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Update on Pharmacotherapies for Cholestatic Liver Disease


Update on Pharmacotherapies for Cholestatic Liver Disease

Ahmad H Ali et al. Hepatol Commun.


Cholestatic liver diseases are conditions with impaired bile formation and/or flow due to genetic, immunologic, environmental, or other causes. Unless successfully treated, this can lead to chronic liver injury and end-stage liver disease. Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) embody the most prominent adult cholestatic liver diseases with regard to incidence, morbidity, and mortality. A considerable proportion of patients with PBC and PSC experience progressive liver disease and ultimately liver-related death due to a paucity of effective pharmacotherapy; however, novel pharmacologic developments offer substantial promise in this regard. Here, we provide a brief review and update on current and emerging pharmacotherapies for PBC and PSC. (Hepatology Communications 2017;1:7-17).


Figure 1
Figure 1
Illustration of drugs and their proposed target site(s) in PBC and PSC. (A) NGM282; (B) fibrates, nor‐UDCA, and UDCA; (C) obeticholic acid; (D) ustekinumab; (E) budesonide; (F) LUM001; (G) antibiotics in PSC; (H) BTT1023; (I) abatacept. Abbreviations: AE2, anion exchanger 2; ASBT, apical sodium bile acid transporter; ATP, adenosine triphosphate; BSEP, bile salt export pump; FGFR4, fibroblast growth factor receptor 4; MRP, multidrug resistance protein; NTCP, sodium/taurocholate co‐transporting polypeptide; OATPs, organic anion transporting polypeptide; OSTα and β, organic solute transporter α and β; t‐ASBT, truncated version of ASBT; TGR5, G protein‐coupled bile acid receptor 1. (A portion of this figure is adapted from Tabibian JH, Lindor KD. Primary sclerosing cholangitis: a review and update on therapeutic developments. Expert Rev Gastroenterol Hepatol 2016;7(2):103‐114. PMID: 23363260 with permission. Copyright Mayo Foundation for Medical Education and Research. All rights reserved.)

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    1. Carey EJ, Ali AH, Lindor KD. Primary biliary cirrhosis. Lancet 2015;386:1565–1575. - PubMed
    1. Boonstra K, Beuers U, Ponsioen CY. Epidemiology of primary sclerosing cholangitis and primary biliary cirrhosis: a systematic review. J Hepatol 2012;56:1181–1188. - PubMed
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    1. Lindor KD, Gershwin ME, Poupon R, Kaplan M, Bergasa NV, Heathcote EJ, et al. Primary biliary cirrhosis. Hepatology 2009;50:291–308. - PubMed

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