https://pubmed.ncbi.nlm.nih.gov/34058057/ Liver Transplant
Liver Transpl. 2021 May 31.

Liver Transplantation in Children with Urea Cycle Disorders: The Importance of Minimizing Waiting Time

Ioannis A Ziogas 1, W Kelly Wu 1, Lea K Matsuoka 1, Anita K Pai 2, Einar T Hafberg 2, Lynette A Gillis 2, Thomas M Morgan 3, Sophoclis P Alexopoulos 1

Abstract

Background: Liver transplantation (LT) for children with urea cycle disorders (UCD) is capable of correcting the enzymatic defect and preventing progressive neurologic injury. We describe the characteristics and outcomes of pediatric LT recipients with UCD.
Methods: We identified all pediatric (<18 years) LT candidates with UCD in the United Network for Organ Sharing database (02/2002-09/2020). Multivariable Cox and logistic regression were used to determine risk factors for graft loss and cognitive delay, respectively.

Results: Of 424 patients, 1.9% (n=8/424) experienced waitlist mortality and 95% underwent LT (n=403/424). The most frequently encountered UCD in our cohort were ornithine transcarbamylase deficiency (46.2%), citrullinemia (20.3%), and argininosuccinic aciduria (ASA) (12.9%). The 1-, 3-, and 5-year graft survival rates were 90.4%, 86.3%, and 85.2%, respectively. Multivariable analysis showed a decreased risk of graft loss with increasing weight at LT (adjusted hazard ratio[aHR]=0.96, 95%CI: 0.94-0.99; p=0.02), male sex (aHR=0.49, 95%CI: 0.28-0.85; p=0.01) and ASA diagnosis (aHR=0.29, 95%CI: 0.09-0.98; p=0.047), when adjusting for location (intensive-care/hospital/home) and graft type (both p≥0.65). In multivariable logistic regression, waitlist time (adjusted odds ratio[aOR]=1.10, 95%CI: 1.02-1.17; p=0.009) and male sex (aOR=1.71, 95%CI: 1.02-2.88; p=0.04) were associated with increased odds of long-term cognitive delay.

Conclusion: Waitlist duration is associated with a long-term risk of cognitive delay. Given excellent long-term outcomes, early LT evaluation should be considered in all children with UCD to prevent progressive neurologic injury and optimize cognitive outcomes.