AIMS: Progressive familial intrahepatic cholestasis (PFIC, Byler's disease) is an autosomal recessive disorder resulting in liver fibrosis/cirrhosis and liver insufficiency. Before the 1990s, liver transplantation was the only effective therapy for these children. During the last 12 years, two alternative methods of surgical treatment have been proposed: partial external biliary diversion (PEBD) and ileal bypass procedure (IB), which allow for effective elimination of bile acids accumulated in the body. In this study, we compare the efficacy of these surgical techniques for PFIC. METHODS: During the last 20 years, we have treated 52 children with PFIC. PEBD was done in 21 patients (since 1995), and IB in 5 patients (since 1998), transplantation was performed in 9 patients (since 1990). The efficacy of non-transplantation surgical treatment was assessed by patients' clinical outcome, liver biochemistry, and survival without transplantation during a follow-up period of 12 to 48 months. RESULTS: In 15 out of 21 patients clinical symptoms improved after PEBD and liver function tests normalised (blood bile acids), 1 patient had to be converted to IB due to too high output biliary fistula, 2 patients were transplanted and 3 are considered for transplantation. Out of the 5 children after IB, 4 improved clinically and biochemically, but, after 12 months, symptoms recurred in 3 patients, one patient was converted successfully to PEBD. No significant influence on growth was observed, irrespective of the type of treatment in this group of patients. CONCLUSIONS: PEBD is more effective than IB for the permanent improvement of symptoms of PFIC. Ileal bypass procedure, although initially effective, does not ensure good long-term results in more than 50 % of patients, probably due to intestinal re-absorption of bile acids increasing over time.
AIMS: Progressive familial intrahepatic cholestasis (PFIC, Byler's disease) is an autosomal recessive disorder resulting in liver fibrosis/cirrhosis and liver insufficiency. Before the 1990s, liver transplantation was the only effective therapy for these children. During the last 12 years, two alternative methods of surgical treatment have been proposed: partial external biliary diversion (PEBD) and ileal bypass procedure (IB), which allow for effective elimination of bile acids accumulated in the body. In this study, we compare the efficacy of these surgical techniques for PFIC. METHODS: During the last 20 years, we have treated 52 children with PFIC. PEBD was done in 21 patients (since 1995), and IB in 5 patients (since 1998), transplantation was performed in 9 patients (since 1990). The efficacy of non-transplantation surgical treatment was assessed by patients' clinical outcome, liver biochemistry, and survival without transplantation during a follow-up period of 12 to 48 months. RESULTS: In 15 out of 21 patients clinical symptoms improved after PEBD and liver function tests normalised (blood bile acids), 1 patient had to be converted to IB due to too high output biliary fistula, 2 patients were transplanted and 3 are considered for transplantation. Out of the 5 children after IB, 4 improved clinically and biochemically, but, after 12 months, symptoms recurred in 3 patients, one patient was converted successfully to PEBD. No significant influence on growth was observed, irrespective of the type of treatment in this group of patients. CONCLUSIONS:PEBD is more effective than IB for the permanent improvement of symptoms of PFIC. Ileal bypass procedure, although initially effective, does not ensure good long-term results in more than 50 % of patients, probably due to intestinal re-absorption of bile acids increasing over time.
Authors: Ludmila Pawlikowska; Sandra Strautnieks; Irena Jankowska; Piotr Czubkowski; Karan Emerick; Anthony Antoniou; Catherine Wanty; Bjorn Fischler; Emmanuel Jacquemin; Sami Wali; Samra Blanchard; Inge-Merete Nielsen; Billy Bourke; Shirley McQuaid; Florence Lacaille; Jane A Byrne; Albertien M van Eerde; Kaija-Leena Kolho; Leo Klomp; Roderick Houwen; Peter Bacchetti; Steven Lobritto; Vera Hupertz; Patricia McClean; Giorgina Mieli-Vergani; Benjamin Shneider; Antal Nemeth; Etienne Sokal; Nelson B Freimer; A S Knisely; Philip Rosenthal; Peter F Whitington; Joanna Pawlowska; Richard J Thompson; Laura N Bull Journal: J Hepatol Date: 2010-04-13 Impact factor: 25.083
Authors: P Ramachandran; N P Shanmugam; S Al Sinani; V Shanmugam; S Srinivas; M Sathiyasekaran; V Tamilvanan; M Rela Journal: Pediatr Surg Int Date: 2014-07-27 Impact factor: 1.827
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