Andrea R. Zammit, Ana W. Capuano, Lisa L. Barnes, Julie A. Schneider, Reisa A. Sperling, David A. Bennett, Francine Grodstein
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Bennett, Francine Grodstein","doi":"10.1002/trc2.70137","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> INTRODUCTION</h3>\n \n <p>We evaluated the statistical power for a theoretical randomized trial of anti-amyloid treatment in preclinical Alzheimer's Disease across five cognitive composites in preclinical Alzheimer's Disease across five cognitive composites: Alzheimer's Prevention Initiative Preclinical Composite Cognitive Test (APCC); Preclinical Alzheimer's Composite with Semantic Processing (PACC5); Preclinical Alzheimer's Cognitive Composite (PACC); and global and episodic memory composites.</p>\n </section>\n \n <section>\n \n <h3> METHODS</h3>\n \n <p>We utilized annual cognitive assessments from 517 decedents (78.2 ± 4.7years; 72% female) with <i>post mortem</i> pathologic Alzheimer's disease (AD) to represent amyloid positivity. We calculated sample sizes to detect 30% reduction in 5-year slopes of cognitive decline for equal size treatment versus placebo groups across composites.</p>\n </section>\n \n <section>\n \n <h3> RESULTS</h3>\n \n <p>Estimated sample sizes for APCC (<i>n</i> = 1633, 95% confidence interval [CI] 1400–1823), PACC (<i>n</i> = 1822, 95% CI 1612–2122), and episodic memory (<i>n</i> = 3141 95%CI 2563–3732) were larger than for PACC5 (<i>n</i> = 1424, 95% CI 1249–1575). Sample size estimates were similar between PACC5 and the global composite (<i>n</i> = 1267, 95%CI 1336–1407).</p>\n </section>\n \n <section>\n \n <h3> DISCUSSION</h3>\n \n <p>Small changes in composites, such as addition of semantic fluency in PACC5, could be considered as part of approaches to improve statistical power.</p>\n </section>\n \n <section>\n \n <h3> HIGHLIGHTS</h3>\n \n <div>\n <ul>\n \n <li>We evaluated statistical power of a theoretical 5-year randomized trial testing anti-amyloid treatments in early Alzheimer's across five cognitive composite endpoints.</li>\n \n <li>We leveraged annual cognitive assessment in Rush Alzheimer's Disease Center cohorts and used <i>post mortem</i> pathologic AD to represent amyloid positivity.</li>\n \n <li>Preclinical Alzheimer's Composite with Semantic Processing (PACC5) required significantly lower sample size to achieve power for a 30% reduction in cognitive slope than Alzheimer's Disease Cooperative Study-Preclinical Alzheimer's Cognitive Composite (PACC).</li>\n \n <li>PACC5 had better statistical power than Alzheimer's Prevention Initiative Preclinical Composite Cognitive Test (APCC) and an episodic memory composite.</li>\n \n <li>Small changes in cognitive composites can improve detection of cognitive decline.</li>\n </ul>\n </div>\n </section>\n </div>","PeriodicalId":53225,"journal":{"name":"Alzheimer''s and Dementia: Translational Research and Clinical Interventions","volume":"11 3","pages":""},"PeriodicalIF":4.9000,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/trc2.70137","citationCount":"0","resultStr":"{\"title\":\"Cognitive decline across five cognitive batteries: Sample size implications for clinical trials\",\"authors\":\"Andrea R. 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Cognitive decline across five cognitive batteries: Sample size implications for clinical trials
INTRODUCTION
We evaluated the statistical power for a theoretical randomized trial of anti-amyloid treatment in preclinical Alzheimer's Disease across five cognitive composites in preclinical Alzheimer's Disease across five cognitive composites: Alzheimer's Prevention Initiative Preclinical Composite Cognitive Test (APCC); Preclinical Alzheimer's Composite with Semantic Processing (PACC5); Preclinical Alzheimer's Cognitive Composite (PACC); and global and episodic memory composites.
METHODS
We utilized annual cognitive assessments from 517 decedents (78.2 ± 4.7years; 72% female) with post mortem pathologic Alzheimer's disease (AD) to represent amyloid positivity. We calculated sample sizes to detect 30% reduction in 5-year slopes of cognitive decline for equal size treatment versus placebo groups across composites.
RESULTS
Estimated sample sizes for APCC (n = 1633, 95% confidence interval [CI] 1400–1823), PACC (n = 1822, 95% CI 1612–2122), and episodic memory (n = 3141 95%CI 2563–3732) were larger than for PACC5 (n = 1424, 95% CI 1249–1575). Sample size estimates were similar between PACC5 and the global composite (n = 1267, 95%CI 1336–1407).
DISCUSSION
Small changes in composites, such as addition of semantic fluency in PACC5, could be considered as part of approaches to improve statistical power.
HIGHLIGHTS
We evaluated statistical power of a theoretical 5-year randomized trial testing anti-amyloid treatments in early Alzheimer's across five cognitive composite endpoints.
We leveraged annual cognitive assessment in Rush Alzheimer's Disease Center cohorts and used post mortem pathologic AD to represent amyloid positivity.
Preclinical Alzheimer's Composite with Semantic Processing (PACC5) required significantly lower sample size to achieve power for a 30% reduction in cognitive slope than Alzheimer's Disease Cooperative Study-Preclinical Alzheimer's Cognitive Composite (PACC).
PACC5 had better statistical power than Alzheimer's Prevention Initiative Preclinical Composite Cognitive Test (APCC) and an episodic memory composite.
Small changes in cognitive composites can improve detection of cognitive decline.
期刊介绍：
Alzheimer''s & Dementia: Translational Research & Clinical Interventions (TRCI) is a peer-reviewed, open access,journal from the Alzheimer''s Association®. The journal seeks to bridge the full scope of explorations between basic research on drug discovery and clinical studies, validating putative therapies for aging-related chronic brain conditions that affect cognition, motor functions, and other behavioral or clinical symptoms associated with all forms dementia and Alzheimer''s disease. The journal will publish findings from diverse domains of research and disciplines to accelerate the conversion of abstract facts into practical knowledge: specifically, to translate what is learned at the bench into bedside applications. The journal seeks to publish articles that go beyond a singular emphasis on either basic drug discovery research or clinical research. Rather, an important theme of articles will be the linkages between and among the various discrete steps in the complex continuum of therapy development. For rapid communication among a multidisciplinary research audience involving the range of therapeutic interventions, TRCI will consider only original contributions that include feature length research articles, systematic reviews, meta-analyses, brief reports, narrative reviews, commentaries, letters, perspectives, and research news that would advance wide range of interventions to ameliorate symptoms or alter the progression of chronic neurocognitive disorders such as dementia and Alzheimer''s disease. The journal will publish on topics related to medicine, geriatrics, neuroscience, neurophysiology, neurology, psychiatry, clinical psychology, bioinformatics, pharmaco-genetics, regulatory issues, health economics, pharmacoeconomics, and public health policy as these apply to preclinical and clinical research on therapeutics.
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