Related Experiment Video
Updated: Jun 15, 2025
Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Liver transplantation for pediatric genetic and metabolic disorders
Sara Hassan1, Ahmad Anouti2, Queenie K-G Tan3
1Division of Pediatric Gastroenterology, Department of Pediatrics, Hepatology and Nutrition, Mayo Clinic, Rochester, Minnesota, USA.
Insights
Pediatric genetic and metabolic liver diseases are a major reason for liver transplants in children. This review covers their causes, symptoms, and how liver transplantation is used to treat them.
Area of Science:
- Pediatric Hepatology
- Metabolic Disorders
- Genetic Diseases
Background:
- Genetic and metabolic liver diseases are common in children.
- They are the second leading cause for pediatric liver transplantation.
- Biliary atresia is the primary indication.
Purpose of the Study:
- To review pediatric genetic and metabolic liver diseases.
- To discuss their pathophysiology and clinical presentation.
- To examine the role of liver transplantation in management.
Main Methods:
- Literature review of pediatric genetic and metabolic liver diseases.
- Analysis of pathophysiology and clinical manifestations.
- Evaluation of liver transplantation indications and outcomes.
Main Results:
- These diseases present a wide spectrum of conditions.
- Transplantation decisions involve hepatic and extrahepatic factors.
- Outcomes after transplantation are a key consideration.
Conclusions:
- Genetic and metabolic liver diseases significantly impact pediatric liver health.
- Liver transplantation is a critical therapeutic option.
- Careful patient selection is essential for successful outcomes.
Abstract:
Pediatric genetic and metabolic liver diseases comprise a broad spectrum of conditions and represent the second most common indication for liver transplantation following biliary atresia. The decision to transplant can be challenging and requires consideration of several factors, including hepatic involvement, extrahepatic manifestations, and anticipated posttransplant outcomes. This review examines pediatric genetic and metabolic liver diseases, their pathophysiology, clinical presentation, and the role of liver transplantation.
