Primary sclerosing cholangitis and primary biliary cirrhosis. How effective is medical therapy?

Although a great deal of investigation has led to some novel approaches in treating primary sclerosing cholangitis (PSC) and primary biliary cirrhosis (PBC), no method has been proven to definitively alter the long-term outcome of either disease. In addition, many of the medical regimens reviewed in this article have serious toxicity, which limits their widespread use. Furthermore, the natural history of PSC and PBC may vary widely between individual patients, complicating the decision of whom and when to treat. Ursodeoxycholic acid (Actigall) appears to be a safe and possibly effective agent for both diseases in their early stages, and its use in selected patients may be warranted. Methotrexate (Folex, Mexate, Rheumatrex Dose Pack) may improve liver enzyme levels in some patients, but it is hepatotoxic and should be used cautiously. The general application of toxic immunosuppressive agents should await the results of large, long-term, prospective trials. Liver transplantation is the only proven effective therapy for advanced, decompensated PSC and PBC.