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推进儿童癫痫的管理。

Advancing the management of childhood epilepsies.

机构信息

UCL-Institute of Child Health, Great Ormond Street Hospital for Children NHS Foundation Trust, London.

出版信息

Eur J Paediatr Neurol. 2013 Jul;17(4):334-47. doi: 10.1016/j.ejpn.2013.02.003. Epub 2013 Apr 1.

Abstract

Childhood epilepsies comprise a heterogeneous group of disorders and syndromes that vary in terms of severity, prognosis and treatment requirements. Effective management requires early, accurate recognition and diagnosis, and a holistic approach that addresses each individual's medical and psychosocial needs within the context of their overall health status and quality of life. With increasing understanding of underlying aetiologies, new approaches to management and treatment are emerging. For example, genetic testing is beginning to provide a tool to aid differential diagnosis and a means of predicting predisposition to particular types of epilepsy. Despite the availability of an increasing number of antiepileptic drugs (AEDs)--due not only to the development of new AEDs, but also to changes in regulatory requirements that have facilitated clinical development--seizure control and tolerability continue to be suboptimal in many patients, and there is therefore a continuing need for new treatment strategies. Surgery and other non-pharmacological treatments (e.g. vagus nerve stimulation, ketogenic diet) are already relatively well established in paediatric epilepsy. New pharmacological treatments include generational advances on existing AEDs and AEDs with novel modes of action, and non-AED pharmacological interventions, such as immunomodulation. Emerging technologies include novel approaches allowing the delivery of medicinal agents to specific areas of the brain, and 'closed-loop' experimental devices employing algorithms that allow treatment (e.g., electrical stimulation) to be targeted both spatially and temporally. Although in early stages of development, cell-based approaches (e.g., focal targeting of adenosine augmentation) and gene therapy may also provide new treatment choices in the future.

摘要

儿童癫痫包括一组异质性疾病和综合征,其严重程度、预后和治疗需求各不相同。有效的管理需要早期、准确的识别和诊断,以及一种整体方法,在考虑到个体的整体健康状况和生活质量的情况下,满足每个人的医疗和社会心理需求。随着对潜在病因的理解不断加深,新的管理和治疗方法正在出现。例如,基因测试开始提供一种辅助鉴别诊断的工具,并有助于预测特定类型癫痫的易感性。尽管越来越多的抗癫痫药物(AEDs)可供使用——不仅是由于新 AEDs 的开发,还由于监管要求的变化使临床开发变得更加便利——但许多患者的癫痫发作控制和耐受性仍不理想,因此仍然需要新的治疗策略。手术和其他非药物治疗(例如迷走神经刺激、生酮饮食)在儿科癫痫中已经相对成熟。新的药物治疗包括现有 AED 的代际进步和具有新型作用模式的 AED,以及非 AED 药物干预,如免疫调节。新兴技术包括允许将药物递送到大脑特定区域的新方法,以及采用允许治疗(例如电刺激)在空间和时间上靶向的算法的“闭环”实验设备。尽管处于早期开发阶段,但基于细胞的方法(例如,腺苷增强的靶向焦点)和基因治疗也可能在未来提供新的治疗选择。

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