Gripp K W, Stolle C A, McDonald-McGinn D M, Markowitz R I, Bartlett S P, Katowitz J A, Muenke M, Zackai E H
Division of Human Genetics and Molecular Biology, The Children's Hospital of Philadelphia, Pennsylvania 19104, USA.
Am J Med Genet. 1998 Jul 24;78(4):356-60. doi: 10.1002/(sici)1096-8628(19980724)78:4<356::aid-ajmg10>3.0.co;2-h.
We present a patient with pansynostosis, hydrocephalus, seizures, extreme proptosis with luxation of the eyes out of the lids, apnea and airway obstruction, intestinal non-rotation, and severe developmental delay. His skeletal abnormalities include bilateral elbow ankylosis, radial head dislocation, and unilateral broad and deviated first toe. The phenotype of this patient is consistent with that previously reported in Pfeiffer syndrome type III, but is unusual for the lack of broad thumbs. Our patient most closely resembles the case described by Kerr et al. [1996: Am J Med Genet 66:138-143] as Pfeiffer syndrome type III with normal thumbs. Mutations in the genes for fibroblast growth factor receptors (FGFR) 1 and 2 have previously been seen in patients with Pfeiffer syndrome type I. The mutation identified in our patient, Ser351Cys in FGFR2, represents the first reported cause of Pfeiffer syndrome type III. An identical mutation was described once previously by Pulleyn et al., in a patient whose brief clinical description included cloverleaf skull, significant developmental delay, and normal hands and feet [Eur. J. Hum. Genet. 4: 283-291, 1996]. In our patient, previously performed single-strand conformation polymorphism analysis failed to detect a band shift; the mutation was identified only after independent sequence analysis.
我们报告了一名患有全颅缝早闭、脑积水、癫痫、眼球极度突出伴眼睑脱出、呼吸暂停和气道梗阻、肠道旋转不良以及严重发育迟缓的患者。他的骨骼异常包括双侧肘关节强直、桡骨头脱位以及单侧大脚趾增宽并向外侧偏斜。该患者的表型与先前报道的III型 Pfeiffer 综合征一致,但因缺乏宽大的拇指而较为罕见。我们的患者与 Kerr 等人[1996年:《美国医学遗传学杂志》66:138 - 143]所描述的具有正常拇指的III型 Pfeiffer 综合征病例最为相似。成纤维细胞生长因子受体(FGFR)1和2基因的突变先前在I型 Pfeiffer 综合征患者中被发现。在我们患者中鉴定出的突变,即FGFR2基因中的Ser351Cys,是首次报道的III型 Pfeiffer 综合征的病因。Pulleyn 等人曾在一名患者中描述过相同的突变,该患者的简要临床描述包括三叶形颅骨、显著发育迟缓以及手足正常[《欧洲人类遗传学杂志》4: 283 - 291, 1996]。在我们的患者中,先前进行的单链构象多态性分析未能检测到条带移动;该突变仅在独立序列分析后才被鉴定出来。