Department of Neurology, University Hospital of Bonn, Sigmund-Freud-Str. 25, D-53105 Bonn, Germany.
Neurology. 2010 Feb 23;74(8):678-84. doi: 10.1212/WNL.0b013e3181d1a6c9.
To determine the longitudinal metric properties of recently developed clinical assessment tools in spinocerebellar ataxia (SCA).
A subset of 171 patients from the EUROSCA natural history study cohort (43 SCA1, 61 SCA2, 37 SCA3, and 30 SCA6) were examined after 1 year of follow-up. Score changes and effect size indices were calculated for clinical scales (Scale for the Assessment and Rating of Ataxia [SARA], Inventory of Non-Ataxia Symptoms [INAS]), functional tests (SCA Functional Index [SCAFI] and components), and a patient-based scale for subjective health status (EQ-5D visual analogue scale [EQVAS]). Responsiveness was determined in relation to the patient's global impression (PGI) of change and reproducibility described as retest reliability for the stable groups and smallest detectable change.
Within the 1-year follow-up period, SARA, INAS, and SCAFI but not EQVAS indicated worsening in the whole group and in the groups with subjective (PGI) worsening. SCAFI and its 9-hole pegboard (9HPT) component also deteriorated in the stable groups. Standardized response means were highest for 9HPT (-0.67), SARA (0.50), and SCAFI (-0.48) with accordingly lower sample size estimates of 143, 250, or 275 per group for a 2-arm interventional trial that aims to reduce disease progression by 50%. SARA and EQVAS performed best to distinguish groups classified as worse by PGI. All scales except EQVAS reached the criterion for retest reliability.
While both the Scale for the Assessment and Rating of Ataxia and the SCA Functional Index (SCAFI) (and its 9-hole pegboard component) had favorable measurement precision, the clinical relevance of SCAFI and 9-hole pegboard score changes warrants further exploration. The EQ-5D visual analogue scale proved insufficient for longitudinal assessment, but validly reflected patients' impression of change.
确定最近开发的脊髓小脑共济失调（SCA）临床评估工具的纵向度量属性。
EUROSCA 自然史研究队列中的 171 名患者中有一部分（43 名 SCA1、61 名 SCA2、37 名 SCA3 和 30 名 SCA6）在随访 1 年后接受了检查。为临床量表（共济失调评估量表[SARA]、非共济失调症状量表[INAS]）、功能测试（SCA 功能指数[SCAFI]和组成部分）和基于患者的主观健康状况量表（EQ-5D 视觉模拟量表[EQVAS]）计算了评分变化和效应大小指数。根据患者的总体印象（PGI）变化和稳定性组的重测可靠性和最小可检测变化来确定反应性。
在 1 年的随访期间，SARA、INAS 和 SCAFI 但不是 EQVAS 表明整个组和主观（PGI）恶化的组中情况恶化。SCAFI 和其 9 孔钉板（9HPT）组件在稳定组中也恶化。标准化反应均值最高的是 9HPT（-0.67）、SARA（0.50）和 SCAFI（-0.48），每组需要 143、250 或 275 个样本，用于旨在减少疾病进展 50%的 2 臂干预试验。SARA 和 EQVAS 最适合区分被 PGI 评为更差的组。除 EQVAS 外，所有量表均达到重测可靠性标准。
虽然 SARA 和 SCAFI（及其 9 孔钉板组件）都具有良好的测量精度，但 SCAFI 和 9 孔钉板评分变化的临床相关性需要进一步探索。EQ-5D 视觉模拟量表在纵向评估中证明不足，但有效反映了患者对变化的印象。
