TREATMENT OF WILSONS-DISEASE WITH ZINC - XV - LONG-TERM FOLLOW-UP-STUDIES

Wilson's disease is an inherited disease of copper accumulation caused by a failure of biliary excretion of excess copper. Accumulated coppe r causes liver disease in these patients, and in perhaps two thirds of patients, it causes brain damage leading to clinical neurologic or ps ychiatric dysfunction. Maintenance treatment involves reversing the po sitive copper balance. The earliest approaches have used chelators, su ch as penicillamine or trientine, which increase the urinary excretion of copper. A more recent approach has used zinc, which blocks the abs orption of copper and increases copper excretion in the stool. Because of the high level of endogenously secreted copper in alimentary secre tions, the reabsorption of which is partially blocked by zinc therapy, zinc acts to remove accumulated copper from the body as well as preve nt its reaccumulation. In the present article we present data on the l ong-term follow-up (up to 10 years) of maintenance zinc treatment of 1 41 patients with Wilson's disease. The data presented document that zi nc is effective as a sole therapy in the long-term maintenance treatme nt of Wilson's disease and that if has a low toxicity. The results dem onstrate the efficacy of zinc therapy in treating the presymptomatic p atient from the beginning of therapy. We also present limited data on the use of zinc in the treatment of pregnant patients and children who have Wilson's disease; these data also indicate efficacy and low toxi city. The median follow-up period for the group as a whole is 4.8 year s; for the presymptomatic patients it is 6.5 years; for the children i t is 3.6 years.