Chronic pediatric liver disease is a devastating group of conditions that have a profound effect on children, their families, and our society. Biliary atresia and the other cholestatic liver diseases studied by this network account for over half of liver transplants performed in children in the United States. A better understanding of the diseases studied by this network will help provide better care of patients with chronic liver disease. The gaps in our current knowledge regarding these diseases provide compelling scientific justification for the continuation of ChiLDReN. As stated in the RFA, the primary goal of this proposal is to continue clinical and translational research on rare pediatric liver disease that include: biliary atresia;Alagille syndrome, alpha-1-antitrypsin deficiency, progressive familial intrahepatic cholestasis syndromes, bile acid synthesis defects, mitochondrial hepatopathies, idiopathic neonatal hepatitis, and cystic fibrosis liver disease. It is anticipated that the network will consist of up to 15 clinical sites and a single data coordinating center (DCC). The DCC for the ChiLDReN study is charged with providing coordination, communications and logistical support, clinical study design, centralized data management, biosample management, quality assurance, and analytical support to the research sites and the NIDDK Project Scientist for all ChiLDReN studies. The proposing team is submitting this response to RFA-DK-13-011 to continue as the DCC for the network.

Public Health Relevance

The Childhood Liver Disease Research Network (ChiLDReN)* studies babies, children, and young adults who have rare mostly cholestatic liver diseases. The researchers will ask patients to participate in studies that will help us: 1) Develop new tests to better diagnose these diseases, 2) Learn more about what causes these diseases, 3) Develop better treatment options for children living with these diseases, 4) Develop better treatment options for children who receive a liver transplant for these diseases *When referencing the current research network, we use the acronym ChiLDREN. When referencing the proposed network described in RFA-DK-13-011: Continuation of ChiLDReN, we use the acronym ChiLDReN.

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Research Project--Cooperative Agreements (U01)
Project #
2U01DK062456-13
Application #
8774729
Study Section
Special Emphasis Panel (ZDK1-GRB-7 (M1))
Program Officer
Sherker, Averell H
Project Start
2002-09-18
Project End
2019-05-31
Budget Start
2014-08-20
Budget End
2015-05-31
Support Year
13
Fiscal Year
2014
Total Cost
$2,800,068
Indirect Cost
$559,575
Name
University of Michigan Ann Arbor
Department
Surgery
Type
Schools of Medicine
DUNS #
073133571
City
Ann Arbor
State
MI
Country
United States
Zip Code
48109
Loomes, Kathleen M; Spino, Cathie; Goodrich, Nathan P et al. (2018) Bone Density in Children With Chronic Liver Disease Correlates With Growth and Cholestasis. Hepatology :
Ng, Vicky L; Sorensen, Lisa G; Alonso, Estella M et al. (2018) Neurodevelopmental Outcome of Young Children with Biliary Atresia and Native Liver: Results from the ChiLDReN Study. J Pediatr 196:139-147.e3
Shneider, Benjamin L; Spino, Cathie; Kamath, Binita M et al. (2018) Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome. Hepatol Commun 2:1184-1198
Alonso, Estella M; Ye, Wen; Hawthorne, Kieran et al. (2018) Impact of Steroid Therapy on Early Growth in Infants with Biliary Atresia: The Multicenter Steroids in Biliary Atresia Randomized Trial. J Pediatr 202:179-185.e4
Ye, Wen; Narkewicz, Michael R; Leung, Daniel H et al. (2018) Variceal Hemorrhage and Adverse Liver Outcomes in Patients With Cystic Fibrosis Cirrhosis. J Pediatr Gastroenterol Nutr 66:122-127
Wang, Kasper S; Tiao, Greg; Bass, Lee M et al. (2017) Analysis of surgical interruption of the enterohepatic circulation as a treatment for pediatric cholestasis. Hepatology 65:1645-1654
Shneider, Benjamin L; Moore, Jeff; Kerkar, Nanda et al. (2017) Initial assessment of the infant with neonatal cholestasis-Is this biliary atresia? PLoS One 12:e0176275
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
Tsai, Ellen A; Gilbert, Melissa A; Grochowski, Christopher M et al. (2016) THBS2 Is a Candidate Modifier of Liver Disease Severity in Alagille Syndrome. Cell Mol Gastroenterol Hepatol 2:663-675.e2
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

Showing the most recent 10 out of 34 publications