Primary Sclerosing Cholangitis Medication

Updated: Nov 21, 2019
  • Author: Vikas Khurana, MD, FACP, FACG; Chief Editor: Vinay Kumar Kapoor, MBBS, MS, FRCS, FAMS  more...
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Medication Summary

No pharmacologic therapy has been proven effective for primary sclerosing cholangitis (PSC). Drug therapy is aimed at treating symptoms and managing complications. Immunosuppressants, chelators, and steroids are used in an attempt to control the disease process but have not shown significant benefit. Ursodeoxycholic acid (UDCA) improves the liver function profile in some patients and, in conjunction with endoscopic dilation, has shown a survival benefit in some studies. Trials using UDCA in higher doses and earlier in the disease course are ongoing.

Biologics (eg, anti-TNF-α) may benefit PSC when used to treat concomitant IBD. [40]

Phase II and III clinical trials are investigating new drugs designed to target specific disease mechanisms for precision medicine treatment of PSC. [14]


Immunosuppressant agents

Class Summary

With the possibility of an autoimmune pathogenesis for PSC, immunosuppressive therapy has been used in treatment of PSC. Results of therapeutic trials, however, have been disappointing.

Azathioprine (Imuran, Azasan)

Azathioprine antagonizes purine metabolism and inhibits synthesis of DNA, RNA, and proteins. It may decrease proliferation of immune cells, which results in lower autoimmune activity.

Cyclosporine (Sandimmune, Neoral)

Cyclosporine is a cyclic polypeptide that suppresses some humoral immunity and, to a greater extent, cell-mediated immune reactions, such as delayed hypersensitivity, allograft rejection, experimental allergic encephalomyelitis, and graft versus host disease for a variety of organs. For both children and adults, dosing is based on ideal body weight.


Prednisone is an immunosuppressant for the treatment of autoimmune disorders. It may decrease inflammation by reversing increased capillary permeability and suppressing polymorphonuclear activity. It stabilizes lysosomal membranes and suppresses lymphocyte and antibody production.

Methotrexate (Trexall, Rheumatrex)

Methotrexate is an antimetabolite used in the treatment of certain neoplastic diseases, severe psoriasis, and adult rheumatoid arthritis. It inhibits dihydrofolic acid reductase. Dihydrofolates must be reduced to tetrahydrofolates by this enzyme before they can be used as carriers of single-carbon groups in the synthesis of purine nucleotides and thymidylate. Methotrexate therefore interferes with DNA synthesis, repair, and cellular replication.



Class Summary

Observation of increased serum, urinary, and hepatic copper concentrations in patients with primary sclerosing cholangitis has prompted the use of penicillamine, which is a chelator.

Penicillamine (Cuprimine, Depen)

Penicillamine is a chelating agent that is recommended for the removal of excess copper in patients with Wilson disease. It depresses circulating IgM rheumatoid factor and T-cell activity but not B-cell activity.


Gallstone Solubilizing Agents

Class Summary

Ursodiol is thought to remove toxic bile acids from the enterohepatic circulation and to offer protection to the bile duct from injury.

Ursodiol (Actigall)

Ursodiol suppresses hepatic synthesis and secretion of cholesterol and inhibits intestinal absorption of cholesterol. It may displace natural, toxic, and endogenous bile acids from the enterohepatic circulation and provide a cytoprotective effect, which may lead to decreased cholestasis and improved liver functions.


Lipid-Lowering Agents, Other

Class Summary

Cholestyramine is thought to decrease pruritus by combining with bile acids in the intestine and by causing them to be excreted because of nonreabsorption.

Cholestyramine (Prevalite, Questran)

Cholestyramine forms a nonabsorbable complex with bile acids in the intestine, which, in turn, inhibits enterohepatic reuptake of intestinal bile salts. In patients with partial biliary obstruction, the reduction of serum bile acid levels by cholestyramine reduces excess bile acids deposited in dermal tissue, which decreases pruritus.