MRC Prion Unit at University College London (UCL), Institute of Prion Diseases, UCL, London, W1W 7FF, UK.
Dementia Research Centre, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Brain. 2023 Oct 3;146(10):3991-4014. doi: 10.1093/brain/awad193.
Cerebral amyloid angiopathy (CAA) is an important cerebral small vessel disease associated with brain haemorrhage and cognitive change. The commonest form, sporadic amyloid-β CAA, usually affects people in mid- to later life. However, early-onset forms, though uncommon, are increasingly recognized and may result from genetic or iatrogenic causes that warrant specific and focused investigation and management. In this review, we firstly describe the causes of early-onset CAA, including monogenic causes of amyloid-β CAA (APP missense mutations and copy number variants; mutations of PSEN1 and PSEN2) and non-amyloid-β CAA (associated with ITM2B, CST3, GSN, PRNP and TTR mutations), and other unusual sporadic and acquired causes including the newly-recognized iatrogenic subtype. We then provide a structured approach for investigating early-onset CAA, and highlight important management considerations. Improving awareness of these unusual forms of CAA amongst healthcare professionals is essential for facilitating their prompt diagnosis, and an understanding of their underlying pathophysiology may have implications for more common, late-onset, forms of the disease.
脑淀粉样血管病(Cerebral amyloid angiopathy,CAA)是一种与脑出血和认知改变相关的重要脑小血管疾病。最常见的形式是散发性淀粉样蛋白-β CAA,通常影响中老年人群。然而,尽管少见,但早发性形式越来越被认识到,可能由遗传或医源性原因引起,需要进行特定和有针对性的调查和管理。在这篇综述中,我们首先描述了早发性 CAA 的原因,包括淀粉样蛋白-β CAA 的单基因原因(APP 错义突变和拷贝数变异;PSEN1 和 PSEN2 突变)和非淀粉样蛋白-β CAA(与 ITM2B、CST3、GSN、PRNP 和 TTR 突变相关),以及其他不常见的散发性和获得性原因,包括新认识的医源性亚型。然后,我们提供了一种用于调查早发性 CAA 的结构化方法,并强调了重要的管理注意事项。提高医疗保健专业人员对这些不常见 CAA 形式的认识对于促进其快速诊断至关重要,并且对更常见的晚发性疾病形式的潜在病理生理学的理解可能具有重要意义。