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Original Contribution |

Amyloid-β–Associated Clinical Decline Occurs Only in the Presence of Elevated P-tau

Rahul S. Desikan, MD, PhD; Linda K. McEvoy, PhD; Wesley K. Thompson, PhD; Dominic Holland, PhD; James B. Brewer, MD, PhD; Paul S. Aisen, MD; Reisa A. Sperling, MD; Anders M. Dale, PhD; for the Alzheimer's Disease Neuroimaging Initiative
Arch Neurol. 2012;69(6):709-713. doi:10.1001/archneurol.2011.3354.
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Objective  To elucidate the relationship between the 2 hallmark proteins of Alzheimer disease (AD), amyloid-β (Aβ) and tau, and clinical decline over time among cognitively normal older individuals.

Design  A longitudinal cohort of clinically and cognitively normal older individuals assessed with baseline lumbar puncture and longitudinal clinical assessments.

Setting  Research centers across the United States and Canada.

Patients  We examined 107 participants with a Clinical Dementia Rating (CDR) of 0 at baseline examination.

Main Outcome Measures  Using linear mixed effects models, we investigated the relationship between cerebrospinal fluid (CSF) phospho-tau 181(p-tau181p), CSF Aβ1-42, and clinical decline as assessed using longitudinal change in global CDR, CDR–Sum of Boxes, and the Alzheimer Disease Assessment Scale–cognitive subscale.

Results  We found a significant relationship between decreased CSF Aβ1-42 and longitudinal change in global CDR, CDR–Sum of Boxes, and Alzheimer Disease Assessment Scale–cognitive subscale in individuals with elevated CSF p-tau181p. In the absence of CSF p-tau181p, the effect of CSF Aβ1-42 on longitudinal clinical decline was not significantly different from 0.

Conclusions  In cognitively normal older individuals, Aβ-associated clinical decline during a mean of 3 years may occur only in the presence of ongoing downstream neurodegeneration.

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Grahic Jump Location

Figure. Box and whisker plots for all participants illustrating changes in Clinical Dementia Rating–Sum of Boxes (CDR-SB) (A) and Alzheimer Disease Assessment Scale-cognitive (ADAS-cog) subscale (B) scores measured as annualized percentage change based on cerebrospinal fluid amyloid-β1-42 (Aβ1-42) and cerebrospinal fluid phospho-tau 181 (p-tau181p) status. For each plot, the thick black lines show the median value. Regions above and below the black line show the upper and lower quartiles, respectively. The dashed lines extend to the minimum and maximum values, with outliers shown as circles. As illustrated, the Aβ-positive/p-tau–positive individuals demonstrated the largest change in CDR-SB and ADAS-cog scores.

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