Primary binary cirrhosis (PBC) is a disease of unknown etiology characterized by slowly progressive intrahepatic cholestasis due to non-suppurative, destruction of small intrahepatic bile ducts. Because some other autoimmune diseases appear to respond favorably to certain immunosuppressive agents, trials of selected immunosuppressive drugs are being undertaken in patients with symptomatic PBC. In a randomized controlled trial of chlorambucil therapy, treatment with the drug was associated with a decrease in the rate of increase of serum bilirubin, normalization of elevated serum IgM levels and an improvement in inflammatory cell infiltration in liver biopsies. These findings have prompted a search for safer and more effective immunosuppressive drugs with less carcinogenic potential than chlorambucol. Ten patients with PBC have been entered into an open trial of methotrexate therapy and are tolerating the drug well.
