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多发性硬化症的新治疗策略。

New treatment strategies in multiple sclerosis.

机构信息

Dept. of Clinical Neurosciences, Box 165 Addenbrooke's Hospital, Hills Road, Cambridge, CB2 0QQ, UK.

出版信息

Exp Neurol. 2010 Sep;225(1):34-9. doi: 10.1016/j.expneurol.2010.06.003. Epub 2010 Jun 12.

DOI:10.1016/j.expneurol.2010.06.003
PMID:20547155
Abstract

Multiple sclerosis is the most common, non-traumatic, disabling neurological disease of young adults, affecting an estimated two million people worldwide. At onset multiple sclerosis can be categorised clinically into relapsing remitting MS (RRMS - 85-90% of patients) or primary progressive MS (PPMS). Relapses typically present sub-acutely over hours to days with neurological symptoms persisting for days to weeks before they gradually dissipate. At first full recovery is the norm, later patients accumulate deficits and ultimately most convert to a secondary progressive phase (SPMS), characterised by deficits that increase in the absence of further relapses. The clinical picture reflects the complex interplay of focal inflammation, demyelination and axonal degeneration occurring within the central nervous system. Since the introduction of a genuine disease-modifying drug, interferon-beta1b in 1993, there has been a growing interest from academia and pharmaceutical companies alike in multiple sclerosis therapy. In part this effort has focused on investigating the "window of therapeutic opportunity" within the natural history of the disease: it is becoming increasingly clear that immunotherapies are not useful in the secondary phase of the disease but may offer long-term benefit if given early in the relapsing-remitting phase. In part, attention is being paid to the details of dosing and administration of the various licensed therapies, but there is also a significant research effort to explore new ways to treat the disease. In this review, we first sketch the landscape of novel therapies in multiple sclerosis and then discuss in detail approaches which are likely to emerge over the next few years.

摘要

多发性硬化症是最常见的、非外伤性的、导致年轻人残疾的神经退行性疾病,全球估计有 200 万人受其影响。多发性硬化症在发病时可临床分为复发缓解型多发性硬化症(RRMS-约 85-90%的患者)或原发性进展型多发性硬化症(PPMS)。复发通常在数小时到数天内亚急性发作,神经症状持续数天到数周后逐渐消退。起初完全恢复是常态,随后患者逐渐出现缺陷,最终大多数患者转化为继发性进展期(SPMS),其特征是在没有进一步复发的情况下,缺陷不断增加。临床表现反映了中枢神经系统内局灶性炎症、脱髓鞘和轴突变性的复杂相互作用。自 1993 年真正的疾病修饰药物干扰素-β1b 问世以来,学术界和制药公司对多发性硬化症治疗的兴趣日益浓厚。部分原因是因为人们专注于研究疾病自然史中的“治疗机会窗口”:越来越清楚的是,免疫疗法在疾病的继发性阶段没有用,但如果在复发缓解阶段早期给予治疗,可能会带来长期益处。部分原因是关注各种已批准治疗方法的剂量和管理细节,但也有大量研究工作旨在探索治疗该疾病的新方法。在这篇综述中,我们首先概述多发性硬化症的新型治疗方法,并详细讨论未来几年可能出现的治疗方法。

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New treatment strategies in multiple sclerosis.多发性硬化症的新治疗策略。
Exp Neurol. 2010 Sep;225(1):34-9. doi: 10.1016/j.expneurol.2010.06.003. Epub 2010 Jun 12.
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