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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)

A prospective trial of D-penicillamine in primary biliary cirrhosis.

We evaluated D-penicillamine in the treatment of primary biliary cirrhosis. In a prospective double-blind trial, 26 patients received D-penicillamine (250 mg four times a day), and 26 received an identical placebo. Although the desired urinary excretion of copper was achieved in patients taking D-penicillamine, there was no improvement in survival or symptoms after 28 months. Serum bilirubin and alkaline phosphatase increased equally in both groups. Alanine and aspartate aminotransferases were lower in the D-penicillamine group, but serum albumin was also lower in this group. Liver histology worsened equally in both groups. Major side effects, some appearing more than 24 months after the start of treatment, occurred in 31 per cent of the patients receiving D-penicillamine. Less serious side effects occurred in an additional 46 per cent. We conclude that D-penicillamine at the dosage we used is not effective in the treatment of primary biliary cirrhosis and is associated with a high incidence of serious side effects.[1]


  1. A prospective trial of D-penicillamine in primary biliary cirrhosis. Matloff, D.S., Alpert, E., Resnick, R.H., Kaplan, M.M. N. Engl. J. Med. (1982) [Pubmed]
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