Efficacy of targeted multimodal psychosocial rehabilitation on residual neuropsychiatric symptoms in Wilson's disease: A propensity score-matched cohort study.

BACKGROUND

Although standard chelation therapy effectively controls copper load, many patients with Wilson's disease (WD) suffer from persistent, refractory residual neuropsychiatric symptoms. This study evaluated the real-world efficacy of a structured multimodal psychosocial rehabilitation program on neurological, cognitive, and functional outcomes in adult patients with WD.

METHODS

We retrospectively analyzed adults with neurologic or mixed-phenotype WD who retained symptoms despite ≥12 months of standard anti-copper therapy. The intervention group underwent an 8 to 12 weeks multimodal rehabilitation program (integrating fine motor, cognitive, and psychosocial training), while controls received standard care. We employed 1:1 propensity score matching (PSM) to rigorously control for confounders. The primary outcome was the Global Assessment Scale for Wilson's Disease (GAS for WD) Tier 2 score at 6 months.

RESULTS

After PSM, 68 patients (34 per group) with well-balanced baseline characteristics were analyzed. Analysis of covariance (ANCOVA) revealed that rehabilitation significantly improved the primary outcome compared to controls (adjusted mean difference in GAS Tier 2: -3.90; 95% CI: -4.51 to -3.30; P < 0.001). The rehabilitation group also achieved significant gains in cognition (Montreal Cognitive Assessment difference: 2.15; P < 0.001) and activities of daily living (difference: 8.60; P < 0.001).

CONCLUSION

Adjunctive multimodal psychosocial rehabilitation safely and significantly ameliorates residual neurological, cognitive, and functional deficits in adult WD patients. These findings support integrating non-pharmacological interventions into comprehensive WD management to maximize functional recovery.