Olney R S, Hoyme H E, Roche F, Ferguson K, Hintz S, Madan A
Division of Medical Genetics, Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA.
Am J Med Genet. 2001 Nov 1;103(4):295-301.
Schinzel phocomelia syndrome is characterized by limb/pelvis hypoplasia/aplasia: specifically, intercalary limb deficiencies and absent or hypoplastic pelvic bones. The phenotype is similar to that described in a related multiple malformation syndrome known as Al-Awadi/Raas-Rothschild syndrome. The additional important feature of large parietooccipital skull defects without meningocele, encephalocele, or other brain malformation has thus far been reported only in children with Schinzel phocomelia syndrome. We recently evaluated a boy affected with Schinzel phocomelia born to nonconsanguineous healthy parents of Mexican origin. A third-trimester fetal ultrasound scan showed severe limb deficiencies and an absent pelvis. The infant died shortly after birth. Dysmorphology examination, radiographs, and autopsy revealed quadrilateral intercalary limb deficiencies with preaxial toe polydactyly; an absent pelvis and a 7 x 3-cm skull defect; and extraskeletal anomalies including microtia, telecanthus, micropenis with cryptorchidism, renal cysts, stenosis of the colon, and a cleft alveolar ridge. A normal 46,XY karyotype was demonstrated, and autosomal recessive inheritance was presumed on the basis of previously reported families. This case report emphasizes the importance of recognizing severe pelvic and skull deficiencies (either post- or prenatally) in differentiating infants with Schinzel phocomelia from other multiple malformation syndromes that feature intercalary limb defects, including thalidomide embryopathy and Roberts-SC phocomelia.
申泽尔短肢畸形综合征的特征为肢体/骨盆发育不全/发育不全:具体表现为肢体中段缺陷以及骨盆骨缺如或发育不全。其表型与一种相关的多重畸形综合征——阿拉瓦迪/拉斯-罗斯柴尔德综合征中所描述的相似。迄今为止,仅在申泽尔短肢畸形综合征患儿中报道了具有大的顶枕部颅骨缺损且无脑脊膜膨出、脑膨出或其他脑畸形这一额外重要特征。我们最近评估了一名患有申泽尔短肢畸形综合征的男孩,其父母为非近亲结婚的健康墨西哥裔。孕晚期胎儿超声扫描显示严重的肢体缺陷和骨盆缺如。婴儿出生后不久死亡。畸形学检查、X线片和尸检显示肢体中段四边形缺损伴轴前多指畸形;骨盆缺如和一个7×3厘米的颅骨缺损;以及骨骼外异常,包括小耳、眼距过宽、伴有隐睾的小阴茎、肾囊肿、结肠狭窄和牙槽嵴裂。核型分析显示为正常的46,XY,根据既往报道的家族情况推测为常染色体隐性遗传。本病例报告强调了在鉴别患有申泽尔短肢畸形综合征的婴儿与其他具有肢体中段缺陷的多重畸形综合征(包括沙利度胺胚胎病和罗伯茨-SC短肢畸形)时,识别严重骨盆和颅骨缺陷(无论是产后还是产前)的重要性。