Butzkueven Helmut, Kappos Ludwig, Pellegrini Fabio, Trojano Maria, Wiendl Heinz, Patel Radhika N, Zhang Annie, Hotermans Christophe, Belachew Shibeshih
Department of Medicine, Melbourne Brain Centre at the Royal Melbourne Hospital, University of Melbourne, Melbourne, Victoria, Australia Department of Neurology, Box Hill Hospital, Monash University, Victoria, Victoria, Australia.
Departments of Neurology and Biomedicine, University Hospital Basel, Basel, Switzerland.
J Neurol Neurosurg Psychiatry. 2014 Nov;85(11):1190-7. doi: 10.1136/jnnp-2013-306936. Epub 2014 Feb 14.
Clinical trials established the efficacy and safety of natalizumab. Data are needed over longer periods of time and in the clinical practice setting.
To evaluate long-term safety of natalizumab and its impact on annualised relapse rate and Expanded Disability Status Scale (EDSS) progression in patients with relapsing-remitting multiple sclerosis (RRMS).
The Tysabri (natalizumab) Observational Program (TOP) is an open-label, multinational, 10-year prospective study in clinical practice settings.
In this 5-year interim analysis, 4821 patients were enrolled. Follow-up for at least 4 years from natalizumab commencement in 468 patients and at least 2 years in 2496 patients revealed no new safety signals. There were 18 cases of progressive multifocal leucoencephalopathy reported, following 11-44 natalizumab infusions. Mean annualised relapse rate decreased from 1.99 in the 12 months prior to baseline to 0.31 on natalizumab therapy (p<0.0001), remaining low at 5 years. Lower annualised relapse rates were observed in patients who used natalizumab as first MS therapy, in patients with lower baseline EDSS scores, and in patients with lower prenatalizumab relapse rates. Mean EDSS scores remained unchanged up to 5 years.
Interim TOP data confirm natalizumab's overall safety profile and the low relapse rate and stabilised disability levels in natalizumab-treated patients with RRMS in clinical practice.
NCT00493298.
临床试验已证实那他珠单抗的疗效和安全性。还需要更长时间以及临床实践环境中的数据。
评估那他珠单抗的长期安全性及其对复发缓解型多发性硬化症（RRMS）患者年化复发率和扩展残疾状态量表（EDSS）进展的影响。
那他珠单抗观察项目（TOP）是一项在临床实践环境中开展的开放标签、多国、为期10年的前瞻性研究。
在这项5年中期分析中，共纳入4821例患者。468例患者自开始使用那他珠单抗起至少随访4年，2496例患者至少随访2年，未发现新的安全信号。报告了18例进行性多灶性白质脑病病例，发生在接受11 - 44次那他珠单抗输注之后。年化复发率均值从基线前12个月的1.99降至那他珠单抗治疗时的0.31（p<0.0001），在5年时仍维持在较低水平。在将那他珠单抗作为首次MS治疗的患者、基线EDSS评分较低的患者以及那他珠单抗治疗前复发率较低的患者中观察到较低的年化复发率。EDSS评分均值在5年内保持不变。
TOP中期数据证实了那他珠单抗的总体安全性概况，以及在临床实践中接受那他珠单抗治疗的RRMS患者的低复发率和稳定的残疾水平。
NCT00493298。
