Lynch David R, Farmer Jennifer M, Wilson Robert L, Balcer Laura J
Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104-4318, USA.
Mov Disord. 2005 Jul;20(7):777-82. doi: 10.1002/mds.20449.
Although several neuroprotective agents have been proposed as potential therapies in Friedreich ataxia (FA), clinical trials of their efficacy are limited by a lack of sensitive outcome measures. We assessed whether performance measures (nine-hole peg test, the timed 25-foot walk, and low-contrast letter acuity) provide valid measures of disease status in FA. Scores for each measure correlated significantly with neurologic disability and disease duration. Rank correlations between scores for performance measures were moderate in magnitude, suggesting that the each test captures separate yet related dimensions of neurological function in FA. Linear regression models demonstrated that scores from the nine-hole peg test and the timed 25-foot walk (after reciprocal transformation) were predicted by age and triplet repeat length in patients with FA. In addition, comparison of the temporal courses of change for each performance measure demonstrated that scores from the timed 25-foot walk change early in the course of FA, nine-hole peg test scores change slowly over the full course of the disorder, and low-contrast letter acuity scores change in the later stages of the disease. Thus, a composite scale derived from these performance measures may provide the best overall measure for assessing disease progression throughout the illness.
尽管有几种神经保护剂已被提议作为弗里德赖希共济失调（FA）的潜在治疗方法，但其疗效的临床试验因缺乏敏感的结局指标而受到限制。我们评估了行为测试指标（九孔插板试验、25英尺定时步行试验和低对比度字母视力测试）是否能有效衡量FA的疾病状态。每项测试的得分与神经功能障碍和疾病持续时间均显著相关。行为测试指标得分之间的等级相关性中等，这表明每项测试捕捉到了FA神经功能中不同但相关的维度。线性回归模型表明，FA患者的年龄和三联体重复长度可预测九孔插板试验和25英尺定时步行试验（经倒数转换后）的得分。此外，对每项行为测试指标变化时间进程的比较表明，25英尺定时步行试验的得分在FA病程早期就会发生变化，九孔插板试验得分在疾病整个病程中变化缓慢，而低对比度字母视力测试得分在疾病后期发生变化。因此，由这些行为测试指标得出的综合量表可能是评估整个疾病过程中疾病进展的最佳总体指标。
