Adam Rahn Davis1, Philip Rosenthal, Thomas B Newman. 1. Division of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, University of California, San Francisco, CA 94143, USA. davisa@peds.ucsf.edu
Abstract
BACKGROUND: Progressive familial intrahepatic cholestasis (PFIC) is a family of rare childhood diseases that was universally fatal until the development of liver transplant. In the last 20 years, the use of nontransplant surgery to treat PFIC has become the standard of care. There are various surgical techniques that have been performed. There are no reviews evaluating the outcome of these operations. METHODS: A systematic search of the literature for articles evaluating the outcome of nontransplant surgical interventions in PFIC patients was performed. Data from these studies was abstracted and summarized. RESULTS: No trials have been performed addressing nontransplant surgical interventions in PFIC patients. We analyzed 11 case series and case reports. Generally, patients had successful outcomes (81%) with cessation of progression of disease and resolution of symptoms. Treatment failures were often associated with more advanced disease. DISCUSSION: There is no evidence to demonstrate a superiority of one type of nontransplant surgical intervention in PFIC patients. We propose the development of a registry and standardization of outcomes measurements to allow improved comparison of results.
BACKGROUND: Progressive familial intrahepatic cholestasis (PFIC) is a family of rare childhood diseases that was universally fatal until the development of liver transplant. In the last 20 years, the use of nontransplant surgery to treat PFIC has become the standard of care. There are various surgical techniques that have been performed. There are no reviews evaluating the outcome of these operations. METHODS: A systematic search of the literature for articles evaluating the outcome of nontransplant surgical interventions in PFICpatients was performed. Data from these studies was abstracted and summarized. RESULTS: No trials have been performed addressing nontransplant surgical interventions in PFICpatients. We analyzed 11 case series and case reports. Generally, patients had successful outcomes (81%) with cessation of progression of disease and resolution of symptoms. Treatment failures were often associated with more advanced disease. DISCUSSION: There is no evidence to demonstrate a superiority of one type of nontransplant surgical intervention in PFICpatients. We propose the development of a registry and standardization of outcomes measurements to allow improved comparison of results.
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