克拉屈滨片治疗晚期复发缓解型多发性硬化症：一项4年随访的真实世界、多中心、回顾性队列研究。
Cladribine tablets as therapy for advanced relapsing-remitting multiple sclerosis: a 4-year follow-up real-world, multi-center, retrospective, cohort study.
作者信息
Pogoda-Wesołowska Aleksandra, Stępień Adam, Wnuk Marcin, Marona Monika, Tokarz-Kupczyk Elżbieta, Piasecka-Stryczyńska Karolina, Rejdak Konrad, Jamroz-Wiśniewska Anna, Adamczyk-Sowa Monika, Kubicka-Bączyk Katarzyna, Kurkowska-Jastrzębska Iwona, Kurowska Katarzyna, Puz Przemysław, Kułakowska Alina, Chorąży Monika, Brola Waldemar, Bartosik-Psujek Halina
机构信息
Department of Neurology, Military Institute of Medicine, Warsaw, Poland.
Department of Neurology, Jagiellonian University Medical College, University Hospital in Krakow, Kraków, Poland.
出版信息
Front Neurol. 2025 Jul 3;16:1626317. doi: 10.3389/fneur.2025.1626317. eCollection 2025.
INTRODUCTION
Cladribine tablets (CladT) are a high-efficacy disease-modifying therapy recommended for the treatment of relapsing-remitting multiple sclerosis (RRMS) particularly in early disease. This study is aimed to evaluate the long-term efficacy of CladT in population of Polish RRMS patients, with more advanced disease.
METHODS
This retrospective observational study included patients with RRMS who started CladT treatment between December 2019 and November 2023. Collected data included prior treatments, annualized relapse rate (ARR), magnetic resonance imaging (MRI) activity, Expanded Disability Status Scale (EDSS) score, no evidence of disease activity (NEDA-3), lymphocyte counts, and safety outcomes were collected.
RESULTS
Of the 230 patients (8.3% treatment-naïve, mean disease duration 9.2 years), follow-up data were available up to year 1 for 222 patients, year 2 for 154 patients, year 3 for 87 patients and year 4 for 31 patients. The ARR decreased from 1.42 at baseline to 0.26, 0.22, and 0.36 in years 1, 2, and 3, respectively. The proportion of relapse-free patients increased from 13.9% at baseline to 76.8% in year 1, 82% in year 2 and 75.4% in year 3 with no relapses reported in year 4. The proportion of patients with active MRI lesions declined from 90.4% at baseline to 36.3% in year 1, 25.2% in year 2, 45.9% in year 3 and 8.3% in year 4. Stable or improved EDSS was observed in 85.9% of patients in year 1, 80.8% in year 2, 73.7% in year 3 and 88.9% in year 4. NEDA-3 status was achieved in 47.4% of patients in year 1, 51.0% in year 2, 40.4% in year 3 and 71.4% in year 4. Adverse events were reported in 16.7% of patients in years 1-2 and in 6.3% of patients in year 3.
DISCUSSION
The results indicate that CladT is effective and safe in Polish patients with RRMS, characterized by high disease activity, delayed treatment initiation, and multiple number of prior therapies.
引言
克拉屈滨片（CladT）是一种高效的疾病修饰疗法，推荐用于治疗复发缓解型多发性硬化症（RRMS），尤其是在疾病早期。本研究旨在评估CladT在波兰RRMS患者群体中的长期疗效，这些患者的疾病更为晚期。
方法
这项回顾性观察性研究纳入了2019年12月至2023年11月开始接受CladT治疗的RRMS患者。收集的数据包括既往治疗情况、年化复发率（ARR）、磁共振成像（MRI）活动、扩展残疾状态量表（EDSS）评分、无疾病活动证据（NEDA-3）、淋巴细胞计数，并收集了安全性结果。
结果
230例患者（8.3%为初治患者，平均病程9.2年）中，222例患者有1年的随访数据，154例患者有2年的随访数据，87例患者有3年的随访数据，31例患者有4年的随访数据。ARR分别从基线时的1.42降至第1年的0.26、第2年的0.22和第3年的0.36。无复发患者的比例从基线时的13.9%增至第1年的76.8%、第2年的82%和第3年的75.4%，第4年无复发报告。有MRI活动病灶的患者比例从基线时的90.4%降至第1年的36.3%、第2年的25.2%、第3年的45.9%和第4年的8.3%。第1年85.9%的患者、第2年80.8%的患者、第3年73.7%的患者和第4年88.9%的患者观察到EDSS稳定或改善。第1年47.4%的患者、第2年51.0%的患者、第3年40.4%的患者和第4年71.4%的患者达到NEDA-3状态。第1 - 2年16.7%的患者和第3年6.3%的患者报告了不良事件。
讨论
结果表明，CladT在波兰RRMS患者中有效且安全，这些患者疾病活动度高、治疗起始延迟且既往接受过多种治疗。
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