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 the Childhood Liver Disease Research Network (ChiLDReN). As stated in the RFA, the primary goal of this proposal is to continue clinical and translational research on rare pediatric liver diseases that include: biliary atresia; Alagille syndrome; alpha-1-antitrypsin deficiency; progressive familial intrahepatic cholestasis syndromes; bile acid synthesis defects; mitochondrial hepatopathies; idiopathic neonatal hepatitis; cystic fibrosis liver disease; and primary sclerosing cholangitis. It is anticipated that the network will consist of up to 13 clinical sites and a single scientific and data coordinating center (SDCC). The SDCC 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-18-502 to continue as the SDCC for the network.
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: ? Develop new tests to better diagnose these diseases ? Learn more about what causes these diseases ? Develop better treatment options for children living with these diseases
