Ancora Caterina, Ortigoza-Escobar Juan Dario, Valletti Margherita Aluffi, Furia Francesca, Nielsen Jens Erik Klint, Møller Rikke S, Gardella Elena
Department of Epilepsy Genetics and Personalized Treatment, Danish Epilepsy Centre, Dianalund, Denmark.
Pediatric Neurology and Neurophysiology Unit, Department of Women's and Children's Health, Padova University Hospital, Padova, Italy.
Epileptic Disord. 2024 Apr;26(2):219-224. doi: 10.1002/epd2.20203. Epub 2024 Mar 4.
Pathogenic variants in SCN8A are associated with a broad phenotypic spectrum, including Self-Limiting Familial Infantile Epilepsy (SeLFIE), characterized by infancy-onset age-related seizures with normal development and cognition. Movement disorders, particularly paroxysmal kinesigenic dyskinesia typically arising after puberty, may represent another core symptom. We present the case of a 1-year-old girl with a familial disposition to self-limiting focal seizures from the maternal side and early-onset orofacial movement disorders associated with SCN8A-SeLFIE. Brain MRI was normal. Genetic testing revealed a maternally inherited SCN8A variant [c.4447G > A; p.(Glu1483Lys)]. After the introduction of valproic acid, she promptly achieved seizure control as well as complete remission of strabismus and a significant decrease in episodes of tongue deviation. Family history, genetic findings, and epilepsy phenotype are consistent with SCN8A-SeLFIE. Movement disorders are an important part of the SCN8A phenotypic spectrum, and this case highlights the novel early-onset orofacial movement disorders associated with this condition. The episodes of tongue deviation and protrusion suggest focal oromandibular (lingual) dystonia. Additionally, while infantile strabismus or esophoria is a common finding in healthy individuals, our case raises the possibility of an ictal origin of the strabismus. This study underscores the importance of recognizing and addressing movement disorders in SCN8A-SeLFIE patients, particularly the rare early-onset orofacial manifestations. It adds to the growing body of knowledge regarding the diverse clinical presentations of SCN8A-associated disorders and suggests potential avenues for clinical management and further research.
SCN8A基因的致病性变异与广泛的表型谱相关,包括自限性家族性婴儿癫痫(SeLFIE),其特征为婴儿期起病的与发育和认知正常相关的年龄相关性癫痫发作。运动障碍,尤其是通常在青春期后出现的发作性运动诱发性运动障碍,可能是另一个核心症状。我们报告了一例1岁女孩的病例,她有来自母亲一方的自限性局灶性癫痫发作的家族倾向,以及与SCN8A - SeLFIE相关的早发性口面部运动障碍。脑部MRI正常。基因检测发现了一个母系遗传的SCN8A变异[c.4447G>A;p.(Glu1483Lys)]。在使用丙戊酸后,她迅速实现了癫痫控制,斜视完全缓解,舌偏斜发作次数显著减少。家族史、基因检测结果和癫痫表型与SCN8A - SeLFIE一致。运动障碍是SCN8A表型谱的重要组成部分,该病例突出了与这种疾病相关的新型早发性口面部运动障碍。舌偏斜和突出发作提示局灶性口下颌(舌)肌张力障碍。此外,虽然婴儿斜视或内斜视在健康个体中很常见,但我们的病例增加了斜视发作起源的可能性。这项研究强调了识别和处理SCN8A - SeLFIE患者运动障碍的重要性,特别是罕见的早发性口面部表现。它增加了关于SCN8A相关疾病多样临床表现的知识,并为临床管理和进一步研究提出了潜在途径。