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Abstract：
Background: Epileptic encephalopathies (EE) are characterized by severe drug-resistant seizures, early onset, and unfavorable developmental outcomes. This article discusses the use of intravenous methylprednisolone (IVMP) pulse therapy in pediatric patients with EE to evaluate its efficacy and tolerability. Methods: This is a retrospective study from 2020 to 2023. Inclusion criteria were ≤18 years at the time of IVMP pulse therapy and at least 6 months of follow-up. Efficacy and outcome, defined as seizure reduction > 50% (responder rate), were evaluated at 6 and 9 months of therapy, and 6 months after therapy suspension; quality of life (QoL) was also assessed. Variables predicting positive post-IVMP outcomes were identified using statistical analysis. Results: The study included 21 patients, with a responder rate of 85.7% at 6 and 9 months of therapy, and 80.9% at 6 months after therapy suspension. Variables significantly predicting favorable outcome were etiology (p = 0.0475) and epilepsy type (p = 0.0475), with the best outcome achieved in patients with genetic epilepsy and those with encephalopathy related to electrical status epilepticus during slow-wave sleep (ESES). All patients evidenced improvements in QoL at the last follow-up, with no relevant adverse events reported. Conclusions: Our study confirmed the efficacy and high tolerability of IVMP pulse therapy in pediatric patients with EE. Genetic epilepsy and ESES were positive predictors of a favorable clinical outcome. QOL, EEG tracing, and postural-motor development showed an improving trend as well. IVMP pulse therapy should be considered earlier in patients with EE.
摘要：
背景：癫痫脑病（EE）的特征是严重的耐药性癫痫发作，早期发病，和不利的发展结果。本文讨论了静脉注射甲基强的松龙（IVMP）脉冲疗法在EE患儿中的应用，以评估其疗效和耐受性。方法：这是一项2020年至2023年的回顾性研究。纳入标准为IVMP脉冲治疗时≤18年，随访至少6个月。疗效和结果，定义为癫痫发作减少>50%(应答率)，在治疗6个月和9个月时进行评估，和治疗暂停后6个月；还评估了生活质量（QoL）。使用统计分析确定预测IVMP后阳性结果的变量。结果：该研究包括21例患者，在治疗6个月和9个月时，应答率为85.7%，治疗暂停后6个月为80.9%。显著预测有利结局的变量是病因(p=0.0475)和癫痫类型(p=0.0475)。在患有遗传性癫痫和患有与慢波睡眠（ESES）期间癫痫持续状态相关的脑病的患者中取得了最好的结果。所有患者在最后一次随访时都证明QoL有所改善，未报告相关不良事件。结论：我们的研究证实了IVMP脉冲疗法在小儿EE患者中的疗效和高耐受性。遗传性癫痫和ESES是良好临床结局的阳性预测因子。QOL,脑电图追踪,姿势运动发育也呈现改善趋势。对于EE患者，应尽早考虑IVMP脉冲治疗。
