Current and future therapies for inherited cholestatic liver diseases
| Autor: | Stan F.J. van de Graaf, Wendy L. van der Woerd, Roderick H. J. Houwen |
|---|---|
| Rok vydání: | 2017 |
| Předmět: |
0301 basic medicine
medicine.medical_treatment Benign Recurrent Intrahepatic Cholestasis Familial intrahepatic cholestasis Review Disease Liver transplantation Gastroenterology 0302 clinical medicine Molecular Targeted Therapy Precision Medicine ABCB11 ATP Binding Cassette Transporter Subfamily B Member 11 Adenosine Triphosphatases Intrahepatic Cholestasis Progressive familial intrahepatic cholestasis ABCB4 General Medicine Ursodeoxycholic acid Personalized treatment 030211 gastroenterology & hepatology medicine.drug medicine.medical_specialty ATP Binding Cassette Transporter Subfamily B Biliary diversion Cholestasis Intrahepatic Mutation-targeted therapy Bile Acids and Salts 03 medical and health sciences ATP8B1 Internal medicine Journal Article medicine Humans P-Glycoproteins business.industry medicine.disease Liver Transplantation 030104 developmental biology Mutation ATP-Binding Cassette Transporters business Inherited liver disease |
| Zdroj: | World Journal of Gastroenterology World Journal of Gastroenterology, 23(5), 763. WJG Press |
| ISSN: | 1007-9327 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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