Rebecca Silverstein, Michael Kuwabara, Brian Appavu
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引用次数: 1
摘要
锚蛋白重复结构域17 (ANKRD17)被认为在血管完整性中发挥作用,并已报道与发育迟缓,癫痫和生长限制有关。尽管ANKRD17缺陷小鼠已被证明会经历灾难性的出血,但在ANKRD17致病性变异的人类患者中尚未报道血管畸形。我们报道了一例患有ANKRD17杂合新生变异的足月男性新生儿(ANKRD17;[6988] C > G, P.[P2330a])因左侧大脑中动脉动脉瘤破裂而发生蛛网膜下腔出血。他经历了急性症状性癫痫发作,在35天的时候需要切除他的动脉瘤,后来发展为多灶性耐药癫痫。据我们所知,该病例是ANKRD17患者脑血管畸形的首例报告。需要进一步研究ANKRD17的致病变异是否会导致儿童脑动脉瘤或其他脑血管畸形。
Neonatal Aneurysm Rupture in a Child with a De Novo Variant to ANKRD17.
Ankyrin repeat domain 17 (ANKRD17) is postulated to play a role in the integrity of blood vessels and has been reported to be associated with developmental delays, epilepsy, and growth restriction. Whereas ANKRD17-deficient mice have been demonstrated to experience catastrophic hemorrhages, vascular malformations have not been reported in human patients with pathogenic variants to ANKRD17. We report a term male neonate with a heterozygous de novo variant to ANKRD17 (ANKRD17; c6988 C > G, P.[P2330a]) who experienced subarachnoid hemorrhage from a ruptured aneurysm involving the left middle cerebral artery. He experienced acute symptomatic seizures and required clipping of his aneurysm at 35 days of life, later progressing to developing multifocal drug-resistant epilepsy. To our knowledge, this case represents the first report of a cerebrovascular malformation from a patient with ANKRD17. Further work is needed to investigate whether pathogenic variants to ANKRD17 can lead to cerebral aneurysms or other cerebrovascular malformations in children.