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脑颅皮肤脂肪瘤病综述。

Review of encephalocraniocutaneous lipomatosis.

作者信息

Lopes Fabiana Castro Porto Silva, Schroeder Camryn, Patel Bhairav, Levy Moise L

机构信息

Dell Medical School, The University of Texas at Austin, Austin, TX, USA.

Dell Medical School, The University of Texas at Austin, Austin, TX, USA.

出版信息

Semin Pediatr Neurol. 2024 Dec;52:101166. doi: 10.1016/j.spen.2024.101166. Epub 2024 Nov 6.

Abstract

Encephalocraniocutaneous lipomatosis (ECCL), also known as Haberland syndrome, is a sporadic tumor predisposition neurocutaneous disorder, included in the oculoectodermal syndrome group of mosaic RASopathies. ECCL primarily affects the skin, central nervous system and eyes. Key diagnostic features include nevus psiloliparus, a hallmark subcutaneous lipomatous hamartoma associated with alopecia, along with subcutaneous lipomas, focal skin aplasia, and patchy alopecia. Neurologically, intracranial lipomas, particularly in the cerebellopontine angle, are prevalent, along with cortical dysplasia, ventriculomegaly, and vascular malformations. Ocular findings commonly involve choristomas, lipodermoids, and dermoids, which may impair vision. Diagnosis can be made clinically, but further confirmatory genetic testing can in some cases identify a pathogenic variant in the FGFR1 or KRAS genes. Molecular testing aids diagnosis but is not always conclusive. Management is multidisciplinary with focus on symptomatic management, typically involving dermatological, neurological, and ophthalmologic evaluations with consideration of brain and spine neuroimaging and surgical management of tumors. The prognosis varies, with most individuals leading generally normal lives, though there is a risk of developmental delay, seizures, and low-grade gliomas. The severity of CNS involvement does not consistently correlate with cutaneous or ocular abnormalities.

摘要

脑颅皮肤脂肪瘤病(ECCL),也称为哈伯兰德综合征,是一种散发性肿瘤易感性神经皮肤疾病,属于嵌合型RAS病相关的眼外胚层综合征组。ECCL主要影响皮肤、中枢神经系统和眼睛。关键诊断特征包括无毛脂瘤,这是一种与脱发相关的标志性皮下脂肪瘤性错构瘤,以及皮下脂肪瘤、局灶性皮肤发育不全和斑片状脱发。在神经方面,颅内脂肪瘤很常见,尤其是在小脑脑桥角,同时还伴有皮质发育异常、脑室扩大和血管畸形。眼部表现通常包括迷芽瘤、脂质皮样瘤和皮样瘤,可能会损害视力。诊断可通过临床做出,但在某些情况下,进一步的确诊基因检测可识别FGFR1或KRAS基因中的致病变异。分子检测有助于诊断,但并不总是具有决定性意义。治疗是多学科的,重点是对症治疗,通常包括皮肤科、神经科和眼科评估,并考虑脑和脊柱神经影像学检查以及肿瘤的手术治疗。预后各不相同,大多数人一般过着正常生活,不过存在发育迟缓、癫痫发作和低度胶质瘤的风险。中枢神经系统受累的严重程度与皮肤或眼部异常并不始终相关。

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