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Current and future therapies for inherited cholestatic liver diseases
Familial intrahepatic cholestasis (FIC) comprises a group of rare cholestatic liver diseases associated with canalicular transport defects resulting predominantly from mutations in ATP8B1, ABCB11 and ABCB4. Phenotypes range from benign recurrent intrahepatic cholestasis (BRIC), associated with recur...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Baishideng Publishing Group Inc
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5296193/ https://www.ncbi.nlm.nih.gov/pubmed/28223721 http://dx.doi.org/10.3748/wjg.v23.i5.763 |
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author | van der Woerd, Wendy L Houwen, Roderick HJ van de Graaf, Stan FJ |
author_facet | van der Woerd, Wendy L Houwen, Roderick HJ van de Graaf, Stan FJ |
author_sort | van der Woerd, Wendy L |
collection | PubMed |
description | Familial intrahepatic cholestasis (FIC) comprises a group of rare cholestatic liver diseases associated with canalicular transport defects resulting predominantly from mutations in ATP8B1, ABCB11 and ABCB4. Phenotypes range from benign recurrent intrahepatic cholestasis (BRIC), associated with recurrent cholestatic attacks, to progressive FIC (PFIC). Patients often suffer from severe pruritus and eventually progressive cholestasis results in liver failure. Currently, first-line treatment includes ursodeoxycholic acid in patients with ABCB4 deficiency (PFIC3) and partial biliary diversion in patients with ATP8B1 or ABCB11 deficiency (PFIC1 and PFIC2). When treatment fails, liver transplantation is needed which is associated with complications like rejection, post-transplant hepatic steatosis and recurrence of disease. Therefore, the need for more and better therapies for this group of chronic diseases remains. Here, we discuss new symptomatic treatment options like total biliary diversion, pharmacological diversion of bile acids and hepatocyte transplantation. Furthermore, we focus on emerging mutation-targeted therapeutic strategies, providing an outlook for future personalized treatment for inherited cholestatic liver diseases. |
format | Online Article Text |
id | pubmed-5296193 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2017 |
publisher | Baishideng Publishing Group Inc |
record_format | MEDLINE/PubMed |
spelling | pubmed-52961932017-02-21 Current and future therapies for inherited cholestatic liver diseases van der Woerd, Wendy L Houwen, Roderick HJ van de Graaf, Stan FJ World J Gastroenterol Review Familial intrahepatic cholestasis (FIC) comprises a group of rare cholestatic liver diseases associated with canalicular transport defects resulting predominantly from mutations in ATP8B1, ABCB11 and ABCB4. Phenotypes range from benign recurrent intrahepatic cholestasis (BRIC), associated with recurrent cholestatic attacks, to progressive FIC (PFIC). Patients often suffer from severe pruritus and eventually progressive cholestasis results in liver failure. Currently, first-line treatment includes ursodeoxycholic acid in patients with ABCB4 deficiency (PFIC3) and partial biliary diversion in patients with ATP8B1 or ABCB11 deficiency (PFIC1 and PFIC2). When treatment fails, liver transplantation is needed which is associated with complications like rejection, post-transplant hepatic steatosis and recurrence of disease. Therefore, the need for more and better therapies for this group of chronic diseases remains. Here, we discuss new symptomatic treatment options like total biliary diversion, pharmacological diversion of bile acids and hepatocyte transplantation. Furthermore, we focus on emerging mutation-targeted therapeutic strategies, providing an outlook for future personalized treatment for inherited cholestatic liver diseases. Baishideng Publishing Group Inc 2017-02-07 2017-02-07 /pmc/articles/PMC5296193/ /pubmed/28223721 http://dx.doi.org/10.3748/wjg.v23.i5.763 Text en ©The Author(s) 2017. Published by Baishideng Publishing Group Inc. All rights reserved. http://creativecommons.org/licenses/by-nc/4.0/ This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. |
spellingShingle | Review van der Woerd, Wendy L Houwen, Roderick HJ van de Graaf, Stan FJ Current and future therapies for inherited cholestatic liver diseases |
title | Current and future therapies for inherited cholestatic liver diseases |
title_full | Current and future therapies for inherited cholestatic liver diseases |
title_fullStr | Current and future therapies for inherited cholestatic liver diseases |
title_full_unstemmed | Current and future therapies for inherited cholestatic liver diseases |
title_short | Current and future therapies for inherited cholestatic liver diseases |
title_sort | current and future therapies for inherited cholestatic liver diseases |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5296193/ https://www.ncbi.nlm.nih.gov/pubmed/28223721 http://dx.doi.org/10.3748/wjg.v23.i5.763 |
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