Al Atrash E, Bitar R. Odevixibat improves pruritus and bile acid level in Alagille syndrome: A case report. World J Gastrointest Pharmacol Ther 2025; 16(3): 108257 [DOI: 10.4292/wjgpt.v16.i3.108257]
Corresponding Author of This Article
Rana Bitar, Assistant Professor, Division of Pediatric Gastroenterology, Sheikh Khalifa Medical City, Al Karamah Street, Abu Dhabi 51900, United Arab Emirates. rahmad@seha.ae
Research Domain of This Article
Gastroenterology & Hepatology
Article-Type of This Article
Case Report
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 Gastrointest Pharmacol Ther. Sep 5, 2025; 16(3): 108257 Published online Sep 5, 2025. doi: 10.4292/wjgpt.v16.i3.108257
Odevixibat improves pruritus and bile acid level in Alagille syndrome: A case report
Eman Al Atrash, Rana Bitar
Eman Al Atrash, Rana Bitar, Division of Pediatric Gastroenterology, Sheikh Khalifa Medical City, Abu Dhabi 51900, United Arab Emirates
Author contributions: Al Atrash E and Bitar R contributed to the review of patient notes, writing up the case report, revision of case report.
Informed consent statement: Informed written consent was obtained from the patient for publication of this report and any accompanying images.
Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article.
CARE Checklist (2016) statement: The authors have read the CARE Checklist (2016), and the manuscript was prepared and revised according to the CARE Checklist (2016).
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 Street, Abu Dhabi 51900, United Arab Emirates. rahmad@seha.ae
Received: April 9, 2025 Revised: May 6, 2025 Accepted: July 17, 2025 Published online: September 5, 2025 Processing time: 148 Days and 10.5 Hours
Abstract
BACKGROUND
Alagille syndrome (ALGS) is a rare genetic disorder that affects the liver causing, cholestasis, jaundice and intractable pruritus which can significantly impair health-related quality of life (QoL). The treatment of liver involvement is mainly supportive with the majority eventually requiring liver transplant. Ileal bile acid transporter inhibition is a novel therapeutic concept for cholestatic pruritus and cholestatic liver disease. We report the effects of odevixibat treatment in a patient ALGS over 12 months.
CASE SUMMARY
A male patient presented with jaundice at 3 months. ALGS was suspected clinically and genetic testing identified JAG1 mutation. Mother reported severe pruritus since the age of 18 months disrupting the child’s daily activity including sleeping. He was treated with ursodiol at 10 mg/kg three times a day, rifampin at 5 mg/kg twice daily, cholestyramine at 4 g twice daily and cetirizine 5 mg once daily. The patient continued to have persistent itching affecting the QoL, with abnormal liver enzymes and bile acid level. The patient pediatric end-stage liver disease score was 15. Abdominal ultrasound (June 2023) showed enlarged coarse liver, normal portal vein flow and massive splenomegaly measuring 14 cm in sagittal span (spleen size was 11.2 cm on previous scan 6 months earlier). Odevixibat was administered to the patient at initial dose 40 μg/kg/day increasing up to 120 μg/kg/day to ameliorate gastrointestinal side effects. The patient itching improved very shortly after initiating treatment and was able to sleep overnight for the very first time. The patients’ serum bile acids, bilirubin and alanine transferase improved up to 12 months after treatment. There was no reported side effect.
CONCLUSION
This case demonstrates the use of ileal bile acid transporter inhibitors treating pruritus and improving patient QoL. It has potential to delay the need for liver transplantation. However more long-term studies are needed to help clinicians better understand the benefit of this new treatment.
Core Tip: Alagille syndrome is a rare genetic disorder that affects the liver causing, cholestasis, jaundice and intractable pruritus which can significantly impair health-related quality of life. The treatment is mainly supportive with majority eventually requiring liver transplant. Ileal bile acid transporter inhibition is a novel therapeutic concept for cholestatic pruritus and cholestatic liver disease. We report the effects of odevixibat treatment in a patient Alagille syndrome over 12 months. This case demonstrates the use of ileal bile acid transporter inhibitors treating pruritus and improving patient quality of life and biochemical parameters. This allowed for the delay in the need for liver transplantation. It has potential to delay the need for liver transplantation. However more long-term studies are needed to help clinicians better understand the benefit of this new treatment.
