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Odevixibat Treatment of Alagille Syndrome: A Case Report

A male pediatric patient with elevated liver enzyme and bile acid levels, bile duct hypoplasia, mild liver fibrosis, and pruritus was initially diagnosed with progressive familial intrahepatic cholestasis. The patient did not respond to treatments of ursodeoxycholic acid and naltrexone. Subsequent t...

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Detalles Bibliográficos
Autores principales: Ganschow, Rainer, Maucksch, Christof
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins, Inc. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10187842/
https://www.ncbi.nlm.nih.gov/pubmed/37200711
http://dx.doi.org/10.1097/PG9.0000000000000301
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author Ganschow, Rainer
Maucksch, Christof
author_facet Ganschow, Rainer
Maucksch, Christof
author_sort Ganschow, Rainer
collection PubMed
description A male pediatric patient with elevated liver enzyme and bile acid levels, bile duct hypoplasia, mild liver fibrosis, and pruritus was initially diagnosed with progressive familial intrahepatic cholestasis. The patient did not respond to treatments of ursodeoxycholic acid and naltrexone. Subsequent treatment with odevixibat resulted in improvements in serum bile acid levels and pruritus within a few weeks of initiation. During the course of odevixibat treatment, genetic testing results and additional clinical findings indicated a diagnosis of Alagille syndrome, a condition that shares some clinical features with progressive familial intrahepatic cholestasis. Odevixibat treatment was continued off label, during which time the patient’s serum bile acid levels dropped to within the normal limit and pruritus was completely ameliorated. This report suggests odevixibat may be an effective treatment option for Alagille syndrome.
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spelling pubmed-101878422023-05-17 Odevixibat Treatment of Alagille Syndrome: A Case Report Ganschow, Rainer Maucksch, Christof JPGN Rep Case Report A male pediatric patient with elevated liver enzyme and bile acid levels, bile duct hypoplasia, mild liver fibrosis, and pruritus was initially diagnosed with progressive familial intrahepatic cholestasis. The patient did not respond to treatments of ursodeoxycholic acid and naltrexone. Subsequent treatment with odevixibat resulted in improvements in serum bile acid levels and pruritus within a few weeks of initiation. During the course of odevixibat treatment, genetic testing results and additional clinical findings indicated a diagnosis of Alagille syndrome, a condition that shares some clinical features with progressive familial intrahepatic cholestasis. Odevixibat treatment was continued off label, during which time the patient’s serum bile acid levels dropped to within the normal limit and pruritus was completely ameliorated. This report suggests odevixibat may be an effective treatment option for Alagille syndrome. Lippincott Williams & Wilkins, Inc. 2023-03-24 /pmc/articles/PMC10187842/ /pubmed/37200711 http://dx.doi.org/10.1097/PG9.0000000000000301 Text en Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition. https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the Creative Commons Attribution License 4.0 (CCBY (https://creativecommons.org/licenses/by/4.0/) ), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Case Report
Ganschow, Rainer
Maucksch, Christof
Odevixibat Treatment of Alagille Syndrome: A Case Report
title Odevixibat Treatment of Alagille Syndrome: A Case Report
title_full Odevixibat Treatment of Alagille Syndrome: A Case Report
title_fullStr Odevixibat Treatment of Alagille Syndrome: A Case Report
title_full_unstemmed Odevixibat Treatment of Alagille Syndrome: A Case Report
title_short Odevixibat Treatment of Alagille Syndrome: A Case Report
title_sort odevixibat treatment of alagille syndrome: a case report
topic Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10187842/
https://www.ncbi.nlm.nih.gov/pubmed/37200711
http://dx.doi.org/10.1097/PG9.0000000000000301
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