Ursodeoxycholic acid therapy in pediatric patients with progressive familial intrahepatic cholestasis

摘要: Progressive familial intrahepatic cholestasis (PFIC) is a lethal inherited childhood of hepatocellular origin. Different subtypes PFIC have been described according to serum gamma-glutamyl transpeptidase (GGT) activity. There currently no effective medical therapy available for children with PFIC. We report on 39 patients who received ursodeoxycholic acid (UDCA) orally (20-30 mg/kg b.w./day) period 2 4 years. Group 1 (n = 26) consisted normal GGT activity, and group 13) high Within 1, liver tests normalized in 11 children, improved 5, stabilized or worsened 10. 2, six four, three. Improvement parameters was associated an enrichment the circulating pool bile acids UDCA. Hepatosplenomegaly pruritus disappeared diminished whom normalized. In nine these again after stopping restarting Liver histology assessed four normalization years treatment showed decrease fibrosis. conclude that UDCA should be considered initial therapeutic management PFIC, because it appears resolving improving function clinical status fair proportion children. Chronic might thus avoid need transplantation some