Byers Peter H, Belmont John, Black James, De Backer Julie, Frank Michael, Jeunemaitre Xavier, Johnson Diana, Pepin Melanie, Robert Leema, Sanders Lynn, Wheeldon Nigel
Am J Med Genet C Semin Med Genet. 2017 Mar;175(1):40-47. doi: 10.1002/ajmg.c.31553.
Vascular Ehlers Danlos syndrome (vEDS) is an uncommon genetic disorders characterized by arterial aneurysm, dissection and rupture, bowel rupture, and rupture of the gravid uterus. The frequency is estimated as 1/50,000-1/200,000 and results from pathogenic variants in COL3A1, which encodes the chains of type III procollagen, a major protein in vessel walls and hollow organs. Initial diagnosis depends on the recognitions of clinical features, including family history. Management is complex and requires multiple specialists who can respond to and manage the major complications. A summary of recommendations for management include: Identify causative variants in COL3A1 prior to application of diagnosis, modulate life style to minimize injury, risk of vessel/organ rupture, identify and create care team, provide individual plans for emergency care ("vascular EDS passport") with diagnosis and management plan for use when traveling, centralize management at centers of excellence (experience) when feasible, maintain blood pressure in the normal range and treat hypertension aggressively, surveillance of vascular tree by doppler ultrasound, CTA (low radiation alternatives) or MRA if feasible on an annual basis. These recommendations represent a consensus of an international group of specialists with a broad aggregate experience in the care of individuals with vascular EDS that will need to be assessed on a regular basis as new information develops. © 2017 Wiley Periodicals, Inc.
血管型埃勒斯-当洛综合征(vEDS)是一种罕见的遗传性疾病,其特征为动脉动脉瘤、夹层和破裂、肠道破裂以及妊娠子宫破裂。估计发病率为1/50,000 - 1/200,000,由COL3A1基因的致病性变异引起,该基因编码III型前胶原链,这是血管壁和中空器官中的一种主要蛋白质。初始诊断取决于对临床特征的识别,包括家族史。管理复杂,需要多个专科医生来应对和处理主要并发症。管理建议总结如下:在进行诊断之前确定COL3A1中的致病变异,调整生活方式以尽量减少损伤、血管/器官破裂的风险,确定并组建护理团队,提供针对紧急护理的个人计划(“血管EDS护照”),包括诊断和管理计划以便在旅行时使用,在可行的情况下由卓越中心(有经验的)进行集中管理,将血压维持在正常范围并积极治疗高血压,每年可行时通过多普勒超声、CTA(低辐射替代方法)或MRA对血管系统进行监测。这些建议代表了一组在血管EDS患者护理方面具有广泛综合经验的国际专家的共识,随着新信息的出现,需要定期对其进行评估。© 2017威利期刊公司