Treatment of Wilson's disease.

Abstract

On the basis of literature review and own experience we presented the method of treatment of Wilson's disease. Causative treatment has been impossible so far, although gene therapy could be real in the future. Nowadays the principle of treatment is the elimination of the excess of easily mobilized copper by chelating agents or blocking the intestinal absorption of copper. Chelation therapy, aimed at mobilizing copper from the affected organs and promoting its excretion in the urine or stool is the most important. The major chelating agent is d-penicillamine, which is quite effective but not without some side effects. Alternative chelating agents such as trientine and tetrathiomolybdate have also been successfully employed. Zinc salts are also of therapeutic value. They promote copper excretion by inducing the synthesis of metallothionein in the intestine, thereby blocking copper absorption from the gut. Zinc salts have almost no side effects. They cannot be used as an initial treatment, but are very effective for maintenance therapy. The chelation therapy is ineffective in patients with acute liver failure with encephalopathy and hemolysis. In these cases, liver transplantation is the only hope for survival. Liver transplantations in patients with dominating psychoneurological symptoms are open to discussion.