Pepi Chiara, de Palma Luca, Trivisano Marina, Pietrafusa Nicola, Lepri Francesca Romana, Diociaiuti Andrea, Camassei Francesca Diomedi, Carfi-Pavia Giusy, De Benedictis Alessandro, Rossi-Espagnet Camilla, Vigevano Federico, Marras Carlo Efisio, Novelli Antonio, Bluemcke Ingmar, Specchio Nicola
Rare and Complex Epilepsy Unit, Department of Neuroscience, Bambino Gesù Children's Hospital IRCCS, Member of European Reference Network EpiCARE, 00165 Rome, Italy.
Translational Cytogenomics Research Unit, Laboratory of Medical Genetics, Bambino Gesù Children Hospital, IRCCS, 00165 Rome, Italy.
Brain Sci. 2021 Jun 16;11(6):793. doi: 10.3390/brainsci11060793.
The rare nevus sebaceous (NS) syndrome (NSS) includes cortical malformations and drug-resistant epilepsy. Somatic RAS-pathway genetic variants are pathogenetic in NS, but not yet described within the brain of patients with NSS. We report on a 5-year-old boy with mild psychomotor delay. A brown-yellow linear skin lesion suggestive of NS in the left temporo-occipital area was evident at birth. Epileptic spasms presented at aged six months. EEG showed continuous left temporo-occipital epileptiform abnormalities. Brain MRI revealed a similarly located diffuse cortical malformation with temporal pole volume reduction and a small hippocampus. We performed a left temporo-occipital resection with histopathological diagnosis of focal cortical dysplasia type Ia in the occipital region and hippocampal sclerosis type 1. Three years after surgery, he is seizure-and drug-free (Engel class Ia) and showed cognitive improvement. Genetic examination of brain and skin specimens revealed the c.35G > T (p.Gly12Val) KRAS somatic missense mutation. Literature review suggests epilepsy surgery in patients with NSS is highly efficacious, with 73% probability of seizure freedom. The few histological analyses reported evidenced disorganized cortex, occasionally with cytomegalic neurons. This is the first reported association of a KRAS genetic variant with cortical malformations associated with epilepsy, and suggests a possible genetic substrate for hippocampal sclerosis.
罕见的皮脂腺痣(NS)综合征(NSS)包括皮质发育畸形和耐药性癫痫。体细胞RAS通路基因变异在NS中具有致病性,但尚未在NSS患者的大脑中被描述。我们报告了一名5岁有轻度精神运动发育迟缓的男孩。出生时,左侧颞枕区可见一个提示NS的棕黄色线性皮肤病变。6个月大时出现癫痫痉挛。脑电图显示左侧颞枕区持续存在癫痫样异常。脑部磁共振成像显示在相同位置有弥漫性皮质发育畸形,伴有颞极体积减小和海马体较小。我们进行了左侧颞枕叶切除术,组织病理学诊断为枕叶Ia型局灶性皮质发育不良和1型海马硬化。术后三年,他无癫痫发作且无需服药(恩格尔Ia级),并显示出认知改善。对脑和皮肤标本的基因检测发现了c.35G>T(p.Gly12Val)KRAS体细胞错义突变。文献综述表明,NSS患者的癫痫手术疗效显著,无癫痫发作的概率为73%。少数报道的组织学分析证明皮质结构紊乱,偶尔可见巨细胞神经元。这是首次报道KRAS基因变异与癫痫相关的皮质发育畸形有关,并提示海马硬化可能存在遗传基础。