The Global Alagille Alliance Study (GALA)
Purpose: Our participation in this registry is part of an international, retrospective, multicenter cohort study to determine the prevalence and natural history, socio-economic burden, and outcomes of individuals with Alagille syndrome (ALGS), with a focus on extrahepatic manifestations.
Long-term outcome and risk stratification in autoimmune hepatitis – an international, multicenter retrospective observational study (LORSIAH)
Purpose: Our participation in this registry is part of an international, retrospective, multicenter cohort study to ascertain the long-term outcomes and predictors of death and/or liver transplantation in patients with autoimmune hepatitis (AIH). The goal of this registry is to investigate surrogate end points of long-term outcomes to monitor disease progression and evaluate treatments. This collaborative effort will help us in improving our treatment strategies and give us support in the clinical management and decision-making of treatment of AIH.
Pediatric Primary Sclerosing Cholangitis Outcomes Study
Purpose: Primary sclerosing cholangitis is a chronic, immune-mediated, liver disease which is a rare disease in children with few studies that contain large numbers of children that describe outcomes and risk factors for progressive disease. A multicenter collaborative effort is required to adequately evaluate: long term outcome of pediatric PSC, characteristics of pediatric patients who have poor outcomes, differences between small duct and large duct PSC subtypes, and the effects of ursodeoxycholic acid use in pediatric PSC.
Studies of Pediatric liver Transplantation (SPLIT)
Purpose: The purpose of this study is for the health care community to understand the critical role of registries for disease specific populations and the Studies of Pediatric Liver Transplantation (SPLIT) has the opportunity to be at the front of the wave. This study will serve to assess outcomes and in doing so, will form as a foundation for patient-based research and improvement in care. The objectives of this study are to improve outcomes in children receiving liver transplants by collecting data that will serve as a foundation for targeted clinical studies, whether quality improvement/comparative effectiveness studies or clinical trials. The aim is as follows:
- To collect prospective data from pediatric liver transplant centers which permit opportunities to identify and improve early post-transplant outcomes. The focus will be on the drivers of patient and graft survival, length of stay and readmission. Analyses of the data set will guide quality improvement efforts and patient-based research.
- To prospectively collect data from pediatric liver transplant survivors more than one year after transplant so as to identify emerging outcomes, clarify predictors for known outcome and identify best practice. Analyses of the data set will guide quality improvement efforts and patient-based research and serve to help prevent and mitigate immune and non-immune complications.
A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated with Cholbam®/Kolbam® (Cholic Acid)
Purpose: The primary objective of this study is to describe the long-term safety of treatment with Cholbam, a medication that has been designed to replace a bile acid that is not being produced as a result of a genetic defect in bile acid synthesis.