ORTHOTOPIC LIVER-TRANSPLANTATION FOR CRIGLER-NAJJAR TYPE-I DISEASE IN6 CHILDREN

Hyperbilirubinemia in Crigler-Najjar disease type I (CN) can be partia lly controlled by daily phototherapy, but these children remain at per manent risk of developing brain damage due to kernicterus, Because liv er transplantation is the only available curative treatment for liver- based inborn errors of metabolism, orthotopic liver transplantation (O LT) was performed in six patients with CN, Mean age at surgery was 52. 5 months (range 27 to 100), Despite a mean daily phototherapy of 12.4/-0.8 hr, mean bilirubin of the 6 patients was 388 mu M/L (range 175 t o 703) before OLT; one of them was also being treated with tin-protopo rphyrin. All 6 had elevated AST/ALT, ranging from 1.4 to 6 times upper normal values, Complications occurred in three patients after OLT, in cluding miliary tuberculosis in one, graft rejection and retransplanta tion in one, and hepatic artery thrombosis in one, All patients surviv e with normal serum bilirubin level (follow up 6 to 116 months). Four have normal enzymes on post-OLT follow-up (30 to 95 months), follow a normal education program, and have a normal social life. One recently transplanted patient has progressively normalizing liver function test s 6 months after OLT, One patient transplanted at 8 y.o. (now 116 mont hs post-OLT) has moderate neurological delay due to pretransplant kern icterus, and posttransplant chronic persistent hepatitis, Our series s hows that OLT cures hyperbilirubinemia in CN patients, with an excelle nt survival prospect, The procedure should be decided upon before neur ological sequelae occur, since these persist after transplantation.