UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
Am J Med Genet A. 2021 Dec;185(12):3825-3830. doi: 10.1002/ajmg.a.62422. Epub 2021 Jul 13.
Linear Sebaceous Nevus Syndrome is a rare disorder that presents with nevus sebaceus in association with corneal dermoids, colobomas, choroidal osteomas, and arachnoid cysts. It is thought to represent a mosaic RASopathy. These are disorders characterized by postzygotic somatic mutation in genes involved in RAS/MAPK signaling pathway. In this report we describe two patients with linear sebaceous nevus syndrome found to have mutations in codon 146 of KRAS with evidence of mosaicism. This specific mutation has previously been reported in Oculoectodermal Syndrome and Encephalocraniocutaneous Lipomatosis, two other mosaic RASopathies with predominantly cerebrooculocutaneous manifestations. These findings suggest that, while initially classified as different syndromes, these disorders should be evaluated and managed as a spectrum of related disorders.
线性皮脂痣综合征是一种罕见的疾病,表现为皮脂痣与角膜皮样瘤、脉络膜骨瘤和蛛网膜囊肿相关。它被认为是一种镶嵌性 RAS 病。这些疾病的特征是参与 RAS/MAPK 信号通路的基因在后合子体细胞突变。在本报告中,我们描述了两名线性皮脂痣综合征患者,他们在 KRAS 密码子 146 中发现了突变,并有镶嵌现象的证据。这种特定的突变以前曾在眼外胚层发育不良和脑颅皮脂瘤病中报道过,这两种其他的镶嵌性 RAS 病主要表现为脑眼皮肤表现。这些发现表明,虽然这些疾病最初被分类为不同的综合征,但它们应该作为一系列相关疾病进行评估和管理。