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

• Published in: Transplantation proceedings 2024-07, Vol.56 (6), p.1359-1364
• Main Authors: Miller, William, Wothe, Jillian, Wang, Qi, Vock, David, Bhatt, Heli, Salunke, Amritha, Schwarzenberg, Sara Jane, Berry, Susan, Chinnakotla, Srinath
• Format: Article
• Language: English
• Subjects: Adolescent; Child; Child, Preschool; Female; Graft Survival; Humans; Infant; Liver Transplantation; Male; Metabolism, Inborn Errors - surgery; Quality of Life; Retrospective Studies; Treatment Outcome
• ISSN: 0041-1345; 1873-2623; 1873-2623
• DOI: 10.1016/j.transproceed.2024.03.037

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. 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. 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. 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.