Oreja-Guevara C, García-Merino J A, Saiz A, Rodríguez-Antigüedad A, Álvarez-Cermeño J C, Estrada-Pérez V, Izquierdo G, Fernández O
Hospital Clínico San Carlos, 28040 Madrid, España.
Hospital Universitario Puerta de Hierro-Majadahonda, 28035 Majadahonda, España.
Rev Neurol. 2019 Dec 17;69(s02):1-9. doi: 10.33588/rn.69s02.2019380.
Cladribine is a prodrug, a synthetic analogue of deoxyadenosine, approved for use as selective immune reconstitution therapy in very active recurring multiple sclerosis in adults.
To review the development of the drug, its mechanism of action and the efficacy and safety data obtained to date, as well as to establish recommendations of Spanish experts for its use in clinical practice.
The treatment of multiple sclerosis has been simplified with cladribine tablets, and two short courses of administration for two consecutive years (maximum 20 days) are needed to maintain an efficacy of up to four years after the first dose. Results of clinical trials have demonstrated the safety, tolerability and long-term efficacy of cladribine tablets in patients with recurring multiple sclerosis. Thus, patients treated with cladribine presented a significant reduction in the rate of flare-ups, in the risk of disability progression and in the development of new lesions in magnetic resonance imaging compared to those treated with placebo. In terms of safety, the treated patients had a higher frequency of lymphopenia, in relation to its mechanism of action, and of infections by herpes zoster virus. Long-term results with eight years' follow-up have shown that treated patients are not at greater risk of developing serious events, such as malignant neoplasms or opportunistic infections.
Cladribine is the first short-course oral therapy that has been shown to be effective and safe in patients with very active recurring multiple sclerosis, and with a sustained effect over time. The recommendations of Spanish experts on its usage are a fundamental complement to the considerations described by the regulatory agencies.
克拉屈滨是一种前体药物，是脱氧腺苷的合成类似物，已被批准用于成人活动性复发型多发性硬化症的选择性免疫重建治疗。
回顾该药物的研发历程、作用机制以及迄今获得的疗效和安全性数据，并制定西班牙专家在临床实践中使用该药物的建议。
克拉屈滨片简化了多发性硬化症的治疗，连续两年（最长20天）进行两个短疗程给药，即可在首次给药后维持长达四年的疗效。临床试验结果证明了克拉屈滨片在复发型多发性硬化症患者中的安全性、耐受性和长期疗效。因此，与接受安慰剂治疗的患者相比，接受克拉屈滨治疗的患者在发作率、残疾进展风险以及磁共振成像中新病灶的发生方面均有显著降低。在安全性方面，就其作用机制而言，接受治疗的患者淋巴细胞减少的频率较高，且带状疱疹病毒感染的频率也较高。八年随访的长期结果表明，接受治疗的患者发生严重事件（如恶性肿瘤或机会性感染）的风险并不更高。
克拉屈滨是首个被证明对活动性复发型多发性硬化症患者有效且安全、并具有长期持续疗效的短疗程口服疗法。西班牙专家关于其用法的建议是对监管机构所述考量的重要补充。
