多發性硬化症 (multiple sclerosis, MS) 是一種發生於中樞神經系統 (central nervous system, CNS) 的慢性發炎疾病，一般認為與自體反應性淋巴細胞媒介的免疫發炎機制有關，造成軸突受損、訊號傳遞異常，導致部分或全部的神經性失能。治療方式主要是透過調節免疫發炎反應或減少淋巴球穿透血腦屏壁 (blood-brain barrier, BBB) 進入CNS 等方式，降低復發頻率及延緩殘疾進展的速度，但是目前沒有任何一個藥物能完全預防或逆轉病程的惡化。
Cladribine 於2017 年歐洲藥品管理局 (European Medicines Agency, EMA) 核准使用於治療MS。服藥方式便利，即使停藥後還能維持超過2 年的控制期是其一大特色，對病人而言在藥物的選擇上能提供多一種選擇方案。本文將針對MS、cladribine藥理機轉、治療有效性與安全性等進行文獻回顧，供臨床專業人員參考。
Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system (CNS), in which autoreactive lymphocytes are believed to play a major pathophysiological role in the immune-mediated inflammation, causing axonal injury and the messages traveling abnormal, consequently leading to partial or complete episodes of neurological disability. Several disease-modifying therapies are used to reduce the frequency of relapses and delay disease progression, which act by regulating the inflammatory cytokines or reducing lymphocyte movement across the blood-brain barrier into the CNS. However, none of the therapies have been completely successful in preventing or reversing the progressive neurological deterioration.
The European Medicines Agency approved the use of cladribine for the treatment of MS in 2017. The primary distinguishing features of cladribine are its convenient dosing regimen and the sustainability of its treatment effect for more than 2 years without the need for further treatment. Therefore, cladribine could be an alternative option for several patients with MS. The purpose of this article is to provide an overview of the pathophysiology of MS and the mechanism of action, efficacy, and safety profile of cladribine.
