CASE REPORT article
Front. Pediatr.
Sec. Pediatric Gastroenterology, Hepatology and Nutrition
Real-World Use of Maralixibat in Biliary Atresia: A Case Series
- ND
Natasha Dilwali 1,2
- DB
Douglas B. Mogul 3
- SM
Shannon M. Vandriel 3
- TT
Tony Tokman 3
- CA
Catherine A. Chapin 4
- BR
Bertrand Roquelaure 5
- RR
Rossitsa Rousseva 6
- JF
Johanna Ferreira 7,8
- MM
Mercedes Martinez 2
- BK
Benno Kohlmaier 9
1. Johns Hopkins University, Baltimore, United States
2. Columbia University Irving Medical Center, New York, NY, United States
3. Mirum Pharmaceuticals Inc, Foster City, United States
4. Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, United States
5. APHM, Timone Enfants, Service de pédiatrie multidisciplinaire, Marseille, France, Marseille, France
6. King's College Hospital, London, United Kingdom
7. Cohen Children's Medical Center, New York, United States
8. University of Miami Miller School of Medicine, Miami, United States
9. Medizinische Universitat Graz, Graz, Austria
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Abstract
Biliary atresia (BA) is the most common cause of cholestasis in infants, and pruritus may be a prominent and debilitating complication. Maralixibat is the first US Food and Drug Administration–approved drug for the treatment of cholestatic pruritus in children with Alagille syndrome and has been subsequently approved for treatment of progressive familial intrahepatic cholestasis as well. Several patients with BA have received maralixibat as part of a compassionate use program. We report the use of maralixibat for the treatment of cholestatic pruritus in BA for six patients. Demographics, past medical history, laboratory markers, and medications were collected. Pruritus was assessed using the Clinician Scratch Scale (CSS) at baseline and last clinical follow-up. All patients reported improved CSS with no increased usage of other antipruritic medications, and the medication was well tolerated.
Summary
Keywords
case report, Cholestasis, IBAT inhibitor, liver transplant, Pruritus
Received
17 April 2026
Accepted
07 July 2026
Copyright
© 2026 Dilwali, Mogul, Vandriel, Tokman, Chapin, Roquelaure, Rousseva, Ferreira, Martinez and Kohlmaier. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
*Correspondence: Natasha Dilwali
Disclaimer
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