Potuznik Pavel, Drahota Jiri, Horakova Dana, Peterka Marek, Mazouchova Aneta, Matyas David, Pavelek Zbysek, Vachova Marta, Recmanova Eva, Stetkarova Ivana, Libertinova Jana, Mares Jan, Stourac Pavel, Grunermelova Marketa, Martinkova Alena, Adamkova Jana, Hradilek Pavel, Ampapa Radek, Dufek Michal, Kubala Havrdova Eva, Stastna Dominika
Department of Neurology, Faculty of Medicine and University Hospital in Pilsen, Charles University, Plzen, Czech Republic.
Department of Neurology and Centre of Clinical Neuroscience, First Faculty of Medicine, Charles University in Prague and General University Hospital, Prague, Czech Republic.
J Cent Nerv Syst Dis. 2024 Jul 24;16:11795735241262743. doi: 10.1177/11795735241262743. eCollection 2024.
Cladribine, a selective immune reconstitution therapy, is approved for the treatment of adult patients with highly active multiple sclerosis (MS).
Provide experience with cladribine therapy in a real-world setting.
This is a registry-based retrospective observational cohort study. First, using data from the Czech nationwide registry ReMuS, we analysed patients who initiated cladribine from September 1, 2018 to December 31, 2021. Second, we analysed a subgroup of patients who initiated cladribine between September 1, 2018 to June 30, 2020, thus possessing a follow-up period of at least 2 years. We evaluated demographic and MS characteristics including disease-modifying therapies (DMTs) before and after cladribine administration, relapses, Expanded Disability Status Scale (EDSS), and adherence.
In total, 617 patients (335 with follow-up of at least 2 years) started cladribine therapy in the study period (mean age 37.0, mean disease duration 8.4 years, 74.1% females). In most cases, cladribine was administered as a second-line drug, a total of 80.7% had been escalated from a platform DMT. During 2 years before cladribine initiation, the average annualised relapse rate (ARR) was .67. Following cladribine initiation, the ARR decreased to .28 in the first year and .22 in the second year. Overall, across the entire two-year treatment period, 69.0% of patients were relapse-free and the average ARR was .25. As for EDSS development, the median baseline EDSS was 2.5 and remained stable even after 24 months. The adherence to treatment ranged of around 90%.
This nationwide study confirms the efficacy of cladribine in real-world settings, especially in patients who are not treatment-naïve. In addition, the study shows an exceptionally high adherence rate, a finding that underscores the invaluable role of cladribine, but also the value of registry-based studies in capturing real-world clinical practice.
克拉屈滨是一种选择性免疫重建疗法，已被批准用于治疗成年高度活动性多发性硬化症（MS）患者。
提供克拉屈滨在实际临床环境中的治疗经验。
这是一项基于登记处的回顾性观察队列研究。首先，利用捷克全国性登记处ReMuS的数据，我们分析了2018年9月1日至2021年12月31日开始使用克拉屈滨的患者。其次，我们分析了2018年9月1日至2020年6月30日开始使用克拉屈滨的患者亚组，这些患者的随访期至少为2年。我们评估了人口统计学和MS特征，包括克拉屈滨给药前后的疾病修饰疗法（DMT）、复发情况、扩展残疾状态量表（EDSS）以及依从性。
在研究期间，共有617名患者（335名随访期至少为2年）开始使用克拉屈滨治疗（平均年龄37.0岁，平均病程8.4年，女性占74.1%）。在大多数情况下，克拉屈滨作为二线药物使用，共有80.7%的患者从平台DMT升级而来。在开始使用克拉屈滨前的2年中，年化复发率（ARR）平均为0.67。开始使用克拉屈滨后，第一年的ARR降至0.28，第二年降至0.22。总体而言，在整个两年治疗期内，69.0%的患者无复发，平均ARR为0.25。至于EDSS进展，基线EDSS中位数为2.5，即使在24个月后仍保持稳定。治疗依从率约为90%。
这项全国性研究证实了克拉屈滨在实际临床环境中的疗效，尤其是在非初治患者中。此外，该研究显示出极高的依从率，这一发现强调了克拉屈滨的重要作用，也凸显了基于登记处的研究在获取实际临床实践方面的价值。
