Marais A D, Kotze M J, Raal F J, Khine A A, Talmud P J, Humphries S E
Chemical Pathology, Health Sciences Faculty, University of Cape Town, Observatory, South Africa. Email:
Division of Chemical Pathology, Department of Pathology, Faculty of Medicine and Health Sciences, Stellenbosch University; National Health Laboratory Service, Tygerberg Hospital, Tygerberg, South Africa.
Cardiovasc J Afr. 2019 Sep/Oct;30(5):297-304. doi: 10.5830/CVJA-2019-055.
Familial hypercholesterolaemia (FH) is a common autosomal dominantly inherited disorder in which impaired clearance of plasma low-density lipoprotein cholesterol causes premature atherosclerotic vascular disease and tendon xanthomata. This workshop aimed to consolidate information on the diagnosis and management of FH in South Africa. The genetic causes include mutations in the LDL receptor, apolipoprotein B100 and proprotein convertase subtilisin/kexin type 9 (PCSK9). Additionally, the concatenation of multiple gene variants can result in polygenic FH. Therapeutic measures include a healthy lifestyle, statins and cholesterol-absorption inhibitors that will achieve control of the dyslipidaemia in the majority of cases. The recently introduced monoclonal antibodies to PCSK9 can improve achievement of target concentration in severe cases. FH is present in all sectors of the South African population but there is sparse documentation in the indigenous African populations. FH should be actively sought, diagnosed and treated with judicious pharmacotherapy and screening of relatives.
家族性高胆固醇血症(FH)是一种常见的常染色体显性遗传疾病,其中血浆低密度脂蛋白胆固醇清除受损会导致过早的动脉粥样硬化性血管疾病和肌腱黄色瘤。本次研讨会旨在巩固南非FH诊断和管理的相关信息。其遗传原因包括低密度脂蛋白受体、载脂蛋白B100和前蛋白转化酶枯草杆菌蛋白酶/kexin 9型(PCSK9)的突变。此外,多个基因变异的串联可导致多基因FH。治疗措施包括健康的生活方式、他汀类药物和胆固醇吸收抑制剂,在大多数情况下可实现血脂异常的控制。最近引入的针对PCSK9的单克隆抗体可改善重症病例中目标浓度的达成情况。FH在南非各人群中均有存在,但在非洲本土人群中的记录较少。应积极筛查、诊断FH,并通过合理的药物治疗和亲属筛查进行治疗。