*Purpose: Liver transplantation (LT) for acute and chronic end-stage-liver-disease is a well-accepted therapy in pediatrics. However, for children with metabolic disorders (MD) without intrinsic liver disease, transplantation remains controversial. The purpose of this study was to compare LT outcomes in patients with MD to patients with biliary atresia (BA).

*Methods: A retrospective review was performed on all patients under the age of 21 who underwent LT for MD or BA at a single institution. MD patients with cirrhosis were excluded, namely α1 antitrypsin deficiency (n=5), tyrosinemia (n=3), Navajo hepatopathy (n=2), mitochondrial neurogastrointestinal encephalopathy syndrome (n=1), and hemochromatosis (n=1). Outcomes analyzed included length of stay, readmission rate within 1 year of transplant, graft survival, patient survival, cause of graft failure, and cause of patient death. Maintenance immunosuppression was calcineurin inhibitor based. Statistical analysis was performed using SPSS. p < 0.05 was used for statistical significance.

*Results: From 1992 to 2018, LT was performed for 101 MD and 256 BA patients. MD patients were significantly older than BA patients (4.3 vs 1.3 years, p<0.005). There were no significant differences in length of stay (17.7 vs 18.1 days, p=0.83) or readmission rates within 1 year (21.6 vs 14.8%, p = 0.17) between MD and BA patients, respectively. Patient survival at 1, 3, 10, and 25 years was 99.0%, 99.0%, 96.0%, and 96.0% for MD and 94.9%, 94.5%, 92.5%, and 92.5% for BA patients. Graft survival at 1, 3, 10, and 25 years was 95.0%, 91.0%, 89.1%, and 89.1% for MD and 91.0%, 90.6%, 87.1%, and 86.7% for BA patients. There were no significant differences between patient survival (p=0.28) or graft survival (p=0.65) on Kaplan-Meier analysis. Five MD patients had graft failure from vascular complications (n=4 HAT, n=1 HAT with PVT) and 3 from chronic rejection. Infection (n=1), intraoperative metabolic crisis (n=1), chronic rejection (n=1), PTLD (n=1), and intraoperative bleeding (n=1) accounted for 5 deaths. 23 BA patients had graft failure. Causes included PNF (n=6), vascular complications (n=9 HAT, n=3 PVT, n=1 HAT with PVT), chronic rejection (n=3), and PTLD (n=1). There were 24 deaths in the BA population from vascular complications (n=3 HAT, n=1 HAT with PVT), infection (n=9), PNF (n=4), PTLD (n=2), intraoperative bleeding (n=2), chronic rejection (n=1), pulmonary hypertension (n=1), and gastrointestinal bleeding (n=1).

*Conclusions: In our center’s 25 years of experience, short and long term outcomes for LT in metabolic disease is excellent and comparable to BA patients. LT is a safe and acceptable therapeutic modality not only for end stage liver disease but also serves as therapy for preventing or ameliorating future end-organ damage in pediatric patients with inborn errors of metabolism without intrinsic liver disease.

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