Pediatric liver transplantation for inherited metabolic liver disease: a single-center experience.

Objective: We performed single-center outcome comparison of pediatric recipients who underwent liver transplantation for either genetic or metabolic disease including the clinical impact of using heterozygote parents as living donors.

Materials And Methods: Pediatric liver transplant recipients from September 2007 to December 2010 were included. Patients were separated into 2 categories by etiology of liver disease: (1) genetic or metabolic liver disease (G/M) and (2) nongenetic or metabolic liver disease (non-G/M), which included all other remaining etiologies combined. Patient demographics, recipient and donor characteristics, graft type, operative data, recipient complications, allograft and patient survival were analyzed.

Results: Forty liver transplants were performed on 40 patients; 18 were transplanted for G/M; mean waiting time was 101 days for G/M group and 57 days for non-G/M group; 9 patients were listed as status 1, 5 were granted PELD/MELD exceptions; the overall mean PELD/MELD score was 21. Four G/M patients had hepatocellular carcinoma in the explant without microvascular invasion. Overall complications requiring either surgery or interventional radiology occurred in 14 patients-G/M (n = 5); CMV viremia was seen in 11 patients (G/M, n = 1); detectable EBV DNA was detectable in 8 patients (G/M, n = 4), acute cellular rejection was seen in 10 (G/M, n = 5), postransplant lymphoproliferative disease occurred in 2 G/M patients; and 1 G/M patient showed significantly improved posttransplant neurologic motor function. Children with G/M who received a living donor liver transplant from heterozygote parents did well without any signs of expressing underlying metabolic disease. Posttransplant graft and patient overall survival at 12 months for G/M and non-G/M was 100%, and at 36 months, 83% and 100%, respectively.

Conclusion: The majority of children transplanted for either genetic or metabolic disease were status 1 or awarded UNOS exception points. Cadaveric split livers and live donors including obligate heterozygotes resulted in excellent allograft and patient survival outcomes. In metabolic and genetic liver diseases, close follow-up and timely transplantation can preclude malignant spread and prevent disease progression and consequences, as well as reverse neurologic sequelae.