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纳塔利祖马布,芬戈利莫德和注射疗法在儿科发病多发性硬化症中的比较有效性:基于注册表的研究
Tim Spelman1, Gabrielle Simoneau1, Robert Hyde1
1From the MSBase Foundation (T.S.), Melbourne, Australia; Department of Clinical Neuroscience (T.S.), Karolinska Institute, Stockholm, Sweden; Biogen (G.S.), Toronto, Ontario, Canada; Biogen (R.H., Robert Kuhelj), Baar, Switzerland; Division of Neurology (R.A.), Department of Medicine, Amiri Hospital, Sharq, Kuwait; Dokuz Eylul University (S.O.), Konak/Izmir; Hacettepe University (Rana Karabudak), Ankara, Turkey; Nehme and Therese Tohme Multiple Sclerosis Center (B.I.Y., S.J.K.), American University of Beirut Medical Center, Lebanon; 19 Mayis University (M.T.), Samsun; KTU Medical Faculty Farabi Hospital (C.B.), Trabzon, Turkey; Department of Neurology and Center of Clinical Neuroscience (D.H., E.K.H.), First Faculty of Medicine, Charles University in Prague and General University Hospital, Czech Republic; Department of Neurology (B.W.-G.), Buffalo General Medical Center, Buffalo, NY; Department of Medical and Surgical Sciences and Advanced Technologies (F.P.), GF Ingrassia, Catania, Italy; Department of Neurology (A.A.), School of Medicine and Koc University Research Center for Translational Medicine (KUTTAM), Koc University, Istanbul, Turkey; Department of Neurology and Clinical Investigation Center Neurosciences and Mental Health (S.M.), Razi University Hospital; Department of Neurology (R.G.), Razi University Hospital, Tunis, Tunisia; Rashid Hospital (J.I.), Dubai, United Arab Emirates; Isfahan University of Medical Sciences (V.S.), Iran; Department of Neurology (S.E.), Hospital Universitario Virgen Macarena, Sevilla, Spain; Ashfield MedComms (W.L.W.), Middletown, CT; Department of Neuroscience (H.B.), Central Clinical School, Monash University, Melbourne; and Department of Neurology (H.B.), Box Hill Hospital, Monash University, Box Hill, Victoria, Australia.
纳塔利祖马布在减少儿科多发性硬化症 (MS) 患者的复发方面比fingolimod更有效. 这两种治疗方法都优于旧的注射疗法,为患有多发性硬化症的儿童提供更好的疾病控制.
科学领域:
- 神经学 神经学
- 免疫学 免疫学 免疫学
- 儿科 儿科 儿科
背景情况:
- 儿科发病多发性硬化症 (POMS) 呈现出高度炎症和频繁复发.
- 患有POMS的儿童经历了更好的复发恢复,但比成年人更早地达到不可逆转的残疾.
- 对于POMS存在有限的随机试验;大多数数据来自成人批准疗法的观察性研究.
研究的目的:
- 为了比较纳塔利祖马布和芬戈利莫德在小儿多发性硬化症患者的有效性.
- 为了评估治疗疗效与注射疾病修饰疗法 (DMT) 相比,作为参考.
- 分析不同DMT治疗的POMS患者的复发风险和残疾进展.
主要方法:
- 来自MSBase注册表的1,218名POMS患者的回顾性研究 (2006-2021年).
- 包括开始服用纳塔利祖马布,芬戈利莫德或注射DMT的患者.
- 逆概率治疗权重 (IPTW) 用于患者匹配和结果分析,包括时间到第一次复发,复发率和残疾进展.
主要成果:
- 与可注射的DMT相比,芬戈利莫德的复发风险明显较低 (HR 0.49,p=0.008).
- 纳塔利祖马布的复发风险明显低于可注射的DMT (HR 0.15,p<0.001) 和fingolimod (HR 0.37,p=0.049).
- 二次结局结果和敏感性分析证实了这些发现,表明纳塔利祖马布在控制复发方面具有更高的疗效.
结论:
- 纳塔利祖马布在控制POMS患者复发方面比芬戈利莫德更有效.
- 纳塔利祖马布和芬戈利莫德都比POMS的第一线注射疗法更有效.
- 这些发现与先前在成人MS和POMS的研究一致,支持纳塔利祖马布在控制炎症活动中的作用.
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