Department of Neurology with Institute of Translational Neurology, University Hospital Münster, Münster, Germany.
Department of Neurology and Center for Translational and Behavioral Neurosciences (C-TNBS), University Medicine Essen, Essen, Germany.
Mult Scler. 2022 Feb;28(2):257-268. doi: 10.1177/13524585211012227. Epub 2021 May 12.
Oral cladribine has been approved for the treatment of relapsing multiple sclerosis (MS) yet real-world evidence regarding its effectiveness and safety remains scarce.
To evaluate efficacy and safety outcomes of MS patients following induction of cladribine.
We evaluated our prospective cohort of cladribine-treated MS patients from two tertiary centres in Germany. Relapses, disability worsening and occurrence of new or enlarging T2-hyperintense magnetic resonance imaging (MRI) lesions were assessed as well as lymphocyte counts and herpes virus infections.
Among 270 patients treated with cladribine, we observed a profound reduction of both relapses and new or enlarging MRI lesions. Treatment appeared more efficacious, especially in patients without previous therapy or following platform substances. Patients switching from natalizumab were prone to re-emerging disease activity. Among patients following dimethyl fumarate pre-treatment, severe lymphopenia was common and associated with increased rates of herpes virus manifestations.
Overall, we observed an efficacy and safety profile of cladribine consistent with data from the phase 3 clinical trial. However, patients switching from natalizumab experienced suboptimal disease control beyond rebound activity following cessation of natalizumab. Furthermore, dimethyl fumarate pre-treatment was associated with a profound risk of developing severe lymphopenia and subsequent herpes virus infections.
口服克拉屈滨已被批准用于治疗复发型多发性硬化症（MS），但关于其疗效和安全性的真实世界证据仍然很少。
评估接受克拉屈滨诱导治疗的 MS 患者的疗效和安全性结局。
我们评估了来自德国两个三级中心的接受克拉屈滨治疗的 MS 患者的前瞻性队列。评估了复发、残疾恶化以及新出现或扩大的 T2 高信号磁共振成像（MRI）病变的发生情况，以及淋巴细胞计数和疱疹病毒感染情况。
在 270 名接受克拉屈滨治疗的患者中，我们观察到复发和新出现或扩大的 MRI 病变均显著减少。治疗效果更为显著，尤其是在无前期治疗或无平台药物治疗的患者中。从那他珠单抗转换过来的患者容易出现疾病活动再次出现。在接受二甲基呋喃酮预处理的患者中，严重的淋巴细胞减少很常见，且与疱疹病毒表现的发生率增加相关。
总体而言，我们观察到克拉屈滨的疗效和安全性与 3 期临床试验数据一致。然而，从那他珠单抗转换过来的患者在停止那他珠单抗后，除了反弹活动外，疾病控制效果不佳。此外，二甲基呋喃酮预处理与严重淋巴细胞减少和随后的疱疹病毒感染的风险显著增加有关。
