German Center for Neurodegenerative Diseases (DZNE), Munich, Germany.
Department of Neurology, Ludwig-Maximilians-Universität München, Munich, Germany.
Brain. 2019 May 1;142(5):1429-1440. doi: 10.1093/brain/awz050.
Owing to an early and marked deposition of amyloid-β in the basal ganglia, autosomal dominant Alzheimer's disease could distinctly involve motor symptoms. Therefore, we aimed to assess the prevalence and characteristics of motor signs in autosomal dominant Alzheimer's disease. Baseline Unified Parkinson Disease Rating Scale part three scores (UPDRS-III) from 433 participants of the Dominantly Inherited Alzheimer's Network observational study were analysed. Motor symptoms were scrutinized with respect to associations with mutation carrier status, mutation site within PSEN1, basal ganglia amyloid-β as measured by Pittsburgh compound B PET, estimated years to symptom onset and Clinical Dementia Rating Scale-Sum of Boxes. Motor findings in mutation carriers were compared to patients with sporadic Alzheimer's disease using data of the National Alzheimer's Coordination Center. Mutation carriers showed motor findings at a higher frequency (28.4% versus 12.8%; P < 0.001) and severity (mean UPDRS-III scores 2.0 versus 0.4; P < 0.001) compared to non-carriers. Eleven of the 27 UPDRS-III items were statistically more frequently affected in mutation carriers after adjustment for multiple comparisons. Ten of these 11 items were subscale components of bradykinesia. In cognitively asymptomatic mutation carriers, dysdiadochokinesia was more frequent compared to non-carriers (right hand: 3.8% versus 0%; adjusted P = 0.023; left: 4.4% versus 0.6%; adjusted P = 0.031). In this cohort, the positive predictive value for mutation carrier status in cognitively asymptomatic participants (50% a priori risk) of dysdiadochokinesia was 100% for the right and 87.5% for the left side. Mutation carriers with motor findings more frequently were basal ganglia amyloid-β positive (84% versus 63.3%; P = 0.006) and showed more basal ganglia amyloid-β deposition (Pittsburgh compound B-standardized uptake value ratio 2.472 versus 1.928; P = 0.002) than those without. Frequency and severity of motor findings were greater in post-codon 200 PSEN1 mutations (36%; mean UPDRS-III score 3.03) compared to mutations pre-codon 200 PSEN1 (19.3%, P = 0.022; 0.91, P = 0.013). In mutation carriers, motor symptom severity was significantly positively correlated with basal ganglia amyloid-β deposition, Clinical Dementia Rating scores and estimated years to symptom onset. Mutation carriers with a Clinical Dementia Rating global score of 2 exhibited more pronounced motor symptoms than sporadic Alzheimer's disease patients with the same Clinical Dementia Rating global score (mean UPDRS-III scores 20.71 versus 5.96; P < 0.001). With a prevalence of approximately 30% and increasing severity with progression of dementia, motor symptoms are proven as a clinically relevant finding in autosomal dominant Alzheimer's disease, in particular in advanced dementia stages, that correlates with deposition of amyloid-β in the basal ganglia. In a very small per cent of cognitively asymptomatic members of families with autosomal dominant Alzheimer's disease, dysdiadochokinesia may increase the chance of an individual's status as mutation carrier.
由于淀粉样蛋白-β在基底神经节的早期和明显沉积,常染色体显性阿尔茨海默病可能明显涉及运动症状。因此,我们旨在评估常染色体显性阿尔茨海默病中运动体征的患病率和特征。对来自显性遗传性阿尔茨海默病网络观察性研究的 433 名参与者的基线统一帕金森病评定量表第三部分评分(UPDRS-III)进行了分析。根据突变携带者状态、PSEN1 中的突变部位、基底神经节淀粉样蛋白-β(通过匹兹堡化合物 B PET 测量)、估计的发病年限和临床痴呆评定量表-总分进行了运动症状的检查。使用国家阿尔茨海默病协调中心的数据,将突变携带者的运动发现与散发性阿尔茨海默病患者进行了比较。与非携带者相比,突变携带者的运动发现频率更高(28.4%比 12.8%;P < 0.001),严重程度更高(平均 UPDRS-III 评分 2.0 比 0.4;P < 0.001)。在调整了多次比较后,11 个 UPDRS-III 项目中有 11 个在统计学上更频繁地受到突变携带者的影响。这 11 个项目中有 10 个是运动迟缓的亚量表组成部分。在认知无症状的突变携带者中,与非携带者相比,运动失调更常见(右手:3.8%比 0%;调整后的 P = 0.023;左手:4.4%比 0.6%;调整后的 P = 0.031)。在该队列中,认知无症状参与者(先验风险 50%)中运动失调的突变携带者状态阳性预测值为右手 100%,左手 87.5%。有运动发现的突变携带者更常为基底神经节淀粉样蛋白-β阳性(84%比 63.3%;P = 0.006),且基底神经节淀粉样蛋白-β沉积更多(匹兹堡化合物 B-标准化摄取值比 2.472 比 1.928;P = 0.002)。与密码子 200 前 PSEN1 突变(36%,平均 UPDRS-III 评分 3.03)相比,密码子 200 后 PSEN1 突变(19.3%,P = 0.022;0.91,P = 0.013)的运动发现频率和严重程度更高。在突变携带者中,运动症状严重程度与基底神经节淀粉样蛋白-β沉积、临床痴呆评定评分和发病年限呈显著正相关。在临床痴呆评定总分为 2 的突变携带者中,与具有相同临床痴呆评定总评分的散发性阿尔茨海默病患者相比,运动症状更为明显(平均 UPDRS-III 评分 20.71 比 5.96;P < 0.001)。大约 30%的常染色体显性阿尔茨海默病患者存在运动症状,且随着痴呆的进展,严重程度逐渐增加,运动症状是常染色体显性阿尔茨海默病中一种具有临床意义的发现,特别是在晚期痴呆阶段,与基底神经节中淀粉样蛋白-β的沉积相关。在常染色体显性阿尔茨海默病家族中认知无症状的极少数成员中,运动失调可能会增加个体突变携带者状态的可能性。