Comparative assessment of clinical rating scales in Wilson’s disease

Hanna M. Volpert, Jan Pfeiffenberger, Jan B. Gröner, Wolfgang Stremmel, Daniel N. Gotthardt, Mark Schäfer, Karl Heinz Weiss, Markus Weiler

Wilson's disease (WD) is an autosomal recessive disorder of copper metabolism resulting in multifaceted neurological, hepatic, and psychiatric symptoms. The objective of the study was to comparatively assess two clinical rating scales for WD, the Unified Wilson's Disease Rating Scale (UWDRS) and the Global Assessment Scale for Wilson's disease (GAS for WD), and to test the feasibility of the patient reported part of the UWDRS neurological subscale (termed the "minimal UWDRS"). In this prospective, monocentric, cross-sectional study, 65 patients (median age 35 [range: 15-62] years; 33 female, 32 male) with treated WD were scored according to the two rating scales. The UWDRS neurological subscore correlated with the GAS for WD Tier 2 score (r = 0.80; p < 0.001). Correlations of the UWDRS hepatic subscore and the GAS for WD Tier 1 score with both the Model for End Stage Liver Disease (MELD) score (r = 0.44/r = 0.28; p < 0.001/p = 0.027) and the Child-Pugh score (r = 0.32/r = 0.12; p = 0.015/p = 0.376) were weak. The "minimal UWDRS" score significantly correlated with the UWDRS total score (r = 0.86), the UWDRS neurological subscore (r = 0.89), and the GAS for WD Tier 2 score (r = 0.86). The UWDRS neurological and psychiatric subscales and the GAS for WD Tier 2 score are valuable tools for the clinical assessment of WD patients. The "minimal UWDRS" is a practical prescreening tool outside scientific trials.