肝豆状核变性神经症状评价量表-改良Young量表

Improved Young Scale—a scale for the neurological symptoms of Wilson disease

目的设计用于评价肝豆状核变性(Wilson disease,WD)神经系统症状的量表(改良Young量表),并对该量表的信度和效度及临床意义进行了研究。方法选取住院的脑型WD初诊患者60例,对所有WD患者分别用改良Young量表、WD全面评价量表(Globle assessment scale,GAS量表)、帕金森联合量表(Unified parkinson dis-ease rating scale,UPDRS量表)、日常生活活动量表(activity of daily living scale,ADL)进行评分。间隔1 d后再次用改良Young量表进行评分。所有患者进行脑脊液铜检查。接受二巯基丙磺钠排铜治疗1个月后再次用上述量表进行评分。应用统计方法分析量表的信度、效度、反应度。并结合WD患者脑脊液铜来分析量表在WD的临床意义。结果改良Young量表的内部一致性系数Cronbachα系数=0.8733,重测信度系数在0.8以上,评定者间一致性系数维持在0.75到0.94。结构效度因子分析证明量表由肌张力障碍、其他运动障碍、高级神经功能3个方面构成,与临床构想一致。改良Young量表得分与GAS量表、UPDRS量表、ADL量表得分具有相关性。改良Young量表评分与脑脊液铜有相关性,与临床症状严重程度的相关系数高于其他量表。量表的效标尺度((治疗后得分-治疗前得分)/治疗前得分)为0.23。结论改良Young量表有较好的信度和效度,有较高的灵敏度。能有效反映WD的症状严重程度和生活质量,可监测治疗效果,在一定程度上能反映WD患者脑内的代谢异常程度。

Objective To Modify Young Scale to better evaluate neurological symptoms of patients with Wilson disease（WD）.The reliability and the validity of the modified scale were evaluated.Methods Sixty patients with primary encephalic type WD were enrolled in the study.All the patients were scored by the Modified Young Scale,the Novel Globle Assessment for WD（GAS scale）,the United Parkinson＇s Disease Rating Scale（UPDRS scale）,and the ADL scale.The Modified Young Scale was used to evaluate the patients again one day after admission,one month after treatment with dimercaptopropansulfonate.The reliability and the validity of the scale were analyzed.The clinical value of the scale was also evaluated by investigating the cerebral copper of the patients with WD.Results The scale showed a good internal consistency with Cronbach α=0.8733.The coefficient of test-retest reliability was above 0.8.The coefficient of inter-rater reliability was between 0.75 and 0.94.The factorial analysis showed that the scale mainly tested three domains such as dysmyotonia,other dyskinesia and psychogenia.The scores of the Modified Young Scale were correlated with the GAS,UPDRS and ADL,respectively.The coefficient of responsiveness was 0.21.Conclusions The Modified Young Scale showed good reliability,validity,and sensitivity.It can reflect the severity of the neural symptom of WD and the quality of life（QOL）.The scale can also reflect the cerebral abnormal metabolism and thus have the significant clinical value.