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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...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Lippincott Williams & Wilkins, Inc.
2023
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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. |
format | Online Article Text |
id | pubmed-10187842 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Lippincott Williams & Wilkins, Inc. |
record_format | MEDLINE/PubMed |
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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