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Long-term Outcomes of Liver Transplantation for Inborn Errors of Metabolism in Children
William Miller1, Jillian Wothe1, Qi Wang2
1Department of Surgery, University of Minnesota Medical School, Minneapolis, Minnesota.
Insights
Liver transplantation for inborn errors of metabolism shows excellent long-term survival for patients and grafts. This procedure also maintains a high quality of life for pediatric recipients.
Area of Science:
- Hepatology
- Transplantation immunology
- Pediatric gastroenterology
Background:
- Liver transplantation is a common treatment for inborn errors of metabolism with historically good outcomes.
- Consideration of post-transplant quality of life is crucial for patients undergoing this procedure.
Purpose of the Study:
- To evaluate long-term patient and graft survival after liver transplantation in pediatric patients.
- To assess the quality of life in pediatric liver transplant recipients with metabolic indications.
Main Methods:
- Retrospective, observational cohort study of pediatric liver transplant recipients (2010-2020).
- Comparison of metabolic versus non-metabolic indications for liver transplant.
- Prospective administration of PedsQL Transplant Module and RAND 36-Item Health Survey 1.0 for metabolic group.
Main Results:
- Ten-year patient survival was significantly higher in the metabolic group compared to the non-metabolic group (p < .05).
- No significant difference in 10-year graft survival between the two groups.
- Metabolic recipients reported a median quality of life score of 88, comparable to healthy children.
Conclusions:
- Liver transplantation for inborn errors of metabolism yields excellent long-term patient and graft survival.
- This intervention effectively maintains a high quality of life in pediatric recipients.
Background:
Liver transplantation for inborn errors of metabolism is increasingly common and has historically had positive outcomes. However, this therapeutic modality is not without risks, and patient post-transplant quality of life should be part of the consideration.
Methods:
This retrospective, observational cohort study included all pediatric patients receiving liver transplant from 2010 through 2020 at a single center. Recipients were split into 2 groups based on metabolic or non-metabolic indications for liver transplant. Ten-year patient survival and graft survival were analyzed. The PedsQL Transplant Module and RAND 36-Item Health Survey 1.0 were administered prospectively to those recipients with metabolic indications.
Results:
Ten-year patient survival was statistically significantly higher in the metabolic group than in the non-metabolic (p < .05), and there was no difference in 10-year graft survival between groups. Of the 12 patients in the metabolic group who completed the PedsQL Transplant Module or RAND 36-Item Health Survey 1.0, the median score was 88, similar to the score seen in healthy children.
Conclusions:
Liver transplantation for inborn errors of metabolism provides excellent long-term outcomes in terms of patient and graft survival, while maintaining a high quality of life.
