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000279454 0247_ $$2doi$$a10.1016/j.tjpad.2025.100133
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000279454 1001_ $$aCash, David M$$b0
000279454 245__ $$aSample size estimates for biomarker-based outcome measures in clinical trials in autosomal dominant Alzheimer's disease.
000279454 260__ $$aCham$$bSpringer International Publishing$$c2025
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000279454 520__ $$aAlzheimer disease (AD)-modifying therapies are approved for treatment of early-symptomatic AD. Autosomal dominant AD (ADAD) provides a unique opportunity to test therapies in presymptomatic individuals.Using data from the Dominantly Inherited Alzheimer Network (DIAN), sample sizes for clinical trials were estimated for various cognitive, imaging, and CSF outcomes. Sample sizes were computed for detecting a reduction of either absolute levels of AD-related pathology (amyloid, tau) or change over time in neurodegeneration (atrophy, hypometabolism, cognitive change).Biomarkers measuring amyloid and tau pathology had required sample sizes below 200 participants per arm (examples CSF Aβ42/40: 47[95 %CI 25,104], cortical PIB 49[28,99], CSF p-tau181 74[48,125]) for a four-year trial in presymptomatic individuals (CDR=0) to have 80 % power (5 % statistical significance) to detect a 25 % reduction in absolute levels of pathology, allowing 40 % dropout. For cognitive, MRI, and FDG, it was more appropriate to detect a 50 % reduction in rate of change. Sample sizes ranged from 250 to 900 (examples hippocampal volume: 338[131,2096], cognitive composite: 326[157,1074]). MRI, FDG and cognitive outcomes had lower sample sizes when including indivduals with mild impairment (CDR=0.5 and 1) as well as presymptomatic individuals (CDR=0).Despite the rarity of ADAD, presymptomatic clinical trials with feasible sample sizes given the number of cases appear possible.
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000279454 650_7 $$2Other$$aAlzheimer's disease
000279454 650_7 $$2Other$$aAutosomal dominant
000279454 650_7 $$2Other$$aCSF
000279454 650_7 $$2Other$$aClinical trials
000279454 650_7 $$2Other$$aLinear mixed effects models
000279454 650_7 $$2Other$$aLongitudinal
000279454 650_7 $$2Other$$aMRI
000279454 650_7 $$2Other$$aPET
000279454 650_7 $$2Other$$aSample size
000279454 650_7 $$2Other$$aß-amyloid
000279454 650_7 $$2NLM Chemicals$$aBiomarkers
000279454 650_7 $$2NLM Chemicals$$aAmyloid beta-Peptides
000279454 650_7 $$2NLM Chemicals$$atau Proteins
000279454 650_2 $$2MeSH$$aHumans
000279454 650_2 $$2MeSH$$aAlzheimer Disease: genetics
000279454 650_2 $$2MeSH$$aAlzheimer Disease: cerebrospinal fluid
000279454 650_2 $$2MeSH$$aAlzheimer Disease: diagnosis
000279454 650_2 $$2MeSH$$aAlzheimer Disease: diagnostic imaging
000279454 650_2 $$2MeSH$$aAlzheimer Disease: pathology
000279454 650_2 $$2MeSH$$aBiomarkers: cerebrospinal fluid
000279454 650_2 $$2MeSH$$aAmyloid beta-Peptides: cerebrospinal fluid
000279454 650_2 $$2MeSH$$aSample Size
000279454 650_2 $$2MeSH$$atau Proteins: cerebrospinal fluid
000279454 650_2 $$2MeSH$$aMale
000279454 650_2 $$2MeSH$$aFemale
000279454 650_2 $$2MeSH$$aClinical Trials as Topic
000279454 650_2 $$2MeSH$$aOutcome Assessment, Health Care
000279454 650_2 $$2MeSH$$aMagnetic Resonance Imaging
000279454 650_2 $$2MeSH$$aPositron-Emission Tomography
000279454 650_2 $$2MeSH$$aBrain: pathology
000279454 650_2 $$2MeSH$$aBrain: diagnostic imaging
000279454 650_2 $$2MeSH$$aMiddle Aged
000279454 7001_ $$aMorgan, Katy E$$b1
000279454 7001_ $$aO'Connor, Antoinette$$b2
000279454 7001_ $$aVeale, Thomas D$$b3
000279454 7001_ $$aMalone, Ian B$$b4
000279454 7001_ $$aPoole, Teresa$$b5
000279454 7001_ $$aBenzinger, Tammie Ls$$b6
000279454 7001_ $$aGordon, Brian A$$b7
000279454 7001_ $$aIbanez, Laura$$b8
000279454 7001_ $$aLi, Yan$$b9
000279454 7001_ $$aLlibre-Guerra, Jorge J$$b10
000279454 7001_ $$aMcDade, Eric$$b11
000279454 7001_ $$aWang, Guoqiao$$b12
000279454 7001_ $$aChhatwal, Jasmeer P$$b13
000279454 7001_ $$aDay, Gregory S$$b14
000279454 7001_ $$aHuey, Edward$$b15
000279454 7001_ $$0P:(DE-2719)2000010$$aJucker, Mathias$$b16$$udzne
000279454 7001_ $$0P:(DE-2719)2811659$$aLevin, Johannes$$b17$$udzne
000279454 7001_ $$aNiimi, Yoshiki$$b18
000279454 7001_ $$aNoble, James M$$b19
000279454 7001_ $$aRoh, Jee Hoon$$b20
000279454 7001_ $$aSánchez-Valle, Racquel$$b21
000279454 7001_ $$aSchofield, Peter R$$b22
000279454 7001_ $$aBateman, Randall J$$b23
000279454 7001_ $$aFrost, Chris$$b24
000279454 7001_ $$aFox, Nick C$$b25
000279454 7001_ $$aNetwork, Dominantly Inherited Alzheimer$$b26$$eCollaboration Author
000279454 773__ $$0PERI:(DE-600)2782183-3$$a10.1016/j.tjpad.2025.100133$$gVol. 12, no. 6, p. 100133 -$$n6$$p100133$$tThe journal of prevention of Alzheimer's disease$$v12$$x2274-5807$$y2025
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