Department of Human Genetics, Graduate School of Medicine, Yokohama City University, Yokohama, Japan.
Genetics Unit, Instituto da Crianca, Faculdade de Medicina, Universidade de Sao Paulo, SP, Brazil.
J Hum Genet. 2019 Sep;64(9):885-890. doi: 10.1038/s10038-019-0626-0. Epub 2019 Jul 4.
Pediatric hypertension can cause hypertensive emergencies, including hemorrhagic stroke, contributing to rare but serious childhood morbidity and mortality. Renovascular hypertension (RVH) is one of the major causes of secondary hypertension in children. Grange syndrome (MIM#602531) is a rare disease characterized by multiple stenosis or occlusion of the renal, abdominal, coronary, and cerebral arteries, which can cause phenotypes of RVH and fibromuscular dysplasia (MIM#135580). We report the case of a 7-year-old girl with Grange syndrome who showed RVH and multiple seizure episodes. At 1 year of age, she experienced seizures and sequential hemiparesis caused by a left thalamic hemorrhage without cerebral vascular anomalies. Chronic hypertension was observed, and abdominal computed tomography angiography showed characteristic bilateral renal artery stenosis. Whole-exome sequencing revealed a novel homozygous pathogenic variant in the YY1AP1 gene (NM_001198903.1: c.1169del: p.Lys390Argfs*12). Biallelic YY1AP1 mutations are known to cause Grange syndrome. Unlike previously reported patients, our patient presented with intracerebral hemorrhagic stroke without anomalous brain artery or bone fragility. The phenotype in our patient may help better understand this ultra-rare syndrome. Grange syndrome should be considered in patients presenting with childhood-onset hypertension and/or hemorrhagic stroke for early clinical intervention.
儿科高血压可引起高血压急症,包括出血性中风,导致罕见但严重的儿童发病率和死亡率。肾血管性高血压(RVH)是儿童继发性高血压的主要原因之一。Grange 综合征(MIM#602531)是一种罕见疾病,其特征为肾、腹、冠状动脉和脑动脉多处狭窄或闭塞,可引起 RVH 和纤维肌性发育不良(MIM#135580)的表型。我们报告了一例 7 岁女孩的 Grange 综合征病例,该患儿表现为 RVH 和多次癫痫发作。在 1 岁时,她因左丘脑出血而出现癫痫发作和随后的偏瘫,但没有脑血管异常。患儿一直存在慢性高血压,腹部 CT 血管造影显示双侧肾动脉狭窄的特征性表现。全外显子组测序显示 YY1AP1 基因(NM_001198903.1:c.1169del:p.Lys390Argfs*12)存在一个新的纯合致病性变异。双等位基因 YY1AP1 突变已知可导致 Grange 综合征。与先前报道的患者不同,我们的患者表现为颅内出血性中风,没有异常的脑动脉或骨骼脆弱。我们患者的表型有助于更好地理解这种超罕见综合征。对于儿童期起病的高血压和/或出血性中风患者,应考虑 Grange 综合征,以便进行早期临床干预。