Treatment and management of Wilson's disease.

Wilson's disease is an autosomal recessive disorder related to the copper metabolism. The clinical symptoms are due to copper deposition in various tissues, including liver, brain, kidney, cornea and others. The key strategy of treatment is to reduce the amount of copper in the liver and other tissues by administering both copper-chelating agents and a low copper diet. D-Penicillamine is considered to be the first choice as a copper-chelating agent. Patients require 15-25 mg/kg daily in the early stages of treatment and this drug should also be given more than 2 h before meals. Some undesirable or serious side-effects, such as systemic lupus erythematosus (SLE) and nephrotic syndrome, do occur in 20-25% of all patients. In such cases, trienthylene tetramine (trientine) appears to be as effective as penicillamine. This drug is usually used when D-penicillamine has to be withdrawn. It is also sometimes administered to patients with neurological symptoms as a first-choice drug. It is given in doses of 40-50 mg/kg daily, in the same manner as for D-penicillamine. Zinc salt administration has also emerged as an interesting supportive therapy for both treatments. A dose of 5-7.5 mg/kg daily is given before meals. The copper content of the diet should be less than 1 mg/day in the early stages of treatment. Thereafter, it can be increased to 1.0-1.5 mg/day during well-controlled periods. Liver transplantation is now performed in many countries for patients with either the fulminant or chronic progressive types of Wilson's disease.