Persistence with treatment for Wilson disease: a retrospective study

Area of Science:

• Medical Genetics
• Pharmacology
• Clinical Medicine

Background:

• Wilson disease (WD) is a genetic disorder of copper metabolism.
• Early diagnosis and pharmacological treatment are key to favorable patient outcomes.
• Limited data exist on treatment persistence in chronic diseases like WD.

Purpose of the Study:

• To assess treatment persistence among Wilson disease patients.
• To analyze the impact of treatment persistence on patient outcomes.
• To identify factors influencing treatment persistence in WD.

Main Methods:

• Retrospective analysis of 170 newly diagnosed, symptomatic WD patients (1995-2005).
• Treatment non-persistence defined by specific durations of treatment breaks.
• Results analyzed against clinical variables.

Main Results:

• 74.1% of patients demonstrated treatment persistence over a mean of 11.7 years.
• Persistence significantly correlated with improved clinical outcomes and reduced disease progression (p < 0.001).
• Higher education and family support were key factors for persistence; treatment type, gender, and adverse events were not related.

Conclusions:

• Approximately one-quarter of WD patients exhibit irregular anti-copper treatment adherence.
• Non-persistence significantly worsens clinical outcomes.
• Family support is a critical factor in maintaining treatment persistence for Wilson disease patients.