OBJECTIVES: The aim of this study was to examine whether reversion of histological fibrosis followed partial external biliary diversion (PEBD) in patients with progressive familial intrahepatic cholestasis (PFIC); whether the duration of cholestatic episodes after PEBD influenced the evolution of fibrosis; and whether genotyping was helpful in predicting outcome of PEBD. PATIENTS AND METHODS: Children with PFIC who underwent PEBD were investigated with genetic, biochemical, and anthropometric standard methods. Serial liver specimens were assessed histologically without knowledge of genotype and outcome. Findings were evaluated in the contexts of the total duration of cholestasis and the clinical outcome after PEBD. RESULTS: From a total of 18 children with PFIC, 13 underwent PEBD, and 12 of these (among them 10 with identified ABCB11 mutations) were amenable for clinical and histological follow-up. When compared with baseline at PEBD, statistically significant reductions were found in histological cholestasis 1 and 3 years after PEBD, and in fibrosis 5 and >10 years after PEBD. The relative duration of cholestatic episodes after PEBD was positively correlated with the severity of fibrosis. Children homozygous for the missense mutation c.890A>G in ABCB11 responded well to PEBD. CONCLUSIONS: Biliary diversion should be regarded as the first choice of surgical treatment in noncirrhotic patients with severe ABCB11 disease and may also be efficacious in other forms of PFIC.
OBJECTIVES: The aim of this study was to examine whether reversion of histological fibrosis followed partial external biliary diversion (PEBD) in patients with progressive familial intrahepatic cholestasis (PFIC); whether the duration of cholestatic episodes after PEBD influenced the evolution of fibrosis; and whether genotyping was helpful in predicting outcome of PEBD. PATIENTS AND METHODS: Children with PFIC who underwent PEBD were investigated with genetic, biochemical, and anthropometric standard methods. Serial liver specimens were assessed histologically without knowledge of genotype and outcome. Findings were evaluated in the contexts of the total duration of cholestasis and the clinical outcome after PEBD. RESULTS: From a total of 18 children with PFIC, 13 underwent PEBD, and 12 of these (among them 10 with identified ABCB11 mutations) were amenable for clinical and histological follow-up. When compared with baseline at PEBD, statistically significant reductions were found in histological cholestasis 1 and 3 years after PEBD, and in fibrosis 5 and >10 years after PEBD. The relative duration of cholestatic episodes after PEBD was positively correlated with the severity of fibrosis. Children homozygous for the missense mutation c.890A>G in ABCB11 responded well to PEBD. CONCLUSIONS: Biliary diversion should be regarded as the first choice of surgical treatment in noncirrhoticpatients with severe ABCB11 disease and may also be efficacious in other forms of PFIC.
Authors: Hilary S Jericho; Elizabeth Kaurs; Renze Boverhof; Alex Knisely; Benjamin L Shneider; Henkjan J Verkade; Peter F Whitington Journal: J Pediatr Gastroenterol Nutr Date: 2015-03 Impact factor: 2.839
Authors: Kasper S Wang; Greg Tiao; Lee M Bass; Paula M Hertel; Douglas Mogul; Nanda Kerkar; Matthew Clifton; Colleen Azen; Laura Bull; Philip Rosenthal; Dylan Stewart; Riccardo Superina; Ronen Arnon; Molly Bozic; Mary L Brandt; Patrick A Dillon; Annie Fecteau; Kishore Iyer; Binita Kamath; Saul Karpen; Frederick Karrer; Kathleen M Loomes; Cara Mack; Peter Mattei; Alexander Miethke; Kyle Soltys; Yumirle P Turmelle; Karen West; Jessica Zagory; Cat Goodhue; Benjamin L Shneider Journal: Hepatology Date: 2017-03-22 Impact factor: 17.425
Authors: Paula M Hertel; Laura N Bull; Richard J Thompson; Nathan P Goodrich; Wen Ye; John C Magee; Robert H Squires; Lee M Bass; James E Heubi; Grace E Kim; Sarangarajan Ranganathan; Kathleen B Schwarz; Molly A Bozic; Simon P Horslen; Matthew S Clifton; Yumirle P Turmelle; Frederick J Suchy; Riccardo A Superina; Kasper S Wang; Kathleen M Loomes; Binita M Kamath; Ronald J Sokol; Benjamin L Shneider Journal: J Pediatr Gastroenterol Nutr Date: 2021-08-01 Impact factor: 3.288