McGill Center for Studies in Aging, Douglas Mental Health Research Institute, Montreal, Que., Canada.
Neurodegener Dis. 2014;13(2-3):197-9. doi: 10.1159/000353748. Epub 2013 Aug 7.
Randomized clinical trials (RCTs) for putative disease-modifying drugs in Alzheimer's disease (AD) are using cognitive outcomes, such as the Alzheimer's Disease Assessment Scale--cognitive subscale, activities of daily living scales, such as the Alzheimer's Disease Cooperative Study Activities of Daily Living, and time from mild cognitive impairment to AD dementia.
It was the aim of this study to build clinically relevant outcomes for future use in clinical practice into RCT designs and help third-party payers to measure benefit.
We used a literature review for analysis.
The Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) appears to be the most reliable primary outcome for RCT at different stages of AD, with the Relevant Outcome Scale for Alzheimer's Disease (ROSA) as a suitable alternative. The importance of current AD biomarkers vis-à- vis determination of efficacy of disease-modifying drugs has yet to be established; however, it is likely that at least one amyloid-specific test will be required prior to treatment with a drug acting predominantly on β-amyloid (Aβ42). Furthermore, serial MRI may be required to monitor adverse side effects associated with such drugs.
Global clinical scales such as CDR-SB and ROSA should be considered for use with treatments aiming at slowing disease progression.
针对阿尔茨海默病（AD）潜在的疾病修饰药物的随机临床试验（RCT）正在使用认知结果，如阿尔茨海默病评估量表-认知子量表、日常生活活动量表，如阿尔茨海默病合作研究日常生活活动量表，以及从轻度认知障碍到 AD 痴呆的时间。
本研究旨在将未来临床实践中使用的临床相关结果纳入 RCT 设计，并帮助第三方支付者衡量获益。
我们使用文献回顾进行分析。
临床痴呆评定量表总评分（CDR-SB）似乎是 AD 不同阶段 RCT 的最可靠的主要结局，相关的阿尔茨海默病结局量表（ROSA）是一种合适的替代方法。当前 AD 生物标志物在确定疾病修饰药物疗效方面的重要性尚未确定；然而，在使用主要作用于β-淀粉样蛋白（Aβ42）的药物治疗之前，很可能至少需要进行一项淀粉样蛋白特异性检测。此外，可能需要进行连续 MRI 以监测与这些药物相关的不良反应。
对于旨在减缓疾病进展的治疗方法，应考虑使用全球临床量表，如 CDR-SB 和 ROSA。
