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Bile acid transport inhibitors in paediatric hepatology: more than just an itch.

作者信息

Diamond Tamir, Kamath Binita M

机构信息

Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

Division of Gastroenterology Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA, USA.

出版信息

Nat Rev Gastroenterol Hepatol. 2024 Dec;21(12):825-826. doi: 10.1038/s41575-024-01008-w.

DOI:10.1038/s41575-024-01008-w
PMID:39448838
Abstract
摘要

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本文引用的文献

1
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.
2
Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial.奥贝胆酸治疗 Alagille 综合征患者的疗效和安全性（ASSERT）：一项 3 期、双盲、随机、安慰剂对照试验。
Lancet Gastroenterol Hepatol. 2024 Jul;9(7):632-645. doi: 10.1016/S2468-1253(24)00074-8. Epub 2024 Apr 23.
3
Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.
马拉利昔巴特治疗 Alagille 综合征患者的无事件生存与 GALA 真实世界队列的比较。
Hepatology. 2024 Jun 1;79(6):1279-1292. doi: 10.1097/HEP.0000000000000727. Epub 2023 Dec 25.
4
Surgical versus Medical Management of Progressive Familial Intrahepatic Cholestasis-Case Compilation and Review of the Literature.进行性家族性肝内胆汁淤积症的手术与药物治疗——病例汇编及文献综述
Children (Basel). 2023 May 26;10(6):949. doi: 10.3390/children10060949.
5
A3907, a systemic ASBT inhibitor, improves cholestasis in mice by multiorgan activity and shows translational relevance to humans.A3907，一种全身性 ASBT 抑制剂，通过多器官作用改善了小鼠的胆汁淤积，并显示出对人类的转化相关性。
Hepatology. 2023 Sep 1;78(3):709-726. doi: 10.1097/HEP.0000000000000376. Epub 2023 Apr 1.
6
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.
7
Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study.马拉硫磷治疗阿拉吉列综合征和胆汁淤积性瘙痒症患者的疗效和安全性（ICONIC）：一项随机2期研究。
Lancet. 2021 Oct 30;398(10311):1581-1592. doi: 10.1016/S0140-6736(21)01256-3. Epub 2021 Oct 28.
8
Genotype correlates with the natural history of severe bile salt export pump deficiency.基因型与严重胆盐输出泵缺陷的自然病史相关。
J Hepatol. 2020 Jul;73(1):84-93. doi: 10.1016/j.jhep.2020.02.007. Epub 2020 Feb 20.
9
Pharmacological inhibition of apical sodium-dependent bile acid transporter changes bile composition and blocks progression of sclerosing cholangitis in multidrug resistance 2 knockout mice.顶端钠依赖性胆汁酸转运体的药理学抑制改变胆汁成分并阻断多药耐药2基因敲除小鼠硬化性胆管炎的进展。
Hepatology. 2016 Feb;63(2):512-23. doi: 10.1002/hep.27973. Epub 2015 Aug 21.