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.
D000328:Adult; D000368:Aged; D002986:Clinical Trials as Topic; D003300:Copper / Q000378:metabolism; D005260:Female; D006801:Humans; D008099:Liver / Q000378:metabolism; D008105:Liver Cirrhosis, Biliary / Q000175:diagnosis* / Q000188:drug therapy / Q000473:pathology; D008111:Liver Function Tests; D008297:Male; D008875:Middle Aged; D010396:Penicillamine / Q000009:adverse effects / Q000627:therapeutic use; D010919:Placebos
D010919:Placebos; D003300:Copper; D010396:Penicillamine