https://pubmed.ncbi.nlm.nih.gov/39034193/ transplant

Transplant Proc. 2024 Jul 20:S0041-1345(24)00318-X.
doi: 10.1016/j.transproceed.2024.03.037.Online ahead of print.

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

William Miller 1, Jillian Wothe 1, Qi Wang 2, David Vock 2, Heli Bhatt 3, Amritha Salunke 3, Sara Jane Schwarzenberg 3, Susan Berry 3, Srinath Chinnakotla 4
Affiliations expand
PMID: 39034193

DOI: 10.1016/j.transproceed.2024.03.037
Abstract

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.