Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children
Arbor Research Collaborative for Health
Summary
Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. In people with PSC, the bile backs up into the liver and will damage it, causing scarring of the liver. The purposes of this study are to: * Collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly. * Ask questions about how PSC symptoms affect your child's life to learn more about its impact on your child's daily functioning * Children with PSC who are seen at one of the participating clinical sites in the Childhood Liver Disease Research Network (ChiLDReN) will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC.
Description
Pediatric primary sclerosing cholangitis (PSC) is a rare autoimmune biliary fibrosing disease that leads to significant morbidity, the need for liver transplantation in \~50% of patients, and an increased risk for biliary and colorectal cancers in adulthood. The progression of the biliary disease in children is variable and risk factors associated with a more rapid progression of disease have not been adequately studied. Importantly, pediatric hepatologists have never previously collaborated with inflammatory bowel disease (IBD) specialists to rigorously explore interactions between colonic in…
Eligibility
- Age range
- 2–25 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Patients with the clinical diagnosis of large or small duct PSC made at any time prior to enrollment are screened for eligibility to participate in this prospective cohort study. The site PI will determine eligibility following review of MRCP or ERCP images with the site radiologist to confirm presence of an abnormal cholangiogram at the time of diagnosis of large duct PSC. Liver histopathology obtained at the time of diagnosis of small duct PSC will be reviewed with the site pathologist prior to enrollment. Individuals must meet all of the Inclusion criteria in order to…
Locations (12)
- Children's Hospital of Los AngelesLos Angeles, California
- Children's Hospital ColoradoAurora, Colorado
- Children's Healthcare of AtlantaAtlanta, Georgia
- Ann & Robert H Lurie Children's HospitalChicago, Illinois
- Riley Hospital for ChildrenIndianapolis, Indiana
- Cincinnati Children's Hospital MedicalCincinnati, Ohio