AlJumah Mohamed, Alkhawajah Mona Marwan, Qureshi Shireen, Al-Thubaiti Ibtisam, Ayoub Omar, Bohlega Saeed A, Bushnag Areej, Cupler Edward, Daif Abdulkader, El Boghdady Ahmed, Hassan Ahmed, Al Malik Yaser, Saeedi Jameelah, Al-Shamrany Fawzia, Shosha Eslam, Rieckmann Peter
King Fahad Medical City, Ministry of Health, Riyadh, Kingdom of Saudi Arabia.
King Faisal Specialist Hospital and Research Center, Riyadh, Kingdom of Saudi Arabia.
Neurol Ther. 2020 Jun;9(1):11-23. doi: 10.1007/s40120-020-00177-5. Epub 2020 Feb 13.
Immune reconstitution therapy (IRT) is an emerging management concept for multiple sclerosis, whereby a short course of treatment provides long-lasting suppression of disease activity. "Cladribine tablets 10 mg" refers to a total cumulative dose of cladribine given over 2 years (henceforth referred to as cladribine tablets 3.5 mg/kg); it is a relatively new treatment option that is hypothesised to act as an IRT acting preferentially on the adaptive immune system. A randomised, 2-year, placebo-controlled trial (CLARITY) showed that treatment with cladribine tablets reduced indices of disease activity (relapses, lesions on magnetic resonance images, disability progression) and that this effect outlasted the pharmacologic effect of the treatment on the immune system (mainly a reduction in circulating B and T cells, with little effect on components of the innate immune system such as monocytes). CLARITY Extension, a 2-year extension to this trial, demonstrated durable efficacy, also in patients who received the standard 2-year course of cladribine tablets 3.5 mg/kg and were re-randomised to placebo for a further 2 years. Relative risk reductions for relapse rate with cladribine tablets 3.5 mg/kg were similar for patients with or without prior high disease activity. Reductions in disability progression with cladribine tablets 3.5 mg/kg were higher in patients with prior high relapse rates with or without prior treatment non-response. In this review, we describe the therapeutic profile of cladribine tablets 3.5 mg/kg and provide practical information on initiating this treatment option in the most appropriate patients.
免疫重建疗法(IRT)是一种针对多发性硬化症的新兴治疗理念,即通过短期治疗实现对疾病活动的长期抑制。“10毫克克拉屈滨片”指的是两年内给予的克拉屈滨累计总剂量(以下简称3.5毫克/千克克拉屈滨片);这是一种相对较新的治疗选择,据推测其作为一种免疫重建疗法,优先作用于适应性免疫系统。一项为期两年的随机、安慰剂对照试验(CLARITY)表明,服用克拉屈滨片可降低疾病活动指标(复发、磁共振成像显示的病灶、残疾进展),且这种效果持续时间超过了该治疗对免疫系统的药理作用(主要是循环B细胞和T细胞减少,对先天免疫系统成分如单核细胞影响较小)。CLARITY扩展试验是该试验的两年期延伸,证明了其持久疗效,对于接受标准的两年疗程3.5毫克/千克克拉屈滨片治疗且再次随机接受安慰剂治疗两年的患者同样有效。无论之前疾病活动度高低,3.5毫克/千克克拉屈滨片降低复发率的相对风险降低幅度相似。对于之前复发率高且无论之前治疗有无反应的患者,3.5毫克/千克克拉屈滨片降低残疾进展的幅度更大。在本综述中,我们描述了3.5毫克/千克克拉屈滨片的治疗概况,并提供了在最合适的患者中启动这种治疗选择的实用信息。