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色素性外胚叶发育不良伴大理石皮肤和斑状皮肤色素沉着症的皮肤外表现:病例系列及文献复习。

Extracutaneous manifestations in phacomatosis cesioflammea and cesiomarmorata: Case series and literature review.

机构信息

Department of Pediatrics, Stanford School of Medicine, Stanford, California.

Stanford Center for Undiagnosed Diseases, Stanford University, Stanford, California.

出版信息

Am J Med Genet A. 2019 Jun;179(6):966-977. doi: 10.1002/ajmg.a.61134. Epub 2019 Mar 28.

Abstract

Phacomatosis pigmentovascularis (PPV) comprises a family of rare conditions that feature vascular abnormalities and melanocytic lesions that can be solely cutaneous or multisystem in nature. Recently published work has demonstrated that both vascular and melanocytic abnormalities in PPV of the cesioflammea and cesiomarmorata subtypes can result from identical somatic mosaic activating mutations in the genes GNAQ and GNA11. Here, we present three new cases of PPV with features of the cesioflammea and/or cesiomarmorata subtypes and mosaic mutations in GNAQ or GNA11. To better understand the risk of potentially occult complications faced by such patients we additionally reviewed 176 cases published in the literature. We report the frequency of clinical findings, their patterns of co-occurrence as well as published recommendations for surveillance after diagnosis. Features assessed include: capillary malformation; dermal and ocular melanocytosis; glaucoma; limb asymmetry; venous malformations; and central nervous system (CNS) anomalies, such as ventriculomegaly and calcifications. We found that ocular findings are common in patients with phacomatosis cesioflammea and cesiomarmorata. Facial vascular involvement correlates with a higher risk of seizures (p = .0066). Our genetic results confirm the role of mosaic somatic mutations in GNAQ and GNA11 in phacomatosis cesioflammea and cesiomarmorata. Their clinical and molecular findings place these conditions on a clinical spectrum encompassing other GNAQ and GNA11 related disorders and inform recommendations for their management.

摘要

色素血管性斑状错构瘤病(PPV)包括一组罕见疾病,其特征为血管异常和黑色素细胞病变,这些病变可能仅为皮肤性,也可能为多系统性质。最近发表的研究工作表明,cesioflammea 和 cesiomarmorata 亚型的 PPV 中的血管和黑色素细胞异常都可能是由 GNAQ 和 GNA11 基因中的相同体细胞镶嵌激活突变引起的。在此,我们介绍了三个具有 cesioflammea 和/或 cesiomarmorata 亚型特征和 GNAQ 或 GNA11 镶嵌突变的新的 PPV 病例。为了更好地了解此类患者可能面临的潜在隐匿性并发症的风险,我们还回顾了文献中报道的 176 例病例。我们报告了临床发现的频率、它们的共同发生模式以及发表的诊断后监测建议。评估的特征包括:毛细血管畸形;皮肤和眼部黑色素细胞增多;青光眼;肢体不对称;静脉畸形;以及中枢神经系统(CNS)异常,如脑室扩大和钙化。我们发现,色素血管性斑状错构瘤病患者的眼部表现常见。面部血管受累与癫痫发作的风险增加相关(p=0.0066)。我们的遗传结果证实了镶嵌性体细胞突变在 cesioflammea 和 cesiomarmorata 色素血管性斑状错构瘤病中的作用。它们的临床和分子发现将这些疾病置于一个临床谱中,包括其他与 GNAQ 和 GNA11 相关的疾病,并为其管理提供了建议。

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