Fiona McDougall, Genentech 620 E Grand Ave, South San Francisco, CA 94080, USA,
J Prev Alzheimers Dis. 2021;8(2):151-160. doi: 10.14283/jpad.2020.73.
The Clinical Dementia Rating-Sum of Boxes (CDR-SB) has been proposed as a primary outcome for use in prodromal AD trials. However, the psychometric properties of this, and of other commonly used measures, have not been well-established in this patient population.
To describe the psychometric properties of commonly used efficacy measures in a clinical trial of prodromal AD.
Data were gathered as part of a two-year clinical trial.
Patients had biomarker confirmed prodromal AD.
Clinical Dementia Rating (CDR), Functional Activities Questionnaire (FAQ), Alzheimer's Disease Assessment Scale - Cognition Subscale 11 and 13 (ADAS-Cog), Mini Mental State Exam (MMSE), and Free and Cued Selective Reminding Test (FCSRT-IR [words]). Assessments were conducted at least every 24 weeks.
For the CDR-SB, test-retest reliability was good (intra-class correlation coefficient [ICC]=0.83); internal consistency was 0.65 at baseline but above 0.8 at later assessments. Relationships between the CDR-SB and other measures were as expected (higher correlations with more closely related constructs), and the CDR-SB differentiated between patients with different severities of dementia (-2.9 points difference between CDR-Global Score 0.5 and 1, P<.0001). Floor and ceiling effects on the CDR-SB total score were minimal; however, at baseline there were ceiling effects in the personal care domain. Further detail is provided on the psychometric properties of ADAS-Cog, MMSE, FCSRT-IR and FAQ in this population.
The psychometric properties of the CDR-SB are adequate in prodromal AD and continued use is warranted in clinical trials. However, there remains scope for improvement in the assessment of functional constructs and development of novel measures should continue.
临床痴呆评定量表-和箱式测验（CDR-SB）已被提议作为用于前驱期 AD 试验的主要结局指标。然而，在该患者人群中，尚未很好地确定该量表和其他常用量表的心理测量特性。
描述前驱期 AD 临床试验中常用疗效指标的心理测量特性。
数据是作为为期两年的临床试验的一部分收集的。
患者具有生物标志物确认的前驱期 AD。
临床痴呆评定量表（CDR）、功能活动问卷（FAQ）、阿尔茨海默病评估量表-认知分量表 11 和 13（ADAS-Cog）、简易精神状态检查（MMSE）和自由和线索选择性提醒测试（FCSRT-IR[词]）。评估至少每 24 周进行一次。
对于 CDR-SB，重测信度良好（组内相关系数[ICC]=0.83）；基线时的内部一致性为 0.65，但在后期评估中高于 0.8。CDR-SB 与其他测量指标之间的关系符合预期（与更密切相关的结构相关性更高），并且 CDR-SB 区分了不同痴呆严重程度的患者（CDR-全球评分 0.5 和 1 之间相差 2.9 分，P<.0001）。CDR-SB 总分的地板和天花板效应最小；然而，在基线时，个人护理领域存在天花板效应。在该人群中，还提供了 ADAS-Cog、MMSE、FCSRT-IR 和 FAQ 的心理测量特性的详细信息。
CDR-SB 在前驱期 AD 中的心理测量特性良好，在临床试验中继续使用是合理的。然而，在评估功能结构方面仍有改进的空间，应继续开发新的测量工具。
