Zhonghua Nei Ke Za Zhi. 2022 Aug 1;61(8):848-859. doi: 10.3760/cma.j.cn112138-20210814-00553.
Hereditary cerebral small vessel disease (hCVSD) is a group of rare cerebrovascular diseases caused by monogenic mutation. Although there is great variability in the age of onset of hCSVD patients, the age of onset younger than 45 years old is an important indicator. The primary clinical manifestations exhibit cognitive impairment, movement disorder, psychological disturbance, and most of which are accompanied by impairment of extra-neurological systems. The main radiological and pathological features of hCVSD include lacunar infarcts, white matter hyperdensities, cerebral microbleeds and enlargement of Virchow-Robin spaces. Current representative diseases include autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy, autosomal recessive arteriopathy with subcortical infarcts and leukoencephalopathy, HTRA1 related autosomal dominant cerebellar small vessel disease, collagen 4A1/2 microangiopathy, retinal vasculopathy with cerebral leukoencephalopathy and systemic manifestations, hereditary cerebral amyloidosis angiopathy, and Fabry disease. In recent years, Chinese scholars have summarized diagnostic, and treatment processes in hCSVD patients. Therefore, we organize experts engaged in the studies of hCVSD, based on the Chinese reports and referring to the relevant foreign literatures, to put forward clinical working recommendations for clinical and imaging characteristics, as well as diagnosis and treatment workflows of different types of hCVSD. The aim of this recommendation is to guide physicians to recognize hCVSD patients from those of stroke in order to obtain timely diagnosis and standard treatment.
遗传性脑小血管病(hCVSD)是一组由单基因变异引起的罕见脑血管疾病。尽管hCSVD患者的发病年龄差异很大,但45岁以下的发病年龄是一个重要指标。主要临床表现为认知障碍、运动障碍、心理障碍,且大多数伴有神经外系统损害。hCVSD的主要影像学和病理学特征包括腔隙性梗死、白质高密度、脑微出血和血管周围间隙扩大。目前具有代表性的疾病包括常染色体显性遗传性脑动脉病伴皮质下梗死和白质脑病、常染色体隐性遗传性脑动脉病伴皮质下梗死和白质脑病、HTRA1相关的常染色体显性遗传性小脑小血管病、胶原4A1/2微血管病、伴有脑白质脑病和全身表现的视网膜血管病、遗传性脑淀粉样血管病以及法布里病。近年来,中国学者总结了hCSVD患者的诊断和治疗过程。因此,我们组织从事hCVSD研究的专家,基于中国的报道并参考相关国外文献,针对不同类型hCVSD的临床和影像学特征以及诊断和治疗流程提出临床工作建议。本建议的目的是指导医生从卒中患者中识别出hCVSD患者,以便及时诊断并进行规范治疗。