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血管性血友病的罕见类型。

Rare forms of von Willebrand disease.

作者信息

Favaloro Emmanuel J

机构信息

Laboratory Haematology, Institute of Clinical Pathology and Medical Research (ICPMR), NSW Health Pathology, Westmead Hospital, Westmead, NSW, Australia.

Sydney Centres for Thrombosis and Haemostasis, Westmead, NSW, Australia.

出版信息

Ann Transl Med. 2018 Sep;6(17):345. doi: 10.21037/atm.2018.09.10.

Abstract

von Willebrand disease (VWD) arises from deficiency and/or defect(s) of plasma von Willebrand factor (VWF). In turn, plasma VWF is an adhesive protein which primarily functions by anchoring platelets to regions of vascular injury, thereby assisting prevention of bleeding. There is a proportional reduction also in Factor VIII, due to the absence of the stabilizing and anti-proteolytic effect that VWF normally exerts. VWD is reportedly the most common inherited bleeding disorder and can be classified into quantitative and qualitative defects, with type 1 and 3 VWD respectively identifying partial and total quantitative deficiency of VWF, and type 2 VWD identifying qualitative defects of VWF. The relative incidence of each subtype of VWD differs according to the locality and the ability of clinicians and laboratories to correctly diagnose and classify cases. In general, type 1 VWD is considered the most common type of VWD, whereas types 2 and 3 represent rarer forms. However, in developing countries, and partly because of consanguinity, type 3 VWD is over-represented. This review primarily focuses on the rarer forms of VWD, which typically comprise types 2 (A, B, M and N) and 3 VWD. The review also mentions type 1 VWD, largely for completeness and comparability, and since purportedly "severe" type 1 VWD, albeit not a formally recognized subtype of type 1 VWD, would represent a relatively "rare" form of VWD.

摘要

血管性血友病(VWD)源于血浆血管性血友病因子(VWF)的缺乏和/或缺陷。反过来,血浆VWF是一种黏附蛋白,其主要功能是将血小板锚定到血管损伤部位,从而有助于预防出血。由于VWF通常发挥的稳定和抗蛋白水解作用缺失,因子VIII也会相应减少。据报道,VWD是最常见的遗传性出血性疾病,可分为数量缺陷和质量缺陷,1型和3型VWD分别表示VWF的部分和完全数量缺乏,2型VWD表示VWF的质量缺陷。VWD各亚型的相对发病率因地区以及临床医生和实验室正确诊断和分类病例的能力而异。一般来说,1型VWD被认为是VWD最常见的类型,而2型和3型则代表较罕见的形式。然而,在发展中国家,部分由于近亲结婚,3型VWD的比例过高。本综述主要关注VWD较罕见的形式,通常包括2型(A、B、M和N)和3型VWD。该综述还提到了1型VWD,主要是为了完整性和可比性,并且由于所谓的“严重”1型VWD,尽管不是正式认可的1型VWD亚型,但将代表一种相对“罕见”的VWD形式。

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