Holmøy Trygve, Torkildsen Øivind, Myhr Kjell-Morten
a Department of Neurology , Akershus University Hospital , Lørenskog , Norway.
b Institute of Clinical Medicine , Univeristy of Oslo , Oslo , Norway.
Expert Opin Pharmacother. 2017 Oct;18(15):1627-1635. doi: 10.1080/14656566.2017.1372747. Epub 2017 Sep 11.
Despite recent progress, currently available therapies for relapsing remitting multiple sclerosis (MS) are only partly effective, and their use is limited by tolerability and safety issues, as well as high cost. Cladribine was originally rejected by the regulatory authorities in both the European Union and USA in 2011, but in June 2017 the European Medical Agency recommended marketing authorization for treatment of aggressive relapsing MS. Areas covered: We provide an update on chemistry, mechanism of action, efficacy and safety of cladribine for the treatment of MS. Expert opinion: Cladribine is well tolerated, it is dosed orally in cycles of one year, the need for regular blood testing during treatment is likely limited, and the two-year efficacy data for treatment of relapsing MS are at least in the same range as the most efficient licensed treatments. The increased risk of malignancies reported in the pivotal trial seems to be caused by unexpectedly low numbers of malignancies in the placebo group. Cladribine could therefore be an alternative for many patients with relapsing remitting MS. The main caveat is the lack of long term efficacy and safety data. Currently there are insufficient data to guide further treatment of patients who have completed two treatment cycles of cladribine.
尽管最近取得了进展，但目前用于复发缓解型多发性硬化症（MS）的疗法仅部分有效，其应用受到耐受性、安全性问题以及高成本的限制。克拉屈滨最初在2011年被欧盟和美国的监管机构拒绝，但在2017年6月，欧洲药品管理局建议批准其用于治疗侵袭性复发型MS的上市许可。涵盖领域：我们提供了关于克拉屈滨治疗MS的化学性质、作用机制、疗效和安全性的最新信息。专家意见：克拉屈滨耐受性良好，每年口服给药一个周期，治疗期间定期进行血液检测的必要性可能有限，治疗复发型MS的两年疗效数据至少与最有效的已获许可治疗方法处于同一范围。关键试验中报告的恶性肿瘤风险增加似乎是由安慰剂组中意外低的恶性肿瘤数量引起的。因此，克拉屈滨可能是许多复发缓解型MS患者的一种替代选择。主要的警告是缺乏长期疗效和安全性数据。目前，对于已完成两个克拉屈滨治疗周期的患者，没有足够的数据来指导进一步治疗。
