Pivnick E K, Angle B, Kaufman R A, Hall B D, Pitukcheewanont P, Hersh J H, Fowlkes J L, Sanders L P, O'Brien J M, Carroll G S, Gunther W M, Morrow H G, Burghen G A, Ward J C
Department of Pediatrics, The University of Tennessee-Memphis, Tennessee, USA.
Am J Med Genet. 2000 Jan 17;90(2):131-40.
The neonatal progeroid syndrome (NPS), or Wiedemann-Rautenstrauch, is a rare autosomal recessive disorder comprised of generalized lipoatrophy except for fat pads in the suprabuttock areas, hypotrichosis of the scalp hair, eyebrows, and eyelashes, relative macrocephaly, triangular face, natal teeth, and micrognathia. We report on 5 new patients who demonstrate phenotypic variability and who represent the single largest series of NPS reported to date. Two of the patients are from an African-American kindred, an ethnic occurrence not reported previously. The fact that there are 2 pairs of sibs among the 5 patients further supports that NPS is an autosomal recessive condition. This report also includes a review of the previously reported 16 patients and compares them with the 5 new patients. Abnormalities in endocrine and lipid metabolism were found in 3 of 5 patients. Skeletal findings in 2 of our patients demonstrated some new findings as well as the typical radiological abnormalities previously noted in NPS. It is apparent, based on the 21 cases, that mild to moderate mental retardation is common in NPS. Long term follow-up of patients with NPS should provide more information relative to their ultimate psychomotor development. NPS is usually lethal by 7 months; however, on rare occasions, patients have survived into the teens. Our 3 surviving patients range in age from 16-23 months. Variability in the phenotype of NPS is clear; however, the phenotype remains distinct enough to allow a secure diagnosis.
新生儿早老综合征(NPS),即维德曼-劳滕施特劳赫综合征,是一种罕见的常染色体隐性疾病,其特征包括全身脂肪萎缩(除了臀上区域的脂肪垫)、头皮毛发、眉毛和睫毛稀少、相对巨头畸形、三角形脸、 natal牙和小颌畸形。我们报告了5例新患者,他们表现出表型变异性,代表了迄今为止报道的最大的单一NPS病例系列。其中2例患者来自一个非裔美国人家族,这一种族发病情况此前未被报道。5例患者中有2对同胞,这进一步支持了NPS是常染色体隐性疾病这一观点。本报告还对先前报道的16例患者进行了回顾,并将他们与5例新患者进行了比较。5例患者中有3例存在内分泌和脂质代谢异常。我们的2例患者的骨骼检查结果显示了一些新发现以及NPS先前已被注意到的典型放射学异常。基于这21例病例,很明显,轻度至中度智力发育迟缓在NPS中很常见。对NPS患者的长期随访应能提供更多有关其最终心理运动发育的信息。NPS通常在7个月时致命;然而,在极少数情况下,患者能存活到十几岁。我们的3例存活患者年龄在16至23个月之间。NPS表型的变异性很明显;然而,其表型仍然足够独特,能够做出可靠的诊断。