UNASSIGNED: This paper presents the rationale and design of the ZOOM@SVDs study, which aims to establish measures of cerebral small vessel dysfunction on 7T MRI as novel disease markers of SVDs.
UNASSIGNED: ZOOM@SVDs is a prospective observational cohort study with two years follow-up. ZOOM@SVDs recruits participants with Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL, N = 20), sporadic SVDs (N = 60), and healthy controls (N = 40). Participants undergo 7T brain MRI to assess different aspects of small vessel function including small vessel reactivity, cerebral perforating artery flow, and pulsatility. Extensive work-up at baseline and follow-up further includes clinical and neuropsychological assessment as well as 3T brain MRI to assess conventional SVD imaging markers. Measures of small vessel dysfunction are compared between patients and controls, and related to the severity of clinical and conventional MRI manifestations of SVDs.
UNASSIGNED: ZOOM@SVDs will deliver novel markers of cerebral small vessel function in patients with monogenic and sporadic forms of SVDs, and establish their relation with disease burden and progression. These small vessel markers can support etiological studies in SVDs and may serve as surrogate outcome measures in future clinical trials to show target engagement of drugs directed at the small vessels.
■本文介绍了ZOOM@SVDs研究的原理和设计,其目的是在7TMRI上建立脑小血管功能障碍的措施,作为SVDs的新疾病标志物。
■ZOOM@SVDs是一项前瞻性观察性队列研究,随访两年。ZOOM@SVDs招募患有皮质下梗死和白质脑病的常染色体显性动脉病的参与者(CADASIL,N=20),零星SVDs(N=60),和健康对照(N=40)。参与者接受7T脑MRI以评估小血管功能的不同方面,包括小血管反应性。脑穿通动脉血流,和脉动性。基线和随访时的广泛检查还包括临床和神经心理学评估以及3T脑MRI以评估常规SVD成像标记。在患者和对照组之间比较小血管功能障碍的测量值。并与SVDs的临床和常规MRI表现的严重程度有关。
■ZOOM@SVDs将为患有单基因和散发性SVDs的患者提供脑小血管功能的新标记,并建立它们与疾病负担和进展的关系。这些小血管标记物可以支持SVD的病因学研究,并且可以在未来的临床试验中用作替代结果指标,以显示针对小血管的药物的目标参与。