Department of Neurology, Multiple Sclerosis Centre Amsterdam, Vrije University Medical Centre, Amsterdam, Netherlands.
Lancet Neurol. 2011 Nov;10(11):1026-34. doi: 10.1016/S1474-4422(11)70228-9.
The armamentarium for the treatment of relapsing-remitting multiple sclerosis (RRMS) is increasing rapidly. Several oral treatments have shown benefit and will generate much interest because of the convenience of such administration. However, availability of convenient oral drugs will not necessarily translate into clinical effectiveness and safety. Here, we provide an interim report about the mechanisms of action, and efficacy and safety results that have been reported since January, 2010, for five new oral drugs. Additionally, we draw attention to issues that neurologists and patients will encounter when considering the use of new oral drugs.
Positive results have been reported for five new oral drugs for RRMS--fingolimod, cladribine, teriflunomide, laquinimod, and dimethyl fumarate--in phase 3 studies; a few new oral drugs are likely to be approved for RRMS soon. WHERE NEXT?: Emerging oral treatments are ushering in a new era in the treatment of MS, providing not only new treatment options but also new challenges. Since data for some of the new drugs have not been reported in peer-reviewed journals yet and safety profiles are not yet fully developed, opinions about the use of these new oral drugs in practice are preliminary and tentative. Practice will evolve with time as information and experience accumulates. Of importance will be results from comparator trials, information about management of patients with breakthrough disease, results from long-term safety studies, and results of studies to assess the potential for neuroprotective effects of the new drugs.
治疗复发缓解型多发性硬化症（RRMS）的手段正在迅速增加。几种口服治疗方法已显示出疗效，由于其给药的便利性，将会引起极大关注。然而，方便的口服药物的可用性不一定会转化为临床疗效和安全性。在这里，我们提供了自 2010 年 1 月以来，五种新的口服药物的作用机制、疗效和安全性结果的中期报告。此外，我们提请神经病学家和患者注意在考虑使用新的口服药物时会遇到的问题。
五项新的 RRMS 口服药物——芬戈莫德、克拉屈滨、特立氟胺、拉喹莫德和富马酸二甲酯——在 3 期研究中取得了阳性结果；一些新的口服药物可能很快就会被批准用于 RRMS。接下来会怎样？新兴的口服疗法正在为 MS 的治疗带来一个新时代，不仅提供了新的治疗选择，还带来了新的挑战。由于一些新药的数据尚未在同行评议的期刊上报道，且安全性概况尚不完全成熟，因此关于这些新的口服药物在实践中的应用的意见是初步的和试探性的。随着时间的推移，随着信息和经验的积累，实践将不断发展。重要的是来自对照试验的结果、关于突破性疾病患者管理的信息、长期安全性研究的结果以及评估新药潜在神经保护作用的研究结果。
