Forrester Shawnia, Kovach Margaret J, Smith Randell E, Rimer Lisa, Wesson Melissa, Kimonis Virginia E
Department of Pediatrics, Southern Illinois University School of Medicine, Springfield, Illinois 62794-9658, USA.
Am J Med Genet. 2002 Nov 1;112(4):338-42. doi: 10.1002/ajmg.10625.
Kousseff syndrome was originally described by Boris Kousseff in 1984: Pediatrics 74:395-398 in three siblings whose main features were conotruncal heart defects, neural tube defects, and dysmorphic features. The proband is a white male who has spina bifida, shunted hydrocephalus, cleft palate, short stature, cognitive impairment, and the typical craniofacial features of velo-cardio-facial syndrome (VCFS), including low-set and dysplastic ears, broad base of the nose, narrow alae nasi, and retrognathia. The family history is significant for a brother who died at 2 weeks of age with myelomeningocele, hydrocephalus, transposition of the great vessels, and unilateral renal agenesis, and a sister who died at 11 days of age with myelomeningocele, truncus arteriosus, hypocalcemia, and autopsy findings of absent thymus and parathyroid glands, consistent with DiGeorge anomaly. Given the clinical findings, family history, and recent knowledge that open neural tube defects can occur in VCFS/DiGeorge anomaly, FISH analysis for 22q11-13 deletion was performed on the proband. A deletion was detected in him and subsequently confirmed in his father. Molecular analysis on autopsy material confirmed the deletion in the proband's deceased brother. We suggest that individuals with neural tube defects associated with other anomalies such as congenital heart defects or cleft palate be evaluated for 22q deletions.
库塞夫综合征最初由鲍里斯·库塞夫于1984年描述:发表于《儿科学》第74卷,第395 - 398页,涉及三个兄弟姐妹,其主要特征为圆锥动脉干心脏缺陷、神经管缺陷和畸形特征。先证者是一名白人男性,患有脊柱裂、分流性脑积水、腭裂、身材矮小、认知障碍以及腭心面综合征(VCFS)典型的颅面特征,包括低位和发育不良的耳朵、宽鼻根、窄鼻翼以及下颌后缩。家族史显示,一名兄弟在2周龄时死于脊髓脊膜膨出、脑积水、大动脉转位和单侧肾发育不全;一名姐妹在11日龄时死于脊髓脊膜膨出、动脉干、低钙血症,尸检发现胸腺和甲状旁腺缺如,符合迪乔治异常。鉴于临床发现及家族史,以及最近关于开放性神经管缺陷可发生于VCFS/迪乔治异常的认识,对先证者进行了22q11 - 13缺失的荧光原位杂交(FISH)分析。在他身上检测到缺失,随后在其父亲身上得到证实。对尸检材料的分子分析证实了先证者已故兄弟的缺失。我们建议,对于患有与其他异常(如先天性心脏缺陷或腭裂)相关的神经管缺陷的个体,应评估其22q缺失情况。