结节性硬化综合征早期癫痫发作治疗策略的演变:回顾与治疗算法。

IF 4.6 Q2 MATERIALS SCIENCE, BIOMATERIALS ACS Applied Bio Materials Pub Date : 2024-11-01 DOI:10.1016/j.yebeh.2024.110123
Debopam Samanta
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引用次数: 0

摘要

结节性硬化综合征(TSC)是一种遗传性疾病,其特点是多系统受累,80%-90%的患者会患上癫痫,而且通常在婴儿期就开始发作。TSC患者的早期癫痫发作与不良的神经发育预后有关,这凸显了及时有效治疗的重要性。本综述探讨了幼儿TSC相关性癫痫发作不断发展的治疗前景,重点关注最近获批或扩大许可的三种疗法:维格巴曲林、依维莫司和大麻二酚。根据临床试验和实际证据对这些疗法的疗效和安全性进行了研究,重点关注它们在治疗幼儿癫痫发作中的应用。此外,还仔细回顾了在临床研究中抢先使用维加巴曲林的情况。本文提出了一种治疗算法,强调早期诊断、及时开始适当的治疗,并采用循序渐进的方法来控制婴儿痉挛症和局灶性癫痫发作。该算法将这些新疗法与传统的抗癫痫药物和非药物疗法结合起来。文中讨论了在优化治疗策略、减少副作用和改善长期疗效方面所面临的挑战。本综述旨在指导临床医生驾驭与 TSC 相关的早期癫痫发作的复杂局面,最终为改善这一弱势群体的癫痫发作控制和发育预后而努力。
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Evolving treatment strategies for early-life seizures in Tuberous Sclerosis Complex: A review and treatment algorithm
Tuberous sclerosis Complex (TSC) is a genetic disorder characterized by multisystem involvement, with epilepsy affecting 80–90% of patients, often beginning in infancy. Early-life seizures in TSC are associated with poor neurodevelopmental outcomes, underscoring the importance of timely and effective management. This review explores the evolving treatment landscape for TSC-associated seizures in young children, focusing on three recently approved or license-expanded therapies: vigabatrin, everolimus, and cannabidiol. The efficacy and safety profiles of these treatments are examined based on clinical trials and real-world evidence, with a focus on their use in treating seizures in young children. The preemptive use of vigabatrin in clinical studies has also been carefully reviewed. A treatment algorithm is proposed, emphasizing early diagnosis, prompt initiation of appropriate therapy, and a stepwise approach to managing both infantile spasms and focal seizures. The algorithm incorporates these newer therapies alongside traditional antiseizure medications and non-pharmacological approaches. Challenges in optimizing treatment strategies, minimizing side effects, and improving long-term outcomes are discussed. This review aims to guide clinicians in navigating the complex landscape of early-life seizures associated with TSC, ultimately striving for improved seizure control and better developmental outcomes in this vulnerable population.
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来源期刊
ACS Applied Bio Materials
ACS Applied Bio Materials Chemistry-Chemistry (all)
CiteScore
9.40
自引率
2.10%
发文量
464
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