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治疗复发缓解型多发性硬化症的新兴口服药物。

Emerging oral drugs for relapsing-remitting multiple sclerosis.

机构信息

Dipartimento Testa Collo, Azienda Ospedaliera S. Camillo-Forlanini, Rome, Italy.

出版信息

Expert Opin Emerg Drugs. 2011 Dec;16(4):697-712. doi: 10.1517/14728214.2011.642861. Epub 2011 Dec 7.

Abstract

INTRODUCTION

Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system (CNS), traditionally considered to be an autoimmune, demyelinating disease. The last two decades have witnessed the introduction of several therapies for MS. At present, there are five licensed first-line, disease-modifying drugs (DMDs) in MS and two second-line treatments. Nevertheless, in clinical practice DMDs or immunosuppressive treatments are frequently associated with suboptimal response in terms of efficacy and several side effects leading to poor patient adherence.

AREAS COVERED

Since MS is a chronic disease, DMDs require long-term, regular injection or monthly parenteral infusions, which may be uncomfortable and inconvenient for the patient. Thus, there is an important need for new therapeutic strategies, especially those that may offer greater patient satisfaction in order to optimize therapeutic outcomes. Currently, five oral therapies are in Phase III development or have recently been approved for the treatment of relapsing-remitting MS: cladribine and fingolimod, the first approved in Russia and Australia, the latter is more widespread. Fumaric acid (BG-12), teriflunomide (A77126 or HMR1726) and laquinimod (ABR-215062) are in Phase III trials. Details of these five drugs will be covered in this review.

EXPERT OPINION

Preliminary results indicate that oral medications are as effective as, or possibly more effective than, current injectable formulations. It is believable that improved outcomes will translate into higher real and perceived efficacy rates and contribute to improved adherence. The decision to switch established patients from injectable to oral medications will be made on balancing the efficacy and tolerability of the patient's existing therapy and their compliance history, even though safety is likely to become the most important factor in the future development of MS drugs.

摘要

简介

多发性硬化症(MS)是一种中枢神经系统(CNS)的慢性炎症性疾病,传统上被认为是一种自身免疫性、脱髓鞘疾病。在过去的二十年中,已经引入了几种治疗多发性硬化症的方法。目前,多发性硬化症有五种许可的一线、疾病修正药物(DMD)和两种二线治疗方法。然而,在临床实践中,DMD 或免疫抑制治疗经常在疗效方面表现出不理想的反应,并且存在多种副作用,导致患者的依从性较差。

涵盖的领域

由于多发性硬化症是一种慢性疾病,DMD 需要长期、定期的注射或每月的静脉输注,这可能会给患者带来不适和不便。因此,需要新的治疗策略,特别是那些可能提供更高患者满意度的策略,以优化治疗效果。目前,有五种口服疗法正在进行 III 期开发或最近已被批准用于治疗复发缓解型多发性硬化症:克拉屈滨和芬戈莫德,前者在俄罗斯和澳大利亚首次获得批准,后者更为广泛。富马酸(BG-12)、特立氟胺(A77126 或 HMR1726)和拉喹莫德(ABR-215062)正在进行 III 期试验。这五种药物的详细信息将在本综述中介绍。

专家意见

初步结果表明,口服药物与目前的注射制剂一样有效,甚至可能更有效。可以相信,改善的结果将转化为更高的实际和感知疗效率,并有助于提高依从性。在从注射药物转换为口服药物时,应根据患者现有治疗的疗效和耐受性以及他们的依从性历史来平衡,尽管安全性可能成为多发性硬化症药物未来发展的最重要因素。

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