How is disease progress in Friedreich's ataxia best measured? A study of four rating scales
How is disease progress in Friedreich's ataxia best measured? A study of four rating scales
复制标题
DOI：
10.1136/jnnp.2006.096008
复制
发表时间：
2007-04-01
影响因子：
11
通讯作者：
Delatycki, M. B.
中科院分区：
文献类型：
--
作者：
Fahey, M. C.;Corben, L.;Delatycki, M. B.
背景：弗里德赖希共济失调（FRDA）是共济失调最常见的遗传原因，其特征是进行性神经变性和心肌病。最初的治疗可能会减缓病情进展，而不是逆转发病率。一个适当的和敏感的尺度来衡量疾病的进展是至关重要的检测benefitsoftreatment.Objective：比较弗里德赖希共济失调评定量表（法尔斯）与其他尺度提出作为FRDA的结果措施。采用法尔斯量表（FARS）和国际合作共济失调评定量表（ICARS）对76例患者进行评定其中72名参与者还采用功能独立性测量和改良Barthel指数进行评估。43名参与者间隔12个月重复测量。敏感性和反应性进行了评估，使用的效果大小为每个措施和所需的安慰剂对照的临床试验的样本大小。结果：法尔斯表现出较高的相关性与其他三个措施。通过法尔斯、国际合作共济失调评定量表和功能独立性测量检测到12个月内评分的显著变化。的法尔斯有最大的效果大小，需要较少的患者为一个等效的动力study.Conclusions：评估的规模，法尔斯是最好的FRDA的临床试验中使用。这是基于效果大小和功效计算，表明需要较少的参与者来证明干预措施的相同效果。需要进一步开展工作，以制定更敏感和更能作出反应的文书。
Background: Friedreich's ataxia (FRDA), the most common genetic cause of ataxia, is characterised by progressive neurodegeneration and cardiomyopathy. Initial treatments are likely to slow progression rather than reverse morbidity. An appropriate and sensitive scale to measure disease progress is critical to detect the benefit of treatments.Objective: To compare the Friedreich Ataxia Rating Scale (FARS) with other scales proposed as outcome measures for FRDA.Methods: 76 participants were assessed with the FARS and the International Cooperative Ataxia Rating Scale (ICARS) and 72 of these participants were also assessed with the Functional Independence Measure and the Modified Barthel Index. 43 participants had repeat measures at an interval of 12 months. Sensitivity and responsiveness were assessed using the effect size for each measure and the sample size required for a placebo- controlled clinical trial.Results: The FARS showed a high correlation with the other three measures. A significant change in the score over 12 months was detected by the FARS, the International Cooperative Ataxia Rating Scale and the Functional Independence Measure. The FARS had the greatest effect size and requires fewer patients for an equivalently powered study.Conclusions: Of the scales assessed, the FARS is the best to use in clinical trials of FRDA. This is based on effect size, and power calculations that show that fewer participants are required to demonstrate the same effect of an intervention. Further work is required to develop more sensitive and responsive instruments.