Khan Jaffar, Al-Obaidy Khaleel I, Fan Rong
Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, USA.
Cureus. 2021 Aug 16;13(8):e17210. doi: 10.7759/cureus.17210. eCollection 2021 Aug.
Williams syndrome (WS), also known as Williams-Beuren syndrome, is a rare genetic disorder characterized by infantile hypercalcemia, short stature, a varying degree of mental retardation, elfin-like facial features, and cardiovascular abnormalities, including systemic hypertension, aortic hypoplasia, coarctation of the aorta, and valvular heart disease (aortic and pulmonic stenosis, mitral valve prolapsed or bicuspid aortic valve). It is also characterized by friendly and outgoing personality. The majority of WS cases are sporadic, while few are familial. Both sporadic and familial cases are due to deletion of chromosome 7 (7q11.23). Herein, we present an autopsy case of a 16-day-old male infant born to a 25-year-old mother with a history of William syndrome. Prenatal echocardiogram showed supravalvular aortic stenosis and pulmonary stenosis. The postnatal course was complicated by feeding difficulties and desaturation. Gross autopsy findings included generalized edema, macrocephaly with short neck, and multiple facial anomalies (mandibular hypoplasia, depressed nasal bridge, long philtrum, ear malformation, and wide mouth). The heart was hypertrophied with obstructed ventricles and rudimentary, hypoplastic aortic root. An enlarged, dilated, and tortuous left ureter was a unique finding to this case, in addition to variation in the renal arteries' size and an small bowel outpouching located 33 cm from the ileocecal valve. Cytogenetic analysis revealed deletion of chromosome 7 (7q11.23). In conclusion, majority of WS cases are sporadic, and few are familial and are inherited as autosomal dominant.
威廉姆斯综合征(WS),也称为威廉姆斯-贝伦综合征,是一种罕见的遗传性疾病,其特征为婴儿期高钙血症、身材矮小、不同程度的智力迟钝、小精灵样面容以及心血管异常,包括全身性高血压、主动脉发育不全、主动脉缩窄和瓣膜性心脏病(主动脉和肺动脉狭窄、二尖瓣脱垂或二叶式主动脉瓣)。其还具有友善外向的性格特点。大多数WS病例为散发性,少数为家族性。散发性和家族性病例均由7号染色体(7q11.23)缺失所致。在此,我们报告一例尸检病例,患儿为一名16天大的男婴,其母亲25岁,有威廉姆斯综合征病史。产前超声心动图显示主动脉瓣上狭窄和肺动脉狭窄。出生后的病程因喂养困难和血氧饱和度下降而复杂化。大体尸检结果包括全身水肿、头大短颈以及多种面部异常(下颌发育不全、鼻梁凹陷、人中长、耳部畸形和嘴巴宽大)。心脏肥厚,心室梗阻,主动脉根部发育不全且发育不良。除了肾动脉大小变异和距回盲瓣33厘米处的小肠憩室之外,左侧输尿管增粗、扩张且迂曲是该病例的一个独特发现。细胞遗传学分析显示7号染色体(7q11.23)缺失。总之,大多数WS病例为散发性,少数为家族性,呈常染色体显性遗传。