Clinical Neuroanatomy Section, Department of Neurology, Center for Biomedical Research, University of Ulm.
Department of Neurology, University of Ulm and DZNE.
J Neuropathol Exp Neurol. 2020 Apr 1;79(4):378-392. doi: 10.1093/jnen/nlz139.
Although Gaucher disease can be accompanied by Lewy pathology (LP) and extrapyramidal symptoms, it is unknown if LP exists in Fabry disease (FD), another progressive multisystem lysosomal storage disorder. We aimed to elucidate the distribution patterns of FD-related inclusions and LP in the brain of a 58-year-old cognitively unimpaired male FD patient suffering from predominant hypokinesia. Immunohistochemistry (CD77, α-synuclein, collagen IV) and neuropathological staging were performed on 100-µm sections. Tissue from the enteric or peripheral nervous system was unavailable. As controls, a second cognitively unimpaired 50-year-old male FD patient without LP or motor symptoms and 3 age-matched individuals were examined. Inclusion body pathology was semiquantitatively evaluated. Although Lewy neurites/bodies were not present in the 50-year-old individual or in controls, severe neuronal loss in the substantia nigra pars compacta and LP corresponding to neuropathological stage 4 of Parkinson disease was seen in the 58-year-old FD patient. Major cerebrovascular lesions and/or additional pathologies were absent in this individual. We conclude that Lewy body disease with parkinsonism can occur within the context of FD. Further studies determining the frequencies of both inclusion pathologies in large autopsy-controlled FD cohorts could help clarify the implications of both lesions for disease pathogenesis, potential spreading mechanisms, and therapeutic interventions.
虽然戈谢病可伴有路易体病理(LP)和锥体外系症状,但路易体是否存在于另一种进行性多系统溶酶体贮积病——法布里病(FD)中尚不清楚。我们旨在阐明脑内 FD 相关包涵体和 LP 的分布模式,研究对象为一名认知无障碍的 58 岁男性 FD 患者,其主要表现为运动迟缓。对 100µm 切片进行免疫组织化学(CD77、α-突触核蛋白、胶原 IV)和神经病理学分期检测。无法获得肠或周围神经系统的组织。作为对照,我们还检查了另一名认知无障碍的、无 LP 或运动症状的 50 岁男性 FD 患者和 3 名年龄匹配的个体。对包涵体病理进行半定量评估。尽管在 50 岁个体或对照者中未见路易神经突/体,但在 58 岁 FD 患者中,观察到黑质致密部严重神经元丧失和与帕金森病神经病理学分期 4 相对应的 LP。该个体中无重大脑血管病变和/或其他病变。我们的结论是,路易体病伴帕金森病可发生于 FD 中。进一步的研究确定在大型尸检对照 FD 队列中这两种包涵体病变的频率,可能有助于阐明这两种病变对疾病发病机制、潜在传播机制和治疗干预的影响。