Premi Enrico, Grassi Mario, van Swieten John, Galimberti Daniela, Graff Caroline, Masellis Mario, Tartaglia Carmela, Tagliavini Fabrizio, Rowe James B, Laforce Robert, Finger Elizabeth, Frisoni Giovanni B, de Mendonça Alexandre, Sorbi Sandro, Gazzina Stefano, Cosseddu Maura, Archetti Silvana, Gasparotti Roberto, Manes Marta, Alberici Antonella, Cardoso Manuel J, Bocchetta Martina, Cash David M, Ourselin Sebastian, Padovani Alessandro, Rohrer Jonathan D, Borroni Barbara
Neurology Unit, Department of Clinical and Experimental Sciences, University of Brescia, Brescia, Italy.
Department of Brain and Behavioral Science, Medical and Genomic Statistics Unit, University of Pavia, Italy.
Brain. 2017 Jun 1;140(6):1784-1791. doi: 10.1093/brain/awx103.
Frontotemporal dementia is a heterogeneous neurodegenerative disorder with around a third of cases having autosomal dominant inheritance. There is wide variability in phenotype even within affected families, raising questions about the determinants of the progression of disease and age at onset. It has been recently demonstrated that cognitive reserve, as measured by years of formal schooling, can counteract the ongoing pathological process. The TMEM106B genotype has also been found to be a modifier of the age at disease onset in frontotemporal dementia patients with TDP-43 pathology. This study therefore aimed to elucidate the modulating effect of environment (i.e. cognitive reserve as measured by educational attainment) and genetic background (i.e. TMEM106B polymorphism, rs1990622 T/C) on grey matter volume in a large cohort of presymptomatic subjects bearing frontotemporal dementia-related pathogenic mutations. Two hundred and thirty-one participants from the GENFI study were included: 108 presymptomatic MAPT, GRN, and C9orf72 mutation carriers and 123 non-carriers. For each subject, cortical and subcortical grey matter volumes were generated using a parcellation of the volumetric T1-weighted magnetic resonance imaging brain scan. TMEM106B genotyping was carried out, and years of education recorded. First, we obtained a composite measure of grey matter volume by graph-Laplacian principal component analysis, and then fitted a linear mixed-effect interaction model, considering the role of (i) genetic status; (ii) educational attainment; and (iii) TMEM106B genotype on grey matter volume. The presence of a mutation was associated with a lower grey matter volume (P = 0.002), even in presymptomatic subjects. Education directly affected grey matter volume in all the samples (P = 0.02) with lower education attainment being associated with lower volumes. TMEM106B genotype did not influence grey matter volume directly on its own but in mutation carriers it modulated the slope of the correlation between education and grey matter volume (P = 0.007). Together, these results indicate that brain atrophy in presymptomatic carriers of common frontotemporal dementia mutations is affected by both genetic and environmental factors such that TMEM106B enhances the benefit of cognitive reserve on brain structure. These findings should be considered in evaluating outcomes in future disease-modifying trials, and support the search for protective mechanisms in people at risk of dementia that might facilitate new therapeutic strategies.
额颞叶痴呆是一种异质性神经退行性疾病,约三分之一的病例为常染色体显性遗传。即使在受影响的家族中,其表型也存在很大差异,这引发了关于疾病进展的决定因素和发病年龄的问题。最近有研究表明,通过正规教育年限衡量的认知储备可以抵消正在进行的病理过程。TMEM106B基因型也被发现是TDP-43病理型额颞叶痴呆患者发病年龄的一个调节因子。因此,本研究旨在阐明环境因素(即通过教育程度衡量的认知储备)和遗传背景(即TMEM106B多态性,rs1990622 T/C)对一大群携带额颞叶痴呆相关致病突变的症状前受试者脑灰质体积的调节作用。纳入了GENFI研究中的231名参与者:108名症状前MAPT、GRN和C9orf72突变携带者以及123名非携带者。对于每个受试者,使用容积性T1加权磁共振成像脑部扫描的分割方法生成皮质和皮质下灰质体积。进行了TMEM106B基因分型,并记录了教育年限。首先,我们通过图拉普拉斯主成分分析获得了灰质体积的综合测量值,然后拟合了一个线性混合效应交互模型,考虑了(i)遗传状态;(ii)教育程度;以及(iii)TMEM106B基因型对灰质体积的作用。即使在症状前受试者中,突变的存在也与较低的灰质体积相关(P = 0.002)。教育直接影响所有样本中的灰质体积(P = 0.02),教育程度较低与灰质体积较低相关。TMEM106B基因型本身并不直接影响灰质体积,但在突变携带者中,它调节了教育与灰质体积之间相关性的斜率(P = 0.007)。总之,这些结果表明,常见额颞叶痴呆突变的症状前携带者的脑萎缩受到遗传和环境因素的影响,使得TMEM106B增强了认知储备对脑结构的益处。在评估未来疾病修饰试验的结果时应考虑这些发现,并支持在有痴呆风险的人群中寻找可能有助于新治疗策略的保护机制。