van Pesch Vincent, Bartholomé Emmanuel, Bissay Véronique, Bouquiaux Olivier, Bureau Michel, Caekebeke Jo, Debruyne Jan, Declercq Inge, Decoo Dany, Denayer Pierre, De Smet Eric, D'hooghe Marie, Dubois Bénédicte, Dupuis Michel, Sankari Souraya El, Geens Karine, Guillaume Daniel, van Landegem William, Lysandropoulos Andreas, de Noordhout Alain Maertens, Medaer Robert, Melin Annick, Peeters Katelijne, Ba Rémy Phan, Retif Cécile, Seeldrayers Pierrette, Symons Anoek, Urbain Etienne, Vanderdonckt Patrick, Van Ingelghem Erwin, Vanopdenbosch Ludo, Vanroose Erwin, Van Wijmeersch Bart, Willekens Barbara, Willems Christiana, Sindic Christian
Neurology Department, Cliniques Universitaires St-Luc, Avenue Hippocrate 10, 1200, Woluwe-Saint-Lambert, Belgium,
Acta Neurol Belg. 2014 Sep;114(3):167-78. doi: 10.1007/s13760-014-0308-9. Epub 2014 Jun 11.
Natalizumab (Tysabri(®)) is highly efficacious in controlling disease activity in relapsing multiple sclerosis (MS) patients. As it is one of the more recent therapies for MS, there remains a need for long-term safety and efficacy data of natalizumab in a clinical practice setting. The Tysabri observational program (TOP) is an open-label, multicenter, multinational, prospective observational study, aiming to recruit up to 6,000 patients with relapsing-remitting MS from Europe, Canada and Australia. The objectives of this study are to collect long-term safety and efficacy data on disease activity and disability progression. We report here the interim results of the 563 patients included in TOP between December 2007 and 2012 from Belgium. This patient cohort was older at baseline, had longer disease duration, higher neurological impairment, and a higher baseline annualized relapse rate, when compared to patients included in the pivotal phase III AFFIRM trial. Nevertheless, the efficacy of natalizumab was comparable. The annualized relapse rate on treatment was reduced by 90.70 % (p < 0.0001) with a cumulative probability of relapse of 26.87 % at 24 months. The cumulative probabilities of sustained disability improvement and progression at 24 months were 25.68 and 9.01 %, respectively. There were no new safety concerns over the follow-up period. Two cases of progressive multifocal leukoencephalopathy were diagnosed. Our results are consistent with other observational studies in the post-marketing setting.
那他珠单抗（泰萨比里（®））在控制复发型多发性硬化症（MS）患者的疾病活动方面具有高度疗效。由于它是治疗MS的较新疗法之一，因此仍需要在临床实践环境中获取那他珠单抗的长期安全性和疗效数据。泰萨比里观察项目（TOP）是一项开放标签、多中心、跨国、前瞻性观察性研究，旨在从欧洲、加拿大和澳大利亚招募多达6000例复发缓解型MS患者。本研究的目的是收集有关疾病活动和残疾进展的长期安全性和疗效数据。我们在此报告2007年12月至2012年期间来自比利时纳入TOP的563例患者的中期结果。与关键的III期AFFIRM试验中纳入的患者相比，该患者队列在基线时年龄更大、病程更长、神经功能损害更严重且基线年化复发率更高。然而，那他珠单抗的疗效相当。治疗期间的年化复发率降低了90.70%（p<0.0001），24个月时的累积复发概率为26.87%。24个月时持续残疾改善和进展的累积概率分别为25.68%和9.01%。在随访期间没有新的安全问题。诊断出2例进行性多灶性白质脑病。我们的结果与上市后环境中的其他观察性研究一致。
