多发性硬化症患者使用克拉屈滨片剂的安全性和有效性：来自单中心真实世界队列的结果。
Safety and effectiveness of cladribine tablets for multiple sclerosis: Results from a single-center real-world cohort.
机构信息
Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Biomedical Research Institute (BIOMED), Agoralaan, Diepenbeek 3590, Belgium; Noorderhart, Revalidatie en MS, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Rehabilitation Research Center, Agoralaan, Diepenbeek 3590, Belgium.
Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Biomedical Research Institute (BIOMED), Agoralaan, Diepenbeek 3590, Belgium; UHasselt, Data Science Institute, Agoralaan, Diepenbeek 3590, Belgium; The D-Lab, Department of Precision Medicine, GROW - School for Oncology, Maastricht University, Universiteitssingel 40, Maastricht 6229 ER, the Netherlands.
出版信息
Mult Scler Relat Disord. 2023 Jul;75:104735. doi: 10.1016/j.msard.2023.104735. Epub 2023 Apr 25.
BACKGROUND
Cladribine tablets are a highly effective immune reconstitution therapy licensed for treating relapsing multiple sclerosis (RMS) in Europe since 2017. Currently, there is a high demand for real-world data from different clinical settings on the effectiveness and safety profile of cladribine in MS.
METHODS
Within this report, we retrospectively evaluated the outcomes of RMS patients who received cladribine between August 2018 and November 2021 at our Belgian institute. Patients with data for three effectiveness endpoints, more specifically, relapses, MRI observations, and confirmed disability worsening were incorporated into the analysis of 'no evidence of disease activity' (NEDA-3) re-baselined at 3 months. Safety endpoints included lymphopenia, liver transaminases, and adverse events (AEs) during follow-up. Descriptive statistics and time-to-event analysis were performed, including subgroup analysis by pre-treatment.
RESULTS
Of the 84 RMS patients included in this study (age 42 [33-50], 64.3% female, diagnosis duration 6 [2-11] years, baseline EDSS 2.5 [1.5-3.6]), 14 (16.7%) patients experienced relapses, while disability progression and brain MRI activity occurred in 8.5% (6/71) and 6.3% (5/79). This resulted in 72.6% (n = 69, standard error 6%) retaining NEDA-3 status at the mean follow-up time of 22.6 ± 11.5 months. During the first year after cladribine initiation, disease activity prevailed more in patients with ≥2 prior DMTs and those switching from fingolimod, although both trends were not statistically significant. In terms of safety, 67.9% reported at least one AE during follow-up, the most frequent being fatigue (64.9%) and skin-related problems (38.6%).
CONCLUSION
Overall, our research results confirm cladribine's safety and effectiveness among RMS patients in real-world conditions. After the re-baseline, we observed high rates of NEDA-3-retention, and no new safety signals were noted.
背景
克拉屈滨片是一种高效的免疫重建疗法，自 2017 年以来在欧洲被批准用于治疗复发型多发性硬化症（RMS）。目前，人们非常需要来自不同临床环境的关于克拉屈滨在多发性硬化症中的疗效和安全性的真实世界数据。
方法
在本报告中，我们回顾性评估了 2018 年 8 月至 2021 年 11 月在我们比利时研究所接受克拉屈滨治疗的 RMS 患者的结局。将具有三个有效性终点数据的患者纳入分析，更具体地说，是那些复发、MRI 观察和确认残疾恶化的患者，这些患者在 3 个月时重新进行了“无疾病活动证据”（NEDA-3）的基线评估。安全性终点包括淋巴细胞减少症、肝转氨酶和随访期间的不良事件（AE）。进行了描述性统计和时间事件分析，包括治疗前的亚组分析。
结果
本研究纳入了 84 名 RMS 患者（年龄 42[33-50]岁，64.3%为女性，诊断持续时间 6[2-11]年，基线 EDSS 2.5[1.5-3.6]），其中 14 名（16.7%）患者出现复发，而残疾进展和脑 MRI 活动分别发生在 8.5%（6/71）和 6.3%（5/79）的患者中。这导致在平均 22.6±11.5 个月的随访中，72.6%（n=69，标准误差 6%）保留了 NEDA-3 状态。在克拉屈滨治疗开始后的第一年，在先前接受过≥2 种 DMT 治疗的患者和从芬戈莫德转换而来的患者中，疾病活动更为常见，尽管这两种趋势均无统计学意义。在安全性方面，67.9%的患者在随访期间至少报告了一次 AE，最常见的是疲劳（64.9%）和皮肤相关问题（38.6%）。
结论
总的来说，我们的研究结果证实了克拉屈滨在真实环境中对 RMS 患者的安全性和有效性。重新基线后，我们观察到了较高的 NEDA-3 保留率，并且没有发现新的安全性信号。
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