Treatment of Wilson's disease (hepatolenticular degeneration) with DL‐penicillamine

Abstract

At the Mayo Clinic, 7 patients with Wilson''s disease have been treated with DL-penicillamine; 6 showed some degree of improvement over a period of seventeen to thirty-three months, and 1 patient with advanced disease died of progressive hepatic failure after receiving DL-penicillamine for less than a month. Of these 7 patients, 5 received treatment with dimercaprol (BAL) at some time. The diuresis of copper and the clinical response to BAL did not appear to be as satisfactory as the response to DL-penicillamine. Of the 7 patients, 4 had toxic reactions to DL-penicillamine, but ultimately these prevented treatment with this material in only 1 patient. Of the 5 patients who received BAL, 2 had some type of toxic reaction, but this prevented further treatment with BAL in only 1 patient. However, because of pain at the site of injection, the frequency of treatment with BAL was often suboptimal. Use of DL-penicillamine evoked an initial pronounced cupriuresis. The degree of cupriuresis after long-term treatment with DL-penicillamine was far less than it was during the initial use of this preparation. Despite the decrease in urinary excretion of copper after a year of treatment, clinical improvement continued and there was a decrease in the Kayser- Fleischer rings in 3 patients. It is thought that these observations are compatible with the massive removal of considerable copper from the tissues early in the course of treatment. Some patients may have irremediable tissue damage and thus may not show clinical improvement despite satisfactory cupriuresis. Although prolonged treatment with DL-penicillamine resulted in improvement in the neu-rologic status, the change in hepatic functions was variable.