EVALUATION OF TYROSINEMIC CHILDREN FOR LIVER TRANSPLANTATION

Abstract

We evaluated 19 patients (8/12-16 8/12 years) with Tyrosinemia for possible liver transplantation. Ten children had repeated neurological crises (severe paresthesias, automutilation, paralysis), and 12 had evidence of renal disease as assessed by a GFR < 60 (4), Fanconi-like syndrome (5), nephromegaly with or without nephrocalcinosis (10). The following liver tests were abnormal; AST/ALT (12), Alk. Phos. (8), bilirubin (5), γ GT (13), PT (11), PTT (7), albumin (6). Portal hypertension was present in 8 patients. Nodules were present on liver imaging in 10 children, with α fetoprotein (AFP) levels ranging from normal to 33000 ng/ml. Of the 8 whole livers available for histological examination, 2 contained foci of hepatocarcinoma. No relationship between AFP, hepatocarc inoma, or the presence of nodules could be found. Five children had a liver transplant, and 2 a combined liver-kidney transplant (one of the two died from primary non-function of the liver graft). The 6 remaining transplanted patients are alive and thriving with normal liver tests, and none have had recurrence of neurological crises. Two patients died while on the waiting list and 3 during severe neurological crises. Liver transplantation cures the hepatic metabolic disease, protects from the development of hepatocarcinoma, and prevents further neurological crises. Combined liver-kidney transplantation should be considered in children with low CFR's in light of the long-term effects of CSA.

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