Danish Multiple Sclerosis Center, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark; The Danish Multiple Sclerosis Registry, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
Danish Multiple Sclerosis Center, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
Mult Scler Relat Disord. 2023 Jun;74:104713. doi: 10.1016/j.msard.2023.104713. Epub 2023 Apr 9.
Natalizumab is a widely used high-efficacy treatment in multiple sclerosis (MS). Real-world evidence regarding long-term effectiveness and safety is warranted. We performed a nationwide study evaluating prescription patterns, effectiveness, and adverse events.
A nationwide cohort study using the Danish MS Registry. Patients initiating natalizumab between June 2006 and April 2020 were included. Patient characteristics, annualized relapse rates (ARRs), confirmed Expanded Disability Status Scale (EDSS) score worsening, MRI activity (new/enlarging T2- or gadolinium-enhancing lesions), and reported adverse events were evaluated. Further, prescription patterns and outcomes across different time periods ("epochs") were analysed.
In total, 2424 patients were enrolled, with a median follow-up time of 2.7 years (interquartile range (IQR) 1.2-5.1). In recent epochs, patients were younger, had lower EDSS scores, had fewer pre-treatment relapses and were more often treatment naïve. At 13 years of follow-up, 36% had a confirmed EDSS worsening. On-treatment ARR was 0.30, corresponding to a 72% reduction from pre-initiation. MRI activity was rare, 6.8% had activity within 2-14 months from treatment start, 3.4% within 14-26 months, and 2.7% within 26-38 months. Approximately 14% of patients reported adverse events, with cephalalgia constituting the majority. During the study, 62.3% discontinued treatment. Of these, the main cause (41%) was due to JCV antibodies, while discontinuations due to disease activity (9%) or adverse events (9%) were less frequent.
Natalizumab is increasingly used earlier in the disease course. Most patients treated with natalizumab are clinically stable with few adverse events. JCV antibodies constitute the main cause for discontinuation.
那他珠单抗是一种广泛应用于多发性硬化症（MS）的高效治疗药物。需要有真实世界的证据来证明其长期有效性和安全性。我们进行了一项全国性研究，评估其处方模式、疗效和不良事件。
这是一项使用丹麦多发性硬化症登记处的全国性队列研究。纳入 2006 年 6 月至 2020 年 4 月期间开始使用那他珠单抗的患者。评估患者特征、年化复发率（ARR）、确认的扩展残疾状况量表（EDSS）评分恶化、MRI 活动（新/扩大的 T2 或钆增强病变）和报告的不良事件。此外，还分析了不同时期（“时期”）的处方模式和结果。
共纳入 2424 例患者，中位随访时间为 2.7 年（四分位距 1.2-5.1）。在最近的时期，患者更年轻，EDSS 评分更低，治疗前复发次数更少，且更多为初治患者。在 13 年的随访中，36%的患者确认 EDSS 恶化。治疗期间的 ARR 为 0.30，与治疗前相比降低了 72%。MRI 活动很少见，6.8%的患者在治疗开始后 2-14 个月内有活动，3.4%在 14-26 个月内，2.7%在 26-38 个月内。约 14%的患者报告了不良事件，其中头痛占大多数。在研究期间，62.3%的患者停止了治疗。其中，主要原因（41%）是由于 JCV 抗体，而由于疾病活动（9%）或不良事件（9%）而停药的情况较少。
那他珠单抗在疾病早期的应用越来越多。大多数接受那他珠单抗治疗的患者临床稳定，不良事件较少。JCV 抗体是停药的主要原因。
