Department of Neurology, Rhode Island Hospital & Alpert Medical School of Brown University, Providence, RI, USA.
Eli Lilly & Co, Inc., Indianapolis, IN, USA.
Alzheimers Dement. 2014 Nov;10(6):853-60. doi: 10.1016/j.jalz.2014.07.158. Epub 2014 Nov 15.
Several lines of evidence from Alzheimer's disease (AD) research continue to support the notion that the biological changes associated with AD are occurring possibly several decades before an individual will experience the cognitive and functional changes associated with the disease. The National Institute on Aging-Alzheimer's Association revised criteria for AD provided a framework for this new thinking. As a result of this growing understanding, several research efforts have launched or will be launching large secondary prevention trials in AD. These and other efforts have clearly demonstrated a need for better measures of cognitive and functional change in people with the earliest changes associated with AD. Recent draft guidance from the US Food and Drug Administration further elevated the importance of cognitive and functional assessments in early stage clinical trials by proposing that even in the pre-symptomatic stages of the disease, approval will be contingent on demonstrating clinical meaningfulness. The Alzheimer's Association's Research Roundtable addressed these issues at its fall meeting October 28-29, 2013, in Washington, D.C. The focus of the discussion included the need for improved cognitive and functional outcome measures for clinical of participants with preclinical AD and those diagnosed with Mild Cognitive Impairment due to AD.
来自阿尔茨海默病（AD）研究的几条证据线继续支持这样一种观点，即与 AD 相关的生物学变化可能在个体经历与疾病相关的认知和功能变化之前几十年就已经发生。美国国家老龄化研究所-阿尔茨海默病协会修订的 AD 标准为这种新思维提供了一个框架。由于这种理解的不断加深，一些研究已经或即将在 AD 中开展大型二级预防试验。这些和其他努力清楚地表明，需要更好的认知和功能变化测量方法来衡量与 AD 相关的最早变化的人。美国食品和药物管理局（FDA）的最新草案指南通过提出即使在疾病的无症状阶段，批准也将取决于证明临床意义，进一步提高了认知和功能评估在早期临床试验中的重要性。阿尔茨海默病协会的研究圆桌会议于 2013 年 10 月 28 日至 29 日在华盛顿特区举行秋季会议，讨论了这些问题。讨论的重点包括需要改善临床前 AD 参与者和因 AD 导致轻度认知障碍的参与者的认知和功能结果测量方法。
