Department of Neurology, National Yang-Ming University School of Medicine, Taipei, Taiwan.
Mov Disord. 2011 Sep;26(11):2081-7. doi: 10.1002/mds.23809. Epub 2011 May 28.
We quantitatively investigated the clinical severity and progression of diseases with ataxia, as measured with the Scale for the Assessment and Rating of Ataxia, and examined the potential application of the Scale for the Assessment and Rating of Ataxia for future therapeutic trials. Severity of ataxia was assessed in 238 patients with spinocerebellar ataxia type 2, spinocerebellar ataxia type 3, spinocerebellar ataxia type 6, spinocerebellar ataxia type 17, multiple system atrophy-cerebellar variant, or Gerstman-Sträussler-Scheinker disease. Among them, 119 (50%) were longitudinally examined three to seven times, in a period of 8 to 38 months, resulting in a total set of 535 assessments. The differences between spinocerebellar ataxia and multiple system atrophy-cerebellar variant were ascertained cross-sectionally and longitudinally. Gerstman-Sträussler-Scheinker disease had the fastest progression, followed by multiple system atrophy-cerebellar variant, spinocerebellar ataxia type 17, spinocerebellar ataxia type 3, spinocerebellar ataxia type 2, and spinocerebellar ataxia type 6. Patients with multiple system atrophy-cerebellar variant had a faster progression in gait, sitting, speech, and total score than patients with spinocerebellar ataxias. For a randomized, case-control trial, a sample size of 47 for spinocerebellar ataxia and 85 for multiple system atrophy-cerebellar variant in the treatment or placebo arms would have a sufficient statistical power to demonstrate the efficacy of a new therapy that would retard ataxia progression by 1 point per year as measured by the Scale for the Assessment and Rating of Ataxia. The results will have a significant impact on the planning and implementation of future therapeutic trials of spinocerebellar ataxia and multiple system atrophy-cerebellar variant.
我们使用评定和评测共济失调量表定量地研究了以共济失调为特征的疾病的临床严重程度和进展，并检验了评定和评测共济失调量表在未来治疗试验中的潜在应用。在 238 例脊髓小脑性共济失调 2 型、3 型、6 型、17 型、多系统萎缩-小脑型或 Gerstmann-Sträussler-Scheinker 病患者中评估了共济失调的严重程度。其中 119 例（50%）患者接受了 3 至 7 次纵向检查，随访时间为 8 至 38 个月，共进行了 535 次评估。我们分别在横断面和纵向比较了脊髓小脑性共济失调和多系统萎缩-小脑型之间的差异。Gerstmann-Sträussler-Scheinker 病进展最快，其次是多系统萎缩-小脑型、脊髓小脑性共济失调 17 型、3 型、2 型和 6 型。与脊髓小脑性共济失调患者相比，多系统萎缩-小脑型患者在步态、坐姿、言语和总分方面的进展更快。对于一项随机、病例对照试验，如果在治疗组或安慰剂组中，脊髓小脑性共济失调的样本量为 47 例，多系统萎缩-小脑型为 85 例，则具有足够的统计效力来证明新疗法的疗效，该疗法可以使评定和评测共济失调量表的评分每年延缓 1 分。这些结果将对脊髓小脑性共济失调和多系统萎缩-小脑型的未来治疗试验的规划和实施产生重大影响。
