Comparative assessment of clinical rating scales in Wilson's disease

• Published in: BMC neurology Vol. 17; no. 1; p. 140
• Main Authors: Volpert, Hanna M, Pfeiffenberger, Jan, Gröner, Jan B, Stremmel, Wolfgang, Gotthardt, Daniel N, Schäfer, Mark, Weiss, Karl Heinz, Weiler, Markus
• Format: Journal Article
• Language: English
• Published: England BioMed Central Ltd 21.07.2017; BioMed Central; BMC
• Subjects: Activities of daily living; Adolescent; Adult; Age; Clinical trials; Copper - metabolism; Cross-Sectional Studies; Female; Gastroenterology; German network of hereditary movement disorders (GeNeMove); Global assessment scale for Wilson’s disease (GAS for WD); Hepatolenticular Degeneration - diagnosis; Hereditary diseases; Humans; Hypertension; Liver cirrhosis; Liver diseases; Liver Function Tests; Male; Medical prognosis; Middle Aged; Patients; Prospective Studies; Psychological aspects; Rating scales (Social science research); Risk factors; Unified Wilson’s disease rating scale (UWDRS); Wilson disease; Wilson's disease; Young Adult; German network of hereditary movement disorders (GeNeMove); Copper metabolism; Unified Wilson’s disease rating scale (UWDRS); Global assessment scale for Wilson’s disease (GAS for WD); Wilson disease
• ISSN: 1471-2377; 1471-2377
• DOI: 10.1186/s12883-017-0921-3

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.