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心脏颜面皮肤综合征

The cardiofaciocutaneous syndrome.

作者信息

Roberts A, Allanson J, Jadico S K, Kavamura M I, Noonan J, Opitz J M, Young T, Neri G

机构信息

Harvard Medical School, Partners Healthcare System, Center for Genetics and Genomics, Boston, Massachusetts, USA.

出版信息

J Med Genet. 2006 Nov;43(11):833-42. doi: 10.1136/jmg.2006.042796. Epub 2006 Jul 6.

Abstract

The cardiofaciocutaneous (CFC) syndrome is a condition of sporadic occurrence, with patients showing multiple congenital anomalies and mental retardation. It is characterised by failure to thrive, relative macrocephaly, a distinctive face with prominent forehead, bitemporal constriction, absence of eyebrows, hypertelorism, downward-slanting palpebral fissures often with epicanthic folds, depressed nasal root and a bulbous tip of the nose. The cutaneous involvement consists of dry, hyperkeratotic, scaly skin, sparse and curly hair, and cavernous haemangiomata. Most patients have a congenital heart defect, most commonly pulmonic stenosis and hypertrophic cardiomyopathy. The developmental delay usually is moderate to severe. The syndrome is caused by gain-of-function mutations in four different genes BRAF, KRAS, mitogen-activated protein/extracellular signal-regulated kinase MEK1 and MEK2, all belonging to the same RAS-extracellular signal-regulated kinase (ERK) pathway that regulates cell differentiation, proliferation and apoptosis. The CFC syndrome is a member of a family of syndromes that includes the Noonan and Costello syndromes, presenting with phenotypic similarities. Noonan syndrome is caused by mutations in the protein tyrosine phosphatase SHP-2 gene (PTPN11), with a few people having a mutation in KRAS. Costello syndrome is caused by mutations in HRAS. The protein products of these genes also belong to the RAS-ERK pathway. Thus, the clinical overlap of these three conditions, which often poses a problem of differential diagnosis, is explained by their pathogenetic relatedness.

摘要

心脏颜面皮肤综合征(CFC综合征)是一种散发性疾病,患者表现出多种先天性异常和智力发育迟缓。其特征为生长发育迟缓、相对巨头畸形、面容独特,前额突出、颞部狭窄、无眉毛、眼距增宽、睑裂向下倾斜且常伴有内眦赘皮、鼻根部凹陷和鼻尖呈球状。皮肤受累表现为皮肤干燥、角化过度、鳞屑状、头发稀疏卷曲以及海绵状血管瘤。大多数患者患有先天性心脏缺陷,最常见的是肺动脉狭窄和肥厚型心肌病。发育迟缓通常为中度至重度。该综合征由BRAF、KRAS、丝裂原活化蛋白/细胞外信号调节激酶MEK1和MEK2这四个不同基因的功能获得性突变引起,这些基因均属于同一RAS-细胞外信号调节激酶(ERK)途径,该途径调节细胞分化、增殖和凋亡。CFC综合征是包括努南综合征和科斯特洛综合征在内的一组综合征中的一员,它们具有相似的表型。努南综合征由蛋白酪氨酸磷酸酶SHP-2基因(PTPN11)突变引起,少数人KRAS基因有突变。科斯特洛综合征由HRAS基因突变引起。这些基因的蛋白质产物也属于RAS-ERK途径。因此,这三种疾病的临床重叠现象(这常常造成鉴别诊断的难题)可由它们的发病机制相关性来解释。

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