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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.

DOI:10.1016/j.jhepr.2025.101361
PMID:40535556
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12174988/
Abstract
摘要

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Comment on opinion paper on the diagnosis and treatment of progressive familial intrahepatic cholestasis.关于进行性家族性肝内胆汁淤积症诊断与治疗的观点论文评论
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Reply to: "Comment on opinion paper on the diagnosis and treatment of progressive familial intrahepatic cholestasis".回复：“关于进行性家族性肝内胆汁淤积症诊断与治疗的观点论文评论”
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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.odevixibat作为胆汁淤积性肝病罕见变异型难治性瘙痒的辅助治疗方法。
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.EASL 临床实践指南：遗传性胆汁淤积性肝病。
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.马拉利昔巴特治疗进行性家族性肝内胆汁淤积症（MARCH-PFIC）：一项多中心、随机、双盲、安慰剂对照的 3 期临床试验。
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.ABCB4 杂合性在婴儿和儿童胆汁淤积性肝病中的临床结局。
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.关于进行性家族性肝内胆汁淤积症诊断与治疗的意见书
JHEP Rep. 2023 Oct 27;6(1):100949. doi: 10.1016/j.jhepr.2023.100949. eCollection 2024 Jan.
6
Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy.38例进行性家族性肝内胆汁淤积症3型患者的预后：基因型及对熊去氧胆酸治疗反应的影响
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.奥贝胆酸治疗进行性家族性肝内胆汁淤积症：一项随机、安慰剂对照、3 期临床试验。
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.与 ABCB4 基因突变相关的肝脏疾病谱：病理生理学和临床方面。
Semin Liver Dis. 2010 May;30(2):134-46. doi: 10.1055/s-0030-1253223. Epub 2010 Apr 26.
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The wide spectrum of multidrug resistance 3 deficiency: from neonatal cholestasis to cirrhosis of adulthood.多药耐药3缺乏症的广泛谱系：从新生儿胆汁淤积到成人肝硬化
Gastroenterology. 2001 May;120(6):1448-58. doi: 10.1053/gast.2001.23984.