Liver transplantation in children with progressive familial intrahepatic cholestasis.

摘要: Progressive familial intrahepatic cholestasis (PFIC) is caused by mutations of the bile salt export pump or multidrug resistance P-glycoprotein, resulting in chronic hepatic failure. Partial external diversion ileal bypass effective some cases and, others, liver transplantation (OLT) necessary. Forty-two children were included this study. Twenty-six suffered from PFIC type 2 and 16 3. Symptoms pruritus, cholestasis, cirrhosis, growth retardation. Seventeen patients received biliary diversion. Ten had to undergo OLT following course. As report, three remaining on wait list for OLT. Twenty-three a graft primarily with excellent outcome. Our data show that option choice symptomatic whenever cirrhosis present. We suggest very restrictive recommendation However, gene therapy may be future PFIC.