Intractable Rare Dis Res. 2015;4(1):1-6. (DOI: 10.5582/irdr.2014.01018)

Current research on the treatment of primary sclerosing cholangitis.

Ali AH, Carey EJ, Lindor KD


Primary sclerosing cholangitis (PSC) is a progressive disease of the liver characterized by inflammation and destruction of the intra- and/or extra-hepatic bile ducts, leading to fibrosis and ultimately liver failure, cirrhosis and an increased risk of malignancy. The etiology of PSC is unclear. It is often associated with the inflammatory bowel diseases (IBD), particularly Ulcerative Colitis (UC); up to 75% of PSC patients have UC. PSC is more prevalent in men than in women. Ursodeoxycholic acid (UDCA) has been extensively studied in PSC in randomized clinical trials but failed to show a positive impact on the natural course of the disease. Currently, there is no effective medical therapy for PSC, and the majority of patients will eventually require liver transplantation. PSC is one of the leading indications for liver transplantation. In this paper, we review the current research on the potential therapeutic agents for the treatment of PSC.

KEYWORDS: Primary sclerosing cholangitis, ursodeoxycholic acid, obeticholic acid, vancomycin

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