Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, The Sahlgrenska Academy, University of Gothenburg, Sweden.
Wallenberg Centre for Molecular and Translational Medicine, University of Gothenburg, Sweden.
Brain. 2021 Feb 12;144(1):325-339. doi: 10.1093/brain/awaa399.
Tau phosphorylated at threonine 181 (p-tau181) measured in blood plasma has recently been proposed as an accessible, scalable, and highly specific biomarker for Alzheimer's disease. Longitudinal studies, however, investigating the temporal dynamics of this novel biomarker are lacking. It is therefore unclear when in the disease process plasma p-tau181 increases above physiological levels and how it relates to the spatiotemporal progression of Alzheimer's disease characteristic pathologies. We aimed to establish the natural time course of plasma p-tau181 across the sporadic Alzheimer's disease spectrum in comparison to those of established imaging and fluid-derived biomarkers of Alzheimer's disease. We examined longitudinal data from a large prospective cohort of elderly individuals enrolled in the Alzheimer's Disease Neuroimaging Initiative (ADNI) (n = 1067) covering a wide clinical spectrum from normal cognition to dementia, and with measures of plasma p-tau181 and an 18F-florbetapir amyloid-β PET scan at baseline. A subset of participants (n = 864) also had measures of amyloid-β1-42 and p-tau181 levels in CSF, and another subset (n = 298) had undergone an 18F-flortaucipir tau PET scan 6 years later. We performed brain-wide analyses to investigate the associations of plasma p-tau181 baseline levels and longitudinal change with progression of regional amyloid-β pathology and tau burden 6 years later, and estimated the time course of changes in plasma p-tau181 and other Alzheimer's disease biomarkers using a previously developed method for the construction of long-term biomarker temporal trajectories using shorter-term longitudinal data. Smoothing splines demonstrated that earliest plasma p-tau181 changes occurred even before amyloid-β markers reached abnormal levels, with greater rates of change correlating with increased amyloid-β pathology. Voxel-wise PET analyses yielded relatively weak, yet significant, associations of plasma p-tau181 with amyloid-β pathology in early accumulating brain regions in cognitively healthy individuals, while the strongest associations with amyloid-β were observed in late accumulating regions in patients with mild cognitive impairment. Cross-sectional and particularly longitudinal measures of plasma p-tau181 were associated with widespread cortical tau aggregation 6 years later, covering temporoparietal regions typical for neurofibrillary tangle distribution in Alzheimer's disease. Finally, we estimated that plasma p-tau181 reaches abnormal levels ∼6.5 and 5.7 years after CSF and PET measures of amyloid-β, respectively, following similar dynamics as CSF p-tau181. Our findings suggest that plasma p-tau181 increases are associated with the presence of widespread cortical amyloid-β pathology and with prospective Alzheimer's disease typical tau aggregation, providing clear implications for the use of this novel blood biomarker as a diagnostic and screening tool for Alzheimer's disease.
在血浆中检测到的磷酸化苏氨酸 181 位的 tau(p-tau181)最近被提议作为一种易于获取、可扩展且高度特异的阿尔茨海默病生物标志物。然而,缺乏关于这种新型生物标志物的时间动态的纵向研究。因此,尚不清楚在疾病过程中血浆 p-tau181 何时升高超过生理水平,以及它与阿尔茨海默病特征性病理的时空进展有何关系。我们旨在确定在阿尔茨海默病谱的散发性阿尔茨海默病患者中,血浆 p-tau181 的自然时间过程,并与阿尔茨海默病的已建立的成像和液体制剂生物标志物进行比较。我们检查了来自阿尔茨海默病神经影像学倡议(ADNI)的大型前瞻性队列中老年人的纵向数据(n=1067),这些人涵盖了从正常认知到痴呆的广泛临床谱,并且在基线时进行了血浆 p-tau181 和 18F-氟比他滨淀粉样蛋白-β PET 扫描测量。部分参与者(n=864)还进行了脑脊液中淀粉样蛋白-β1-42 和 p-tau181 水平的测量,另一部分参与者(n=298)在 6 年后进行了 18F-氟ortaucipir tau PET 扫描。我们进行了全脑分析,以研究血浆 p-tau181 基线水平和纵向变化与 6 年后区域淀粉样蛋白-β病理和 tau 负担进展的关系,并使用先前开发的方法估计了血浆 p-tau181 和其他阿尔茨海默病生物标志物的时间过程,该方法用于使用较短的纵向数据构建长期生物标志物时间轨迹。平滑样条表明,最早的血浆 p-tau181 变化甚至发生在淀粉样蛋白-β标志物达到异常水平之前,并且变化率越高,与淀粉样蛋白-β病理的相关性就越大。体素 PET 分析在认知健康个体的早期积累的脑区中产生了与血浆 p-tau181 与淀粉样蛋白-β病理的相对较弱但具有统计学意义的关联,而在轻度认知障碍患者的晚期积累区则观察到与淀粉样蛋白-β最强的关联。血浆 p-tau181 的横断面和特别是纵向测量与 6 年后广泛的皮质 tau 聚集有关,涵盖了阿尔茨海默病神经原纤维缠结分布的颞顶区。最后,我们估计,血浆 p-tau181 在脑脊液和 PET 测量淀粉样蛋白-β后分别达到异常水平约 6.5 和 5.7 年,其动力学与脑脊液 p-tau181 相似。我们的发现表明,血浆 p-tau181 的增加与广泛的皮质淀粉样蛋白-β病理和与前瞻性阿尔茨海默病典型 tau 聚集有关,这为将这种新型血液生物标志物用作阿尔茨海默病的诊断和筛查工具提供了明确的依据。