Asymptomatic primary biliary cirrhosis. Presentation, histology, and results with D-penicillamine.

Of 103 patients with the syndrome of primary biliary cirrhosis (chronic, nonsuppurative destructive cholangitis) who entered a double-blind, randomized, controlled treatment trial with either D-penicillamine or placebo, 21 (20%) were asymptomatic with respect to their liver disease. Study of these 21 patients revealed that (1) 43% of patients with asymptomatic primary biliary cirrhosis had advanced histologic lesions (fibrosis or cirrhosis); (2) asymptomatic patients with advanced histologic lesions likely have had their disease for 10 years or more; (3) stage of primary biliary cirrhosis may remain unchanged for years; and (4) most asymptomatic patients receiving D-penicillamine, when compared with patients given placebo, had improved liver function tests at 1-year follow-up. However, the incidence of major toxicity with D-penicillamine for primary biliary cirrhosis in a maintenance dose of 1 g approximates 20%. Furthermore, one of our patients who was asymptomatic but who had advanced histologic changes died recently from D-penicillamine-associated bone marrow suppression. It remains to be determined whether the benefit-to-risk ratio of D-penicillamine in primary biliary cirrhosis justifies its use.

RCT Entities:   Population Interventions Outcomes