Role of Methotrexate in the Treatment of Chronic Cholestatic Disorders

This article examines the therapeutic potential of methotrexate (MTX) in the treatment of cholestatic liver disease, with specific reference to its use in primary biliary cirrhosis (PBC) and primary sclerosing cholangitis (PSC). In PBC patients, evidence suggests that a particular subset of patients may benefit, either biochemically or symptomatically, from the addition of MTX to their therapeutic regimen. How best to define these potential MTX responders remains unclear, but may warrant a “therapeutic trial” in some patients with more aggressive disease or in patients with refractory and disabling PBC-related symptoms. In contrast, PSC continues to be an immensely challenging disease to effectively treat, and the role of MTX for treating PSC patients remains dubious.