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
Russo, Pierre; Magee, John C; Anders, Robert A et al. (2016) Key Histopathologic Features of Liver Biopsies That Distinguish Biliary Atresia From Other Causes of Infantile Cholestasis and Their Correlation With Outcome: A Multicenter Study. Am J Surg Pathol 40:1601-1615
Shneider, Benjamin L; Magee, John C; Karpen, Saul J et al. (2016) Total Serum Bilirubin within 3 Months of Hepatoportoenterostomy Predicts Short-Term Outcomes in Biliary Atresia. J Pediatr 170:211-7.e1-2
Wang, Kasper S; Tiao, Greg; Bass, Lee M et al. (2016) Analysis of surgical interruption of the enterohepatic circulation as a treatment for pediatric cholestasis. Hepatology :
Grammatikopoulos, Tassos; Sambrotta, Melissa; Strautnieks, Sandra et al. (2016) Mutations in DCDC2 (doublecortin domain containing protein 2) in neonatal sclerosing cholangitis. J Hepatol 65:1179-1187
Leung, Daniel H; Ye, Wen; Molleston, Jean P et al. (2015) Baseline Ultrasound and Clinical Correlates in Children with Cystic Fibrosis. J Pediatr 167:862-868.e2
Thompson, Richard J; Bull, Laura N (2015) Treating genetic disease: Expanding the options. Hepatology 62:349-51
Ye, Wen; Rosenthal, Philip; Magee, John C et al. (2015) Factors Determining δ-Bilirubin Levels in Infants With Biliary Atresia. J Pediatr Gastroenterol Nutr 60:659-63
Tsai, Ellen A; Grochowski, Christopher M; Falsey, Alexandra M et al. (2015) Heterozygous deletion of FOXA2 segregates with disease in a family with heterotaxy, panhypopituitarism, and biliary atresia. Hum Mutat 36:631-7
Kamath, Binita M; Chen, Zhen; Romero, Rene et al. (2015) Quality of Life and Its Determinants in a Multicenter Cohort of Children with Alagille Syndrome. J Pediatr 167:390-6.e3
Teckman, Jeffrey H; Rosenthal, Philip; Abel, Robert et al. (2015) Baseline Analysis of a Young α-1-Antitrypsin Deficiency Liver Disease Cohort Reveals Frequent Portal Hypertension. J Pediatr Gastroenterol Nutr 61:94-101

Showing the most recent 10 out of 25 publications