Daniels K, van der Nat P B, Frequin S T F M, van der Wees P J, Biesma D H, Hoogervorst E L J, van de Garde E M W
Department of Value-Based Healthcare, St. Antonius Hospital, Utrecht/Nieuwegein, Netherlands.
Radboud University Medical Center, Radboud Institute for Health Sciences, Scientific Center for Quality of Healthcare (IQ healthcare), Netherlands.
Mult Scler Int. 2020 Jun 15;2020:5463451. doi: 10.1155/2020/5463451. eCollection 2020.
Recently, ocrelizumab (Ocrevus®) was approved for the treatment of primary progressive multiple sclerosis (PPMS) based on data from the ORATORIO clinical trial. Real-world data about the clinical effectiveness of ocrelizumab has yet to be gathered.
The aim of this study was to provide data about the clinical effectiveness of ocrelizumab for patients diagnosed with PPMS in a real-world setting.
We conducted a retrospective cohort study of all patients with PPMS who started ocrelizumab treatment ( = 21) in St. Antonius Hospital (Utrecht/Nieuwegein, the Netherlands) between April 2018 and December 31, 2018. Primary outcome was pre- versus post-ocrelizumab disability worsening rate (from 96 weeks prior to first ocrelizumab administration up to 24 weeks post first ocrelizumab administration).
Disability worsening rate while on treatment significantly differed (lower) from disability worsening rate in pre-treatment period ( = -2.81, ≤ .01). Three out of 17 patients showed a clinically relevant improvement in disability status after treatment start.
Ocrelizumab can stabilize disability progression in patients with PPMS. Some patients even showed a clinically relevant improvement in disability status. Further research should help to identify which patients benefit most from ocrelizumab.
最近，基于ORATORIO临床试验的数据，奥瑞珠单抗（奥瑞珠单抗）被批准用于治疗原发性进行性多发性硬化症（PPMS）。关于奥瑞珠单抗临床疗效的真实世界数据尚未收集。
本研究的目的是提供在真实世界中确诊为PPMS的患者使用奥瑞珠单抗的临床疗效数据。
我们对2018年4月至2018年12月31日期间在荷兰乌得勒支/新韦根圣安东尼医院开始使用奥瑞珠单抗治疗（n = 21）的所有PPMS患者进行了一项回顾性队列研究。主要结局是奥瑞珠单抗治疗前后的残疾恶化率（从首次使用奥瑞珠单抗前96周直至首次使用奥瑞珠单抗后24周）。
治疗期间的残疾恶化率与治疗前期的残疾恶化率有显著差异（更低）（t = -2.81，P≤.01）。17例患者中有3例在开始治疗后残疾状况有临床相关改善。
奥瑞珠单抗可稳定PPMS患者残疾进展。部分患者甚至在残疾状况上有临床相关改善。进一步研究应有助于确定哪些患者从奥瑞珠单抗中获益最大。
