Department of Neurology, University of Ulm, Ulm, Germany.
Mov Disord. 2012 Jan;27(1):118-24. doi: 10.1002/mds.23847. Epub 2011 Nov 17.
Sensitive outcome measures for patients with Huntington's disease (HD) are required for future clinical trials. Longitudinal data were collected from a 3-year study of 379 patients suffering from early HD who were not treated by antipsychotics. Progression of UHDRS item scores was evaluated by linear regression and slope, whereas correlation coefficient, standard error, and P values were estimated on the basis of the data of eight evaluations from screening to study end (36 months). For the functional assessment dimension, the proportion of "no" responses at baseline and at study end was determined. Linear progression was observed for the motor score and for all three functional measures (i.e., functional assessment score, independence assessment score, and total functional capacity score). In contrast, there was little evidence for progression of the behavioral assessment score over the study period, whereas the cognitive assessment score was intermediate. Twenty-two motor-score items showed linear progression, with a slope of >0.003. These included all chorea items, finger tapping and pronation/supination (left and right), gait, tongue protrusion, and tandem walking. Different symptom domains and individual items evolved at different rates in this group of patients suffering from early HD. It may be possible to select sensitive items to create a simplified version of the UHDRS, which would be more efficient and more sensitive for the assessment of disease progression in clinical trials and natural history studies.
需要针对亨廷顿病（HD）患者的敏感结局指标，以便进行未来的临床试验。对 379 名未接受抗精神病药物治疗的早期 HD 患者进行了为期 3 年的研究，收集了纵向数据。采用线性回归和斜率评估 UHDRS 项目评分的进展，而相关系数、标准误差和 P 值则根据从筛查到研究结束（36 个月）的 8 次评估的数据进行估计。对于功能评估维度，确定了基线和研究结束时“否”的比例。运动评分和所有三个功能测量（即功能评估评分、独立性评估评分和总功能能力评分）均显示出线性进展。相比之下，在研究期间，行为评估评分的进展几乎没有证据，而认知评估评分处于中间水平。22 个运动评分项目显示出线性进展，斜率>0.003。这些项目包括所有舞蹈症项目、手指敲击和旋前/旋后（左右）、步态、伸舌和串联行走。在这群患有早期 HD 的患者中，不同的症状域和单个项目以不同的速度演变。可能可以选择敏感的项目来创建 UHDRS 的简化版本，这将在临床试验和自然史研究中更有效地评估疾病进展，并更敏感。
