flow of clinical information, allow for greater efficiency, permit standardization of process, data, and specimen collection methodology, allow the network to take advantage of available resources offered by lay organizations to expand the number of diseases studied, and include follow-up of children after transplant. Our specific studies at UCSF aim to aid in diagnosis and treatment of biliary atresia and PFIC.

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Research Project--Cooperative Agreements (U01)
Project #
5U01DK062500-11
Application #
8327876
Study Section
Special Emphasis Panel (ZDK1-GRB-S (M1))
Program Officer
Sherker, Averell H
Project Start
2002-09-15
Project End
2014-05-31
Budget Start
2012-06-01
Budget End
2013-05-31
Support Year
11
Fiscal Year
2012
Total Cost
$223,500
Indirect Cost
$94,537
Name
University of California San Francisco
Department
Pediatrics
Type
Schools of Medicine
DUNS #
094878337
City
San Francisco
State
CA
Country
United States
Zip Code
94143
Ng, Vicky Lee; Haber, Barbara H; Magee, John C et al. (2014) Medical status of 219 children with biliary atresia surviving long-term with their native livers: results from a North American multicenter consortium. J Pediatr 165:539-546.e2
Sambrotta, Melissa; Strautnieks, Sandra; Papouli, Efterpi et al. (2014) Mutations in TJP2 cause progressive cholestatic liver disease. Nat Genet 46:326-8
Venkat, Veena L; Shneider, Benjamin L; Magee, John C et al. (2014) Total serum bilirubin predicts fat-soluble vitamin deficiency better than serum bile acids in infants with biliary atresia. J Pediatr Gastroenterol Nutr 59:702-7
Bezerra, Jorge A; Spino, Cathie; Magee, John C et al. (2014) Use of corticosteroids after hepatoportoenterostomy for bile drainage in infants with biliary atresia: the START randomized clinical trial. JAMA 311:1750-9
Sundaram, Shikha S; Alonso, Estella M; Haber, Barbara et al. (2013) Health related quality of life in patients with biliary atresia surviving with their native liver. J Pediatr 163:1052-7.e2
Superina, Riccardo; Magee, John C; Brandt, Mary L et al. (2011) The anatomic pattern of biliary atresia identified at time of Kasai hepatoportoenterostomy and early postoperative clearance of jaundice are significant predictors of transplant-free survival. Ann Surg 254:577-85
Evason, Kimberley; Bove, Kevin E; Finegold, Milton J et al. (2011) Morphologic findings in progressive familial intrahepatic cholestasis 2 (PFIC2): correlation with genetic and immunohistochemical studies. Am J Surg Pathol 35:687-96
Russo, Pierre; Magee, John C; Boitnott, John et al. (2011) Design and validation of the biliary atresia research consortium histologic assessment system for cholestasis in infancy. Clin Gastroenterol Hepatol 9:357-362.e2
Pawlikowska, Ludmila; Strautnieks, Sandra; Jankowska, Irena et al. (2010) Differences in presentation and progression between severe FIC1 and BSEP deficiencies. J Hepatol 53:170-8
DeRusso, Patricia A; Ye, Wen; Shepherd, Ross et al. (2007) Growth failure and outcomes in infants with biliary atresia: a report from the Biliary Atresia Research Consortium. Hepatology 46:1632-8

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