Pinto Anna, Sahin Mustafa, Pearl Phillip L
Department of Neurology, Boston Children's Hospital, Boston, Massachusetts, USA; Department of Neurology, Dartmouth Hitchcock, Manchester, New Hampshire, USA.
Department of Neurology, Boston Children's Hospital, Boston, Massachusetts, USA.
F1000Res. 2016 Mar 18;5. doi: 10.12688/f1000research.7605.1. eCollection 2016.
Epilepsy is a major morbidity in Sturge Weber syndrome, a segmental vascular neurocutaneous disorder classically associated with facial angiomas, glaucoma, and leptomeningeal capillary-venous type vascular malformations. The extent of the latter correlates with neurological outcome. Post-zygotic mosaicism for the activating mutation p.R183Q of the GNAQ gene has been identified as the major cause. GNAQ encodes for an alpha subunit of a heterotrimeric G protein critical to blood vessel development. The earlier the timing of the mutation in development, the more severe the involvement, e.g. from isolated port-wine stains to the full syndrome. The strongest predictors of adverse outcomes are MRI and the presence of angiomas involving any part of the forehead, delineated inferiorly from the outer canthus of the eye to the top of the ear, and including the upper eyelid. The neurological course may be progressive and the typical constellation of symptoms is focal onset seizures, hemiparesis, headache, stroke-like episodes, behavior problems, intellectual disability, and visual field deficits. Antiseizure medications are effective in about half of patients. The presence of localized seizures, focal neurological deficits, and drug resistant epilepsy indicate epilepsy surgical evaluation. Earlier seizure onset, i.e. before six months of age, is associated with a more severe course with significant residual deficits. Factors contributing to epileptogenesis include decreased brain tissue perfusion due to abnormal venous drainage, anoxic injury contributing to cerebral calcification, breakdown of the blood-brain barrier, and the presence of developmental cortical malformations. Pre-symptomatic prophylactic treatment may be a future option to modify the course of the disease including the associated epileptogenesis.
癫痫是斯特奇-韦伯综合征的主要发病症状,该综合征是一种节段性血管神经皮肤疾病,典型表现为面部血管瘤、青光眼和软脑膜毛细血管-静脉型血管畸形。后者的范围与神经学预后相关。已确定GNAQ基因激活突变p.R183Q的合子后镶嵌现象是主要病因。GNAQ编码一种对血管发育至关重要的异源三聚体G蛋白的α亚基。发育过程中突变发生的时间越早,受累越严重,例如从孤立的葡萄酒色斑到完整的综合征。不良预后的最强预测因素是磁共振成像(MRI)以及血管瘤累及从眼外眦到耳顶并包括上眼睑的前额任何部位。神经病程可能是进行性的,典型的症状组合是局灶性发作、偏瘫、头痛、中风样发作、行为问题、智力残疾和视野缺损。抗癫痫药物对大约一半的患者有效。存在局限性发作、局灶性神经功能缺损和药物难治性癫痫表明需要进行癫痫手术评估。较早的癫痫发作,即在6个月龄之前,与更严重的病程及显著的残留缺损相关。导致癫痫发生的因素包括由于异常静脉引流导致的脑组织灌注减少、导致脑钙化的缺氧损伤、血脑屏障的破坏以及发育性皮质畸形的存在。症状前预防性治疗可能是未来改变疾病进程包括相关癫痫发生的一种选择。