Disease severity and minimal clinically important differences in clinical outcome assessments for Alzheimer's disease clinical trials

被引:251
作者
Andrews, J. Scott [1 ]
Desai, Urvi [2 ]
Kirson, Noam Y. [2 ]
Zichlin, Miriam L. [2 ]
Ball, Daniel E. [1 ]
Matthews, Brandy R. [1 ]
机构
[1] Eli Lilly & Co, Lilly Corp Ctr, Indianapolis, IN 46285 USA
[2] Anal Grp Inc, Boston, MA 02199 USA
关键词
Alzheimer's disease; MCID; MMSE; CDR; FAQ; Meaningful decline; HEALTH-STATUS; TASK-FORCE;
D O I
10.1016/j.trci.2019.06.005
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
IntroductionThis study estimated the minimal clinically important difference (MCID) for Mini Mental State Examination, Clinical Dementia Rating Scale sum of boxes, and Functional Activities Questionnaire across the Alzheimer's disease (AD) spectrum. MethodsRetrospective analysis of the National Alzheimer's Coordinating Center Uniform Data Set (9/2005-9/2016) and MCID for clinical outcomes were estimated using anchor-based (clinician's assessment of meaningful decline) and distribution-based (1/2 baseline standard deviation) approaches, stratified by severity of cognitive impairment. ResultsOn average, a 1-3 point decrease in Mini Mental State Examination, 1-2 point increase in Clinical Dementia Scale sum of boxes, and 3-5 point increase in Functional Activities Questionnaire were indicative of a meaningful decline. The MCID values generally increased by disease severity; the effect size and standardized response mean for those with meaningful decline were consistently in the acceptable ranges for MCID. DiscussionThese findings can inform design and interpretation of future clinical trials.
引用
收藏
页码:354 / 363
页数:10
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