Lakhani Dhairya A, Agarwal Amit K, Middlebrooks Erik H
Division of Neuroradiology, Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, USA.
Department of Radiology, Mayo Clinic, USA.
Neuroradiol J. 2024 Apr 21:19714009241247464. doi: 10.1177/19714009241247464.
Fragile X tremor/ataxia syndrome (FXTAS) is an adult-onset neurodegenerative disorder characterized by premutation expansion of fragile X mental retardation 1 (FMR1) gene. It is a common single-gene cause of tremor, ataxia, and cognitive decline in adults. FXTAS affects the central, peripheral and autonomic nervous systems, leading to a range of neurological symptoms from dementia to dysautonomia. A characteristic imaging feature of FXTAS is symmetric T2 hyperintensity in the deep white matter of the cerebellar hemispheres and middle cerebral peduncle. However, recent studies have reported additional findings on diffusion weighted images (DWI), such as a symmetric high-intensity band-like signal at the cerebral corticomedullary junction. These findings, along with the characteristic cerebellar signal alterations, overlap with imaging findings seen in adult-onset neuronal intranuclear inclusion disease (NIID). Importantly, recent pathology studies have shown that both FXTAS and NIID can manifest intranuclear inclusion bodies, posing a diagnostic challenge and potential for misdiagnosis. We describe a 58-year-old man with FXTAS who received an erroneous diagnosis based on imaging and histopathology results. We emphasize the potential pitfalls in distinguishing NIID from FXTAS and stress the importance of genetic analysis in all cases with suspected NIID and FXTAS for confirmation. Additionally, we present the 7T MRI brain findings of FXTAS.
脆性X震颤/共济失调综合征(FXTAS)是一种成人起病的神经退行性疾病,其特征为脆性X智力低下1(FMR1)基因的前突变扩增。它是成人震颤、共济失调和认知衰退常见的单基因病因。FXTAS会影响中枢、外周和自主神经系统,导致从痴呆到自主神经功能障碍等一系列神经症状。FXTAS的一个特征性影像学表现是小脑半球和大脑中脚深部白质的对称性T2高信号。然而,最近的研究报告了扩散加权成像(DWI)上的其他发现,如大脑皮质髓质交界处的对称性高强度带状信号。这些发现,连同特征性的小脑信号改变,与成人起病的神经元核内包涵体病(NIID)的影像学表现重叠。重要的是,最近的病理学研究表明,FXTAS和NIID均可表现出核内包涵体,这带来了诊断挑战和误诊的可能性。我们描述了一名患有FXTAS的58岁男性,其基于影像学和组织病理学结果被误诊。我们强调了区分NIID和FXTAS时的潜在陷阱,并强调在所有疑似NIID和FXTAS的病例中进行基因分析以确诊的重要性。此外,我们展示了FXTAS的7T MRI脑部检查结果。