title: Compliant treatment with anti-copper agents prevents clinically overt Wilson's disease in pre-symptomatic patients.
abstract: OBJECTIVE
  Wilson's disease (WD) is an inherited copper metabolism disorder that leads to dysfunction of affected tissues, mostly in the liver and brain. Anti-copper treatment should prevent clinically overt WD in pre-symptomatic patients but this has not been supported by strong evidence. Our aim was to evaluate the long-term effectiveness of treatment in clinically pre-symptomatic patients, with particular emphasis on patient compliance with treatment.
  
  
  METHODS
  Data were analyzed for 87 consecutive patients with no clinical symptoms of WD who were identified between 1957 and 2009 by family screening. All of them since diagnosis were treated with either zinc sulphate (Zn) (66.7%) or D-penicillamine (DPA) (33.3%).
  
  
  RESULTS
  During a median follow-up of 12 years (range 3-52), 55 (63%) patients remained without clinical symptoms, 13 (15%) developed neuropsychiatric symptoms and 21 (24%) developed hepatic dysfunction, including five deaths from hepatic failure. Non-compliance for at least three consecutive months was observed in 39 patients, and in 29 cases this extended for more than 12 months. Multivariate analysis showed that the odds of developing symptomatic WD were independently increased by non-compliance (odds ratio 24.0, 95% confidence interval 6.0-99.0). According to Kaplan-Meier analysis patients who were compliant to treatment had a significantly higher likelihood of remaining symptom-free, and their overall survival was similar to the survival rate observed in the general population.
  
  
  CONCLUSIONS
  The use of anti-copper agents in clinically pre-symptomatic patients diagnosed with WD allows clinically overt disease to be effectively prevented. However, compliance with therapy is extremely important.
authors: Second Department of Neurology, Institute of Psychiatry and Neurology, Warsaw, Poland.
authors: K
authors: K
authors: Dzieżyc
authors: M
authors: M
authors: Karliński
authors: T
authors: T
authors: Litwin
authors: A
authors: A
authors: Członkowska
mesh_terms: D000293:Adolescent; D000328:Adult; D002614:Chelating Agents / Q000627:therapeutic use*; D002648:Child; D005260:Female; D005500:Follow-Up Studies; D006527:Hepatolenticular Degeneration / Q000188:drug therapy* / Q000401:mortality / Q000517:prevention & control*; D006801:Humans; D008297:Male; D008875:Middle Aged; D010349:Patient Compliance; D010396:Penicillamine / Q000627:therapeutic use*; D015996:Survival Rate; D016896:Treatment Outcome; D055815:Young Adult; D019287:Zinc Sulfate / Q000627:therapeutic use*
chemical_list: D002614:Chelating Agents; D019287:Zinc Sulfate; D010396:Penicillamine
keywords: Wilson's disease; anti-copper treatment; family screening; symptom development