Parrott Ashley, James Jeanne, Goldenberg Paula, Hinton Robert B, Miller Erin, Shikany Amy, Aylsworth Arthur S, Kaiser-Rogers Kathleen, Ferns Sunita J, Lalani Seema R, Ware Stephanie M
Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Heart Institute, Cincinnati, Ohio.
Am J Med Genet A. 2015 Feb;167A(2):363-70. doi: 10.1002/ajmg.a.36859. Epub 2014 Nov 26.
The 7q11.23 microduplication syndrome, caused by the reciprocal duplication of the Williams-Beuren syndrome deletion region, is a genomic disorder with an emerging clinical phenotype. Dysmorphic features, congenital anomalies, hypotonia, developmental delay highlighted by variable speech delay, and autistic features are characteristic findings. Congenital heart defects, most commonly patent ductus arteriosus, have been reported in a minority of cases. Included in the duplicated region is elastin (ELN), implicated as the cause of supravalvar aortic stenosis in patients with Williams-Beuren syndrome. Here we present a series of eight pediatric patients and one adult with 7q11.23 microduplication syndrome, all of whom had aortic dilation, the opposite vascular phenotype of the typical supravalvar aortic stenosis found in Williams-Beuren syndrome. The ascending aorta was most commonly involved, while dilation was less frequently identified at the aortic root and sinotubular junction. The findings in these patients support a recommendation for cardiovascular surveillance in patients with 7q11.23 microduplication syndrome.
7q11.23微重复综合征是由威廉姆斯-贝伦综合征缺失区域的相互重复引起的,是一种具有新出现临床表型的基因组疾病。畸形特征、先天性异常、肌张力减退、以言语发育迟缓为突出表现的发育迟缓以及自闭症特征是其典型表现。少数病例报告有先天性心脏缺陷,最常见的是动脉导管未闭。重复区域包括弹力蛋白(ELN),它被认为是威廉姆斯-贝伦综合征患者瓣膜上主动脉狭窄的病因。在此,我们报告了一系列8例儿科患者和1例成人7q11.23微重复综合征患者,他们均有主动脉扩张,这是威廉姆斯-贝伦综合征中典型瓣膜上主动脉狭窄相反的血管表型。升主动脉最常受累,而主动脉根部和窦管交界处的扩张较少见。这些患者的研究结果支持对7q11.23微重复综合征患者进行心血管监测的建议。