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巨脑回畸形并结节性硬化症:一种罕见但极具挑战性的关联。

Hemimegalencephaly and tuberous sclerosis complex: A rare yet challenging association.

机构信息

1st Paediatric Department, Developmental Centre "A. Fokas", Aristotle University of Thessaloniki, "Hippokration" General Hospital, Thessaloniki, Greece.

1st Paediatric Department, Developmental Centre "A. Fokas", Aristotle University of Thessaloniki, "Hippokration" General Hospital, Thessaloniki, Greece.

出版信息

Eur J Paediatr Neurol. 2021 Jan;30:58-65. doi: 10.1016/j.ejpn.2020.12.007. Epub 2020 Dec 23.

Abstract

Hemimegalencephaly is a rare malformation of cortical development characterised by enlargement of one cerebral hemisphere. The association between hemimegalencephaly and tuberous sclerosis complex, an autosomal dominant genetic disorder, is uncommon and has so far been reported only in a few cases. Intractable epilepsy and severe developmental delay are typical clinical manifestations. Aberrant activation of the mTOR signalling pathway is considered to be the hallmark of the pathogenesis of these two disorders. Thus, mTOR inhibitors such as everolimus represent a promising therapeutic approach to mTOR-associated manifestations. We present a thorough literature review of the association between hemimegaloencephaly and tuberous sclerosis complex.

摘要

巨脑回畸形是一种罕见的皮质发育畸形,其特征为一侧大脑半球增大。巨脑回畸形与结节性硬化症(一种常染色体显性遗传疾病)之间的关联并不常见,迄今为止仅在少数病例中报道过。难治性癫痫和严重的发育迟缓是其典型的临床表现。mTOR 信号通路的异常激活被认为是这两种疾病发病机制的标志。因此,mTOR 抑制剂,如依维莫司,代表了一种有前途的治疗 mTOR 相关表现的方法。我们对巨脑回畸形与结节性硬化症之间的关联进行了全面的文献回顾。

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