Ursodeoxycholic acid therapy in pediatric patients with progressive familial intrahepatic cholestasis
E Jacquemin, D Hermans, A Myara, D Habes, D Debray, M Hadchouel, E M Sokal, O Bernard – 30 December 2003 – Progressive familial intrahepatic cholestasis (PFIC) is a lethal inherited childhood cholestasis of hepatocellular origin. Different subtypes of PFIC have been described according to serum gamma‐glutamyl transpeptidase (GGT) activity. There is currently no effective medical therapy available for children with PFIC. We report on 39 patients with PFIC who received ursodeoxycholic acid (UDCA) orally (20‐30 mg/kg b.w./day) for a period of 2 to 4 years.