Mkarem LE, Batika MAH, Bitar R. New hope in treating progressive familial intrahepatic cholestasis in children. World J Hepatol 2025; 17(7): 108253 [DOI: 10.4254/wjh.v17.i7.108253]
Corresponding Author of This Article
Rana Bitar, Assistant Professor, Division of Pediatric Gastroenterology, Sheikh Khalifa Medical City, Al Karamah St., Abu Dhabi 51900, United Arab Emirates. rahmad@seha.ae
Research Domain of This Article
Gastroenterology & Hepatology
Article-Type of This Article
Minireviews
Open-Access Policy of This Article
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. See: http://creativecommons.org/licenses/by-nc/4.0/
World J Hepatol. Jul 27, 2025; 17(7): 108253 Published online Jul 27, 2025. doi: 10.4254/wjh.v17.i7.108253
New hope in treating progressive familial intrahepatic cholestasis in children
Lama Ebrahim Mkarem, Mohammed Ali Hosny Batika, Rana Bitar
Lama Ebrahim Mkarem, Mohammed Ali Hosny Batika, Department of Paediatrics, Sheikh Khalifa Medical City, Abu Dhabi 767451, United Arab Emirates
Rana Bitar, Division of Pediatric Gastroenterology, Sheikh Khalifa Medical City, Abu Dhabi 51900, United Arab Emirates
Rana Bitar, College of Medicine, Khalifa University, Abu Dhabi 767451, United Arab Emirates
Author contributions: Bitar R identified lack of published reviews on PFIC new treatments and initiated the study idea and design; All authors reviewed published manuscripts and wrote part of the manuscript.
Conflict-of-interest statement: There is no conflict of interest to declare.
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (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. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Rana Bitar, Assistant Professor, Division of Pediatric Gastroenterology, Sheikh Khalifa Medical City, Al Karamah St., Abu Dhabi 51900, United Arab Emirates. rahmad@seha.ae
Received: April 9, 2025 Revised: April 25, 2025 Accepted: June 23, 2025 Published online: July 27, 2025 Processing time: 107 Days and 21.6 Hours
Core Tip
Core Tip: Progressive familial intrahepatic cholestasis is a heterogenous autosomal recessive progressive cholestatic liver disease with varying genotype and phenotype. Identifying the underlying genetic disorder allows better understanding of the clinical phenotype and associated complications. Management is aimed at nutritional support, trying to make bile more hydrophilic by administering ursodeoxycholic acid and control of pruritus. Surgical biliary diversion has proved to reduce bile acid levels and improve pruritis in some patients. New hope exists with ileal bile acid transporter receptor inhibitors, however more data is still required. The ultimate treatment for end stage liver disease and persistent pruritus is liver transplantation.
