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Comment on opinion paper on the diagnosis and treatment of progressive familial intrahepatic cholestasis.

作者信息

Gonzales Emmanuel, Thompson Richard J, Jacquemin Emmanuel

机构信息

Pediatric Hepatology and Liver Transplantation Unit, National Reference Centre for Inflammatory Diseases of the Bile Ducts and Autoimmune Hepatitis, FILFOIE, ERN RARE LIVER, Bicêtre Hospital, Assistance Publique - Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France.

INSERM UMR 1193, Hepatinov, Paris-Saclay University, Orsay, France.

出版信息

JHEP Rep. 2025 Feb 19;7(6):101361. doi: 10.1016/j.jhepr.2025.101361. eCollection 2025 Jun.

Abstract
摘要

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本文引用的文献

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Odevixibat as an adjunctive treatment for refractory pruritus in rare variants of cholestatic liver disease.
JPGN Rep. 2024 Apr 4;5(3):296-302. doi: 10.1002/jpr3.12069. eCollection 2024 Aug.
2
EASL Clinical Practice Guidelines on genetic cholestatic liver diseases.
J Hepatol. 2024 Aug;81(2):303-325. doi: 10.1016/j.jhep.2024.04.006. Epub 2024 Jun 8.
3
Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Lancet Gastroenterol Hepatol. 2024 Jul;9(7):620-631. doi: 10.1016/S2468-1253(24)00080-3. Epub 2024 May 6.
4
Clinical outcomes of ABCB4 heterozygosity in infants and children with cholestatic liver disease.
J Pediatr Gastroenterol Nutr. 2024 Feb;78(2):339-349. doi: 10.1002/jpn3.12080. Epub 2023 Dec 12.
5
Opinion paper on the diagnosis and treatment of progressive familial intrahepatic cholestasis.
JHEP Rep. 2023 Oct 27;6(1):100949. doi: 10.1016/j.jhepr.2023.100949. eCollection 2024 Jan.
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Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy.
JHEP Rep. 2023 Jul 13;5(10):100844. doi: 10.1016/j.jhepr.2023.100844. eCollection 2023 Oct.
7
Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial.
Lancet Gastroenterol Hepatol. 2022 Sep;7(9):830-842. doi: 10.1016/S2468-1253(22)00093-0. Epub 2022 Jul 1.
8
The spectrum of liver diseases related to ABCB4 gene mutations: pathophysiology and clinical aspects.
Semin Liver Dis. 2010 May;30(2):134-46. doi: 10.1055/s-0030-1253223. Epub 2010 Apr 26.
9
The wide spectrum of multidrug resistance 3 deficiency: from neonatal cholestasis to cirrhosis of adulthood.
Gastroenterology. 2001 May;120(6):1448-58. doi: 10.1053/gast.2001.23984.

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