Swash M, Brooks D N, Day N E, Frith C D, Levy R, Warlow C P
Royal London Hospital, UK.
J Neurol Neurosurg Psychiatry. 1991 Feb;54(2):178-81. doi: 10.1136/jnnp.54.2.178.
Recent advances in Alzheimer's disease imply a need for adequate clinical trials of new treatments which require careful design. The disorder is progressive and shows clinical heterogeneity. While large-scale trials of elderly subjects are appropriate in relation to assessment of drugs or other treatments designed to prevent progression of the disorder, the outcome measurements in such biological treatment trials require careful planning. Studies of individual patients are relevant for answering certain specific questions. Relatively short cross-over trial designs may be appropriate to some pharmacological studies. The choice of neuropsychological instruments for measuring change is critically important, particularly in excluding test/retest artefact and in avoiding floor and ceiling effects. Test scales designed for assessment of specific neuropsychological deficits, or forming part of standard IQ assessments are unlikely to prove robust. Tests can be selected and developed for individual patients, but generalisation of the results of such experiments to the disease as a whole is not inevitable. There is a need to develop psychological instruments for measuring change that are robust and relevant to the clinical problem of progressive dementia.
阿尔茨海默病的最新进展表明，需要对新疗法进行充分的临床试验，而这需要精心设计。该疾病具有进行性且表现出临床异质性。虽然针对老年受试者的大规模试验对于评估旨在预防疾病进展的药物或其他治疗方法是合适的，但此类生物治疗试验中的结果测量需要仔细规划。对个体患者的研究对于回答某些特定问题具有相关性。相对较短的交叉试验设计可能适用于某些药理学研究。选择用于测量变化的神经心理学工具至关重要，特别是在排除测试/重测假象以及避免地板效应和天花板效应方面。为评估特定神经心理学缺陷而设计的测试量表，或作为标准智商评估一部分的量表，不太可能证明是可靠的。可以为个体患者选择和开发测试，但此类实验结果推广到整个疾病并非必然。需要开发用于测量变化的心理工具，这些工具应可靠且与进行性痴呆的临床问题相关。
