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Biliary Atresia: A Focus on the Patient Journey from Diagnosis to Transplant
Katie R Conover1, Alyssa R Goldberg1, Krupa R Mysore2
1Department of Pediatrics, Pediatric Liver Center, Digestive Health Institute, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA.
Insights
Biliary atresia is a serious infant liver disease causing bile duct blockage. Current treatments focus on managing symptoms and liver transplantation, with a need for new therapies.
Area of Science:
- Pediatric Gastroenterology and Hepatology
- Infant Cholangiopathies
- Liver Disease Research
Background:
- Biliary atresia is a severe infant cholangiopathy.
- It involves extrahepatic bile duct obstruction and intrahepatic inflammation.
- It is the primary reason for pediatric liver transplantation.
Purpose of the Study:
- To review the current literature on the patient journey in biliary atresia.
- To highlight the disease burden from diagnosis to end-stage liver disease and transplantation.
- To identify areas for future research to improve patient care.
Main Methods:
- Comprehensive literature review of updated studies.
- Analysis of the patient's disease progression and treatment course.
- Identification of unmet needs and research opportunities.
Main Results:
- Biliary atresia significantly impacts children's health and requires liver transplantation.
- There are currently no disease-modifying medical therapies available.
- The patient journey involves progressive liver damage and significant burden.
Conclusions:
- Further research is crucial to develop disease-modifying therapies for biliary atresia.
- Improving early diagnosis and management can transform patient outcomes.
- Addressing the burden of disease is key to advancing pediatric liver care.
Abstract:
Biliary atresia is a complex cholangiopathy of infancy characterized by extrahepatic biliary obstruction and progressive intrahepatic fibroinflammatory injury. It remains the leading indication for liver transplant in children without any disease-modifying medical therapies. Herein the authors review updated literature relevant to the patient journey from diagnosis through end-stage liver disease up to liver transplantation. They focus on the burden of disease throughout this course including a focus on actionable areas of potential future study that could help transform patient care.
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