Department of Neurology, University Hospital Center Zagreb, Zagreb, Croatia; School of Medicine, University of Zagreb, Zagreb, Croatia.
Department of Neurology, University Medical Centre Ljubljana, Ljubljana, Slovenia; Faculty of Medicine, University of Ljubljana, Slovenia.
J Neuroimmunol. 2023 Sep 15;382:578164. doi: 10.1016/j.jneuroim.2023.578164. Epub 2023 Jul 27.
Cladribine is an oral disease-modifying drug authorized by the European Medicine Agency for the treatment of highly active relapsing multiple sclerosis (MS).
To provide real-world evidence of cladribine's effectiveness and safety in people with MS (pwMS).
A retrospective observational multi-center, multi-national study of pwMS who were started on cladribine tablets in ten centers from five European countries.
We identified 320 pwMS treated with cladribine tablets. The most common comorbidities were arterial hypertension and depression. Three patients had resolved hepatitis B infection, while eight had positive Quantiferon test prior to cladribine commencement. There were six pwMS who had malignant diseases, but all were non-active. During year 1, 91.6% pwMS did not have EDSS worsening, 86.9% were relapse-free and 72.9% did not have MRI activity. During the second year, 90.2% did not experience EDSS worsening, 86.5% were relapse-free and 75.5% did not have MRI activity. NEDA-3 was present in 58.0% pwMS in year 1 and in 54.2% in year 2. In a multivariable logistic regression model age positively predicted NEDA-3 in year 1. The most common adverse events were infections and skin-related adverse events. Lymphopenia was noted in 54.7% of pwMS at month 2 and in 35.0% at month 6. Two pwMS had a newly discovered malignant disease, one breast cancer, and one melanoma, during the first year of treatment.
Our real-world data on the effectiveness and safety of cladribine tablets are comparable to the pivotal study and other real-world data with no new safety signals.
克拉屈滨是一种已在欧洲药品管理局获批用于治疗活跃复发型多发性硬化症（MS）的口服疾病修正治疗药物。
提供克拉屈滨治疗多发性硬化症患者（pwMS）的有效性和安全性的真实世界证据。
这是一项回顾性观察性、多中心、多国研究，纳入了来自五个欧洲国家的十个中心开始使用克拉屈滨片的 pwMS。
我们共确定了 320 例接受克拉屈滨片治疗的 pwMS。最常见的合并症是动脉高血压和抑郁症。3 例患者乙型肝炎感染已痊愈，8 例患者在开始使用克拉屈滨前 Quantiferon 检测结果阳性。6 例 pwMS患有恶性疾病，但均处于非活动期。在第 1 年，91.6%的 pwMS 疾病严重程度评估量表（EDSS）无恶化，86.9%无复发，72.9%无磁共振成像（MRI）活动性病变。在第 2 年，90.2%的 pwMS 无 EDSS 恶化，86.5%无复发，75.5%无 MRI 活动性病变。第 1 年和第 2 年分别有 58.0%和 54.2%的 pwMS 达到了无疾病活动、缓解和残疾进展（NEDA-3）。多变量逻辑回归模型显示年龄与第 1 年的 NEDA-3 呈正相关。最常见的不良事件是感染和皮肤相关不良事件。在第 2 个月和第 6 个月，分别有 54.7%和 35.0%的 pwMS 出现淋巴细胞减少。有 2 例 pwMS 在治疗的第 1 年内新发现恶性疾病，分别为乳腺癌和黑色素瘤。
我们的克拉屈滨片有效性和安全性的真实世界数据与关键研究和其他真实世界数据相当，未发现新的安全性信号。
