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[Alpha-1-antitrypsin deficiency. An indication for pediatric liver transplantation]
A De Roover1, O Detry, P Honoré
1Service de Chirurgie Abdominale et Transplantation, Université de Liège.
Insights
Alpha-1-antitrypsin deficiency is a common genetic disorder causing liver disease in children. Successful liver transplantation can restore health, allowing young patients to return to normal activities.
Area of Science:
- Hepatology
- Genetics
- Pediatric Gastroenterology
Background:
- Alpha-1-antitrypsin deficiency (AATD) is the most frequent inherited metabolic disorder impacting the liver.
- It is the second leading indication for pediatric liver transplantation, following biliary atresia.
Observation:
- A 6-year-old female presented with end-stage liver disease attributed to AATD.
- The patient underwent a successful whole liver transplantation using a graft from a 3-year-old donor.
Findings:
- The liver transplant effectively treated the end-stage liver disease caused by AATD.
- The recipient experienced a positive recovery, returning to school within three months post-transplant.
Implications:
- This case highlights liver transplantation as a viable and effective treatment for pediatric AATD.
- Further understanding of AATD pathogenesis and natural history is crucial for managing affected children.
Abstract:
Alpha-1-antitrypsin deficiency is the most common inborn error of metabolism leading to liver transplantation, and the second cause of liver transplantation in children after biliary atresia. The authors report the case of a 6-year-old girl, who was suffering from end-stage liver disease secondary to alpha-1-antitrypsin deficiency. She was successfully treated by whole liver transplantation, the hepatic graft coming from a 3-year-old donor. Three months later she went back to school. The authors discuss the pathogenesis and the natural history of this frequent cause of liver transplantation in children.
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