The Childhood Liver Disease Research and Education Network (ChiLDREN) has recently been funded and combines the Biliary Atresia Research Consortium (BARC) and the Cholestatic Liver Disease Consortium (CLiC), as well as new studies on cystic fibrosis liver disease. This consolidation seeks to facilitate our ability to discover new diagnostics, etiologic, and treatment options for children with liver disease, and those who undergo liver transplantation, and to train the next generation of investigators in rare pediatric liver diseases.
Among the diseases/disorders addressed:
Biliary Atresia Research Consortium (BARC)
The goals of BARC are to:
- Provide long-term follow-up of biliary atresia/cholestatic patients in BARC centers.
- Develop and maintain a database of detailed information on children and young adults with biliary atresia
- Establish a specimen repository
- Implement a clinical trial to investigate the effects of corticosteroids in children who have undergone Kasai surgery
- Stimulate and encourage future research
PROBE: A Prospective Database of Infants with Cholestasis
This is a prospective, comprehensive database study of all infants in the BARC centers fulfilling the inclusion criteria of 0 – 180 days of age with cholestasis (serum direct bilirubin of 2 mg/dl or greater and >20% of the total bilirubin). The factors which might predict clinical outcomes, such as age at diagnosis and age of the portoenterostomy, are being investigated, as are long term assessments of health related quality of life, school performance, and neurodevelopmental outcome. A serum/plasma/tissue bank and immortalized cell lines are also being created to facilitate scientific studies of these diseases.
SUBJECTS ARE ACTIVELY BEING ENROLLED. PLEASE CONTACT Anitha Devadason or Kim Pfeifer FOR FURTHER DETAILS.
START: A randomized double-blinded, placebo-controlled trial of corticosteroid therapy following portoenterostomy in infants with biliary atresia
This randomized controlled trial is designed to study the efficacy and safety of high-dose perioperative corticosteroids for 2 months following portoenterostomy. The primary outcome is serum bilirubin at 6 months after surgery and the secondary outcomes include survival without liver transplantation, complications, and PELD score. Toxicity measures being investigated include adverse events, vaccine responses, infections, neurodevelopmental outcome, and GI bleeding.
ENROLLMENT HAS CLOSED. PLEASE CONTACT Anitha Devadason or Kim Pfeifer FOR FURTHER DETAILS.
BASIC: Biliary Atresia Study in Infants and Children: A Database of Older Children with Biliary Atresia
This long-term follow-up of biliary atresia patients in BARC centers is being conducted to learn more about the natural history and causes of Biliary Atresia in children. This study is also being done to develop a repository of samples of blood and urine from older children with Biliary Atresia to be used in research about liver disease. Children one year of age or older with a confirmed diagnosis of Biliary Atresia who are pre and post liver transplant can take part in this study.
SUBJECTS ARE ACTIVELY BEING ENROLLED. PLEASE CONTACT Anitha Devadason or Kim Pfeifer FOR FURTHER DETAILS.
Cholestatic Liver Disease Consortium (CLiC)
The Cholestatic Liver Disease Consortium (CLiC) studies infants and children with rare cholestatic liver diseases. The goal of the CLiC team is to investigate five genetic causes of intrahepatic cholestasis and to train the next generation of clinical researchers. These five diseases are:
- Alpha-1-antitrypsin (a-1AT) deficiency
- Alagille syndrome (AGS)
- Progressive familial intrahepatic cholestasis (PFIC)
- Bile acid synthesis and metabolism defects
- Mitochondrial Hepatopathies
LOGIC: A longitudinal study of genetic causes of Intrahepatic Cholestasis
The CLiC network collects information from infants’ and children's medical records and scans, blood, urine, and tissue samples in order to understand the causes of these diseases and to improve the diagnosis and treatment of infants and children with these diseases.
SUBJECTS ARE ACTIVELY BEING ENROLLED. PLEASE CONTACT Anitha Devadason or Kim Pfeifer FOR FURTHER DETAILS.
The Cystic Fibrosis Liver Disease (CFLD) network
The Cystic Fibrosis Liver Disease (CFLD) network collects information from children's medical records and scans, blood and urine samples in order to understand the causes of this disease and to improve the diagnosis and treatment of children with this disease.
PUSH: Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis Liver Disease
Currently, the CFLD network is enrolling patients into the PUSH study.
SUBJECTS ARE ACTIVELY BEING ENROLLED. PLEASE CONTACT Wikrom Karnsakul, M.D. FOR FURTHER DETAILS.