Department of Neurosciences, S Camillo Forlanini Hospital, Rome, Italy;
Ther Clin Risk Manag. 2010 Sep 7;6:391-9. doi: 10.2147/tcrm.s6639.
Multiple sclerosis (MS) is a chronic inflammatory disorder of the central nervous system (CNS) that represents one of the first causes of neurological disability in young adults. Although the pathogenesis of MS is still unclear, an autoimmune mechanism has been demonstrated. According to this evidence in the last 15 years different treatments acting on the immune system have been developed. Current disease-modifying drugs (DMDs) for MS require regular and frequent parenteral administration and are associated with limited long-term treatment adherence. Moreover the clinical efficacy of these disease-modifying drugs is suboptimal. Thus, there is an important need for the development of new therapeutic strategies. Several oral therapies (fingolimod, fumaric acid, teriflunomide, laquinimod) are in development; Among these cladribine is the only therapy with the potential for short-course dosing. Cladribine is an immunosuppressant that offers sustained regulation of the immune system through a preferential lymphocyte depleting action. Cladribine has a well-characterized and well-known safety profile, derived from more than 15 years of use of the parenteral formulation both in the oncology field and in MS. This paper reviews the new oral emerging treatments and presents the available data about the use of cladribine in MS and the future perspective of its clinical use.
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,是年轻人神经功能障碍的首要原因之一。尽管 MS 的发病机制尚不清楚,但已证实存在自身免疫机制。根据这一证据,在过去的 15 年中,已经开发了针对免疫系统的不同治疗方法。目前用于 MS 的疾病修正药物(DMD)需要定期和频繁的注射给药,并且与有限的长期治疗依从性相关。此外,这些疾病修正药物的临床疗效并不理想。因此,迫切需要开发新的治疗策略。几种口服疗法(芬戈莫德、富马酸、特立氟胺、拉喹莫德)正在开发中;在这些药物中,克拉屈滨是唯一具有短疗程给药潜力的治疗药物。克拉屈滨是一种免疫抑制剂,通过优先淋巴细胞耗竭作用提供对免疫系统的持续调节。克拉屈滨具有良好的特征和已知的安全性,源自 15 多年来在肿瘤学领域和 MS 中使用的静脉制剂。本文综述了新兴的口服治疗方法,并介绍了克拉屈滨在 MS 中的应用的现有数据及其未来的临床应用前景。