Doody Rachelle S
Baylor College of Medicine, Houston, TX, USA.
Alzheimers Dement. 2008 Jan;4(1 Suppl 1):S21-5. doi: 10.1016/j.jalz.2007.10.010. Epub 2007 Dec 21.
The terms symptomatic and disease-modifying have become standard in discussions of Alzheimer's disease therapeutics, yet there is little justification for their use. Currently marketed drugs are presumed to be symptomatic because they lead to some degree of mean improvement over baseline and because of the widespread belief that their mechanisms are limited and their effects are completely reversible. Current trial methodologies cannot distinguish between symptomatic and disease-modifying effects. Furthermore, it is highly likely that many trials will demonstrate a combination of such effects at the level of the trial or at the level of the individual. The forces that drive this distinction are largely social, as opposed to scientific. It would be preferable for drugs to first seek an antidementia claim, preferably on the background of conventional therapies when possible, in trials that are as small and as short in duration as possible. Further refinements would come by demonstration of how substantial and how enduring the antidementia benefits are.
“对症的”和“疾病修饰性的”这两个术语在阿尔茨海默病治疗学的讨论中已成为标准术语，但它们的使用几乎没有依据。目前上市的药物被认为是对症的，因为它们相较于基线水平能带来一定程度的平均改善，还因为人们普遍认为其作用机制有限且效果完全可逆。当前的试验方法无法区分对症效果和疾病修饰性效果。此外，很有可能许多试验会在试验层面或个体层面显示出这两种效果的组合。推动这种区分的力量很大程度上是社会因素，而非科学因素。药物最好在尽可能小规模、短疗程的试验中，首先寻求抗痴呆的认定，如有可能，最好以传统疗法为背景。通过证明抗痴呆益处的显著程度和持久程度，可进一步完善这一认定。
