Nontransplant surgical interventions in progressive familial intrahepatic cholestasis

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. (C) 2009 Elsevier Inc. All rights reserved.