TREATMENT OF WILSONS-DISEASE WITH AMMONIUM TETRATHIOMOLYBDATE .1. INITIAL THERAPY IN 17 NEUROLOGICALLY AFFECTED PATIENTS

Objective: To test the efficacy and toxicity of a new drug, ammonium t etrathiomolybdate, in the initial treatment of a relatively large seri es of patients presenting with neurologic signs and symptoms caused by Wilson's disease. The key aspect of efficacy was to preserve the neur ologic function present at the onset of therapy. Design: An open study of 17 patients treated for 8 weeks each. Neurologic function was eval uated by frequent quantitative neurologic and speech examinations. Sev eral copper-related variables were studied to evaluate the effect of t he drug on copper, and a large number of biochemical and clinical vari ables were studied to evaluate potential toxicity. Patients were then followed up at yearly intervals, with follow-up periods of 1 to 5 year s reported. Setting: A university hospital referral setting. Intervent ion: Patients were generally treated for 8 weeks with tetrathiomolybda te, followed by zinc maintenance therapy. Main Outcome Measures: Neuro logic function was evaluated by quantitative neurologic and speech exa minations. Results: None of the patients suffered a loss of neurologic function. Copper status and potential further toxic effects were gene rally well controlled quickly: No toxic effects resulted from administ ration of tetrathiomolybdate. During the ensuing period of follow-up o f 1 to 5 years, neurologic recovery in most patients was good to excel lent. Conclusions: Tetrathiomolybdate appears to be an excellent form of initial treatment in patients with Wilson's disease presenting with neurologic signs and symptoms. In contrast to penicillamine therapy, initial treatment with tetrathiomolybdate does not result in further, often irreversible neurologic deterioration.