Long-term Outcomes of Liver Transplantation for Inborn Errors of Metabolism in Children

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.