Long-term follow-up study of seventeen cases with Wilson's disease

We performed a follow-up study of 17 patients with Wilson's disease (6 men, 11 women) in order to evaluate the effect of penicillamine therapy. All patients have been treated with penicillamine for 1 to 17 years. The severity in the patients were evaluated by using the grading system (Grade 0 to 4) reported by Goldstein et al. Six of 17 patients showed improvement, 6 patients showed no change of severity and 4 patients died in spite of the treatment. One patient has been asymptomatic for 7 years by treatment. An improvement was observed in the group treated with penicillamine more than 600 mg/day, and also the group treated for longer than 5 years. There was no relationship between the severity before treatment and the subsequent effects of treatment. These results suggest that a dosage of penicillamine and the duration of the treatment are important factors of clinical improvement of the patients.