Department of Clinical Neurophysiology, Danish Epilepsy Centre, Dianalund, Denmark.
Epilepsy and Movement Disorders, Neurology Unit, Bambino Gesù Children's Hospital, Rome, Italy.
Epileptic Disord. 2024 Aug;26(4):520-526. doi: 10.1002/epd2.20242. Epub 2024 May 23.
Pathogenic variants in CACNA1E are associated with early-onset epileptic and developmental encephalopathy (DEE). Severe to profound global developmental delay, early-onset refractory seizures, severe hypotonia, and macrocephaly are the main clinical features. Patients harboring the recurrent CACNA1E variant p.(Gly352Arg) typically present with the combination of early-onset DEE, dystonia/dyskinesia, and contractures. We describe a 2-year-and-11-month-old girl carrying the p.(Gly352Arg) CACNA1E variant. She has a severe DEE with very frequent drug-resistant seizures, profound hypotonia, and episodes of dystonia and dyskinesia. Long-term video-EEG-monitoring documented subsequent tonic asymmetric seizures during wakefulness and mild paroxysmal dyskinesias of the trunk out of sleep which were thought to be a movement disorder and instead turned out to be focal hyperkinetic seizures. This is the first documented description of the EEG findings in this disorder. Our report highlights a possible overlap between cortical and subcortical phenomena in CACNA1E-DEE. We also underline how a careful electro-clinical evaluation might be necessary for a correct discernment between the two disorders, playing a fundamental role in the clinical assessment and proper management of children with CACNA1E-DEE.
CACNA1E 中的致病性变异与早发性癫痫和发育性脑病(DEE)有关。严重至重度全面发育迟缓、早发性难治性癫痫发作、严重的肌张力低下和大头畸形是主要的临床特征。携带 CACNA1E 变异 p.(Gly352Arg) 的患者通常表现为早发性 DEE、肌张力障碍/运动障碍和挛缩的组合。我们描述了一位 2 岁 11 个月大的女孩携带 p.(Gly352Arg) CACNA1E 变异。她患有严重的 DEE,癫痫发作非常频繁且对药物有抗性,存在严重的肌张力低下和肌张力障碍和运动障碍发作。长期视频脑电图监测记录到清醒时出现后续的强直不对称性发作和睡眠中躯干轻度阵发性运动障碍,最初被认为是一种运动障碍,但后来被证实是局灶性多动性发作。这是该疾病脑电图表现的首次记录描述。我们的报告强调了 CACNA1E-DEE 中皮质和皮质下现象之间可能存在重叠。我们还强调了如何进行仔细的电临床评估对于正确区分这两种疾病的必要性,这在对 CACNA1E-DEE 患儿进行临床评估和适当管理方面发挥着重要作用。