复发缓解型多发性硬化症的治疗进展:口服富马酸二甲酯(BG-12)的作用。

Degenerative Neurological and Neuromuscular Disease Pub Date : 2015-05-21 eCollection Date: 2015-01-01 DOI:10.2147/DNND.S68723
A Scott Nielsen
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引用次数: 1

摘要

多发性硬化症是一种复杂的中枢神经系统慢性炎症性疾病,全球约有230万人受到影响。基因研究已经发现了100多个与该疾病相关的免疫相关基因,并提供了许多潜在的治疗靶点。迄今为止,美国食品和药物管理局批准了13种旨在影响异常免疫系统的疾病修饰疗法,可用于疾病复发形式的适应症。BG-12是一种新型的口服多发性硬化症治疗药物,具有独特的作用机制,可以激活Nrf2抗氧化途径。尽管人们热衷于多种治疗方案,包括口服治疗方案,但医生面临着一项艰巨的任务,即在没有敏感的临床生物标志物的情况下,为患者提供关于最佳治疗方案排序的指导,以使特定治疗的假定作用机制与患者的特定病理生理相匹配。此外,虽然BG-12有首选的给药途径,但用于指导临床咨询的安全性数据有限。富马酸二甲酯(DMF或BG-12)是三种可用的口服疗法之一,本文将从其药代动力学特征、推测的作用机制、临床有效性、安全性、耐受性和患者报告的经验等方面进行讨论。将讨论BG-12作为一线治疗和测序治疗的潜力,以帮助从natalizumab过渡。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Advances in the management of relapsing-remitting multiple sclerosis: role of oral dimethyl fumarate (BG-12).

Multiple sclerosis is a complex and chronic inflammatory disease of the central nervous system which affects an estimated 2.3 million individuals worldwide. Genetic research has uncovered over 100 immune-related genes associated with the disease and has provided a multitude of potential therapeutic targets. To date, 13 US Food and Drug Administration-approved disease-modifying therapies designed to influence the aberrant immune system are available for the indication of relapsing forms of the disease. BG-12 is a novel oral multiple sclerosis therapeutic with a unique putative mechanism of action that activates the Nrf2 anti-oxidant pathway. Despite the enthusiasm for multiple therapeutic options, including oral options, the practitioner is faced with the difficult task of providing guidance for patients regarding optimal sequencing of therapeutics without sensitive clinical biomarkers to match a particular therapy's putative mechanism of action to the patient's specific pathophysiology. Moreover, while BG-12 has a preferred route of administration, there is limited safety data with which to guide counseling in the clinic. Dimethyl fumarate (DMF or BG-12) is one of three available oral therapies which will be discussed in this review in terms of its pharmacokinetic profile, putative mechanism of action, clinical effectiveness, safety, tolerance, and patient-reported experience. BG-12's potential as a first-line therapy and as a sequencing therapeutic to aid in transition off natalizumab will be discussed.

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