小儿多发性硬化症的现有和新兴治疗方案

Sclerosis Pub Date : 2024-04-01 DOI:10.3390/sclerosis2020007
Artemis Mavridi, Maria-Eleni Bompou, Aine Redmond, Paraschos Archontakis-Barakakis, George D. Vavougios, D. Mitsikostas, T. Mavridis
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引用次数: 0

摘要

小儿多发性硬化症(POMS)的特点是在 18 岁之前发病,这种疾病正日益受到重视。约有 5% 的多发性硬化症患者在 18 岁前发病,不到 1% 的患者在 10 岁前发病。尽管少见,但小儿多发性硬化症表现出明显的特征,发病年龄较小,疾病进展相对较慢。尽管病程进展较慢,但与成人发病型多发性硬化症患者相比,小儿多发性硬化症患者达到残疾里程碑的年龄历来较早。虽然各种免疫调节药物在多发性硬化症的治疗中表现出显著的疗效,如降低复发率、减慢磁共振成像(MRI)上脑部病变的累积速度等,但大多数常用于成人多发性硬化症的疾病修饰疗法(DMTs)都没有通过儿科临床试验进行评估。目前的证据主要来自观察性研究。本综述旨在整合现有知识,特别是针对儿科多发性硬化症的现有 DMTs 的作用机制、疗效、安全性和推荐剂量。此外,本综述还概述了最新进展,并探讨了仍处于开发阶段的潜在药物,从而为治疗多发性硬化症的现状和未来前景提供了一个全面的概览。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Current and Emerging Treatment Options in Pediatric Onset Multiple Sclerosis
Pediatric onset multiple sclerosis (POMS), characterized by the onset of multiple sclerosis before the age of 18, is gaining increased recognition. Approximately 5 percent of MS cases manifest before the age of 18, with less than 1 percent occurring before the age of 10. Despite its rarity, pediatric MS exhibits distinct characteristics, with an association between younger age at onset and a comparatively slower disease progression. Despite this slower progression, individuals with POMS historically reach disability milestones at earlier ages than those with adult-onset multiple sclerosis. While various immunomodulatory agents demonstrate significant benefits in MS treatment, such as reduced relapse rates and slower accumulation of brain lesions on magnetic resonance imaging (MRI), the majority of disease-modifying therapies (DMTs) commonly used in adult MS lack evaluation through pediatric clinical trials. Current evidence is predominantly derived from observational studies. This comprehensive review aims to consolidate existing knowledge on the mechanisms of action, efficacy, safety profiles, and recommended dosages of available DMTs specifically in the context of pediatric MS. Furthermore, this review outlines recent advancements and explores potential medications still in developmental stages, providing a thorough overview of the current landscape and future prospects for treating POMS.
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