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Long-term survival after liver transplantation in children with metabolic disorders
Liise K Kayler1, Robert M Merion, Samuel Lee
1Department of Surgery, University of Michigan Health System, Ann Arbor, Michigan 48109, USA.
Insights
Pediatric liver transplantation offers a cure for inherited metabolic disorders, significantly improving patient survival rates. This life-saving procedure leads to excellent long-term outcomes and quality of life for young recipients.
Area of Science:
- Hepatology
- Pediatric Gastroenterology
- Transplant Surgery
Background:
- Liver transplantation is a life-saving intervention for children with inherited metabolic disorders.
- It aims to cure metabolic defects and prevent organ failure.
Purpose of the Study:
- To evaluate the outcomes of pediatric liver transplantation for metabolic disorders.
- To compare survival rates and quality of life with non-metabolic liver transplant recipients.
Main Methods:
- Retrospective analysis of 146 pediatric liver transplants (28 metabolic, 118 non-metabolic) from 1986-2000.
- Included various metabolic conditions such as alpha-1-antitrypsin deficiency, Wilson's disease, and hereditary tyrosinemia.
Main Results:
- Actuarial survival rates at 5 and 10 years were 78% and 68% for metabolic disorder recipients.
- Outcomes compared favorably to non-metabolic transplant recipients (65% and 61% at 5 and 10 years).
- Most school-aged children achieved age-appropriate academic progress, with improved quality of life.
Conclusions:
- Pediatric liver transplantation for metabolic disorders yields excellent clinical and biochemical results.
- The procedure ensures long-term survival and enhances the quality of life for most pediatric recipients.
Background:
Liver transplantation for inherited metabolic disorders aims to save the patient's life when the disorder is expected to progress to organ failure, and to cure the underlying metabolic defect.
Methods:
We retrospectively analyzed 146 pediatric liver transplants (28 metabolic; 118 non-metabolic) performed between 1986 and 2000.
Results:
Twenty-eight transplants were performed in 24 children with metabolic disease (8 females; 16 males; age range 3 months to 17 yr). Indications included alpha-1-antitrypsin deficiency (n = 8), two cases each of hyperoxaluria type 1, Wilson's disease, hereditary tyrosinemia type I, citrullinemia, methylmalonic acidemia, and one case each of propionic acidemia, Crigler-Najjar syndrome type I, neonatal hemachromatosis, hemophilia B, Niemann-Pick disease type B, and cystic fibrosis. Eighteen transplants were whole organ grafts and 10 were lobar or segmental. Auxiliary liver transplants were performed in two patients and three received combined liver-kidney transplants. There were three deaths from sepsis, two from chronic rejection, and one from fulminant hepatitis. Seven of 10 patients currently of school age are within 1 yr of expected grade and three who had pretransplant developmental delay have remained in special education. Actuarial survival rates at 5 and 10 yr are 78% and 68%, respectively, with mean follow-up in excess of 5 yr. These results compare favorably to 100 pediatric patients transplanted for non-metabolic etiologies (65% and 61%, respectively) (p= NS).
Conclusions:
Pediatric liver transplantation for metabolic disorders results in excellent clinical and biochemical outcome with long survival and excellent quality of life for most recipients.
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