Hupperts Raymond, Gasperini Claudio, Lycke Jan, Ziemssen Tjalf, Feys Peter, Xiao Shan, Acosta Carlos, Koster Thijs, Hobart Jeremy
Department of Neurology, Zuyderland Medical Center, 6130 MB Sittard, The Netherlands.
Department of Neurosciences, S. Camillo Forlanini Hospital, Rome, Italy.
Ther Adv Neurol Disord. 2022 May 18;15:17562864221090398. doi: 10.1177/17562864221090398. eCollection 2022.
MOBILE and ENHANCE were similarly designed randomized trials of walking-impaired adults with relapsing-remitting or progressive multiple sclerosis (MS) who received placebo or 10 mg prolonged-release (PR)-fampridine twice daily for 24 weeks. Both studies showed sustained and clinically meaningful improvement in broad measures of walking and balance over 24 weeks of PR-fampridine treatment.
To evaluate the functional benefits and safety of PR-fampridine placebo using a integrated efficacy analysis of MOBILE and ENHANCE data.
Data from the intention-to-treat (ITT) populations of MOBILE and ENHANCE studies were pooled in a analysis based on the following outcome measures: 12-item MS Walking Scale (MSWS-12), Timed Up and Go (TUG) speed, Berg Balance Scale (BBS), MS Impact Scale physical impact subscale (MSIS-29 PHYS), EQ-5D utility index score, visual analogue scale (VAS), and adverse events. The primary analysis was the proportion of people with MS (PwMS) with a mean improvement in MSWS-12 score (⩾8 points) from baseline over 24 weeks. A subgroup analysis based on baseline characteristics was performed.
In the ITT population ( = 765; PR-fampridine, = 383; placebo, = 382), a greater proportion of PR-fampridine-treated PwMS than placebo-treated PwMS achieved a clinically meaningful improvement in the MSWS-12 scale over 24 weeks (44.3% 33.0%; < 0.001). PR-fampridine MSWS-12 responders demonstrated greater improvements from baseline in TUG speed, BBS score, MSIS-29 PHYS score, and EQ-5D utility index and VAS scores PR-fampridine MSWS-12 nonresponders and placebo. Subgroup analyses based on baseline characteristics showed consistency in the effects of PR-fampridine.
The pooled analysis of MOBILE and ENHANCE confirms previous evidence that treatment with PR-fampridine results in clinically meaningful improvements in walking, mobility and balance, self-reported physical impact of MS, and quality of life and is effective across a broad range of PwMS.
MOBILE和ENHANCE是两项设计相似的随机试验，针对患有复发缓解型或进展型多发性硬化症（MS）且行走功能受损的成年人，他们接受安慰剂或每日两次10毫克缓释（PR）-氨吡啶治疗，为期24周。两项研究均显示，在接受PR-氨吡啶治疗的24周内，行走和平衡的广泛指标持续且具有临床意义地改善。
通过对MOBILE和ENHANCE数据进行综合疗效分析，评估PR-氨吡啶与安慰剂相比的功能益处和安全性。
将MOBILE和ENHANCE研究意向性治疗（ITT）人群的数据汇总在一项分析中，基于以下结局指标：12项MS行走量表（MSWS-12）、计时起立行走（TUG）速度、伯格平衡量表（BBS）、MS影响量表身体影响子量表（MSIS-29 PHYS）、EQ-5D效用指数评分、视觉模拟量表（VAS）以及不良事件。主要分析是MS患者（PwMS）中在24周内MSWS-12评分从基线平均改善（⩾8分）的比例。进行了基于基线特征的亚组分析。
在ITT人群（n = 765；PR-氨吡啶组，n = 383；安慰剂组，n = 382）中，与接受安慰剂治疗的PwMS相比，接受PR-氨吡啶治疗的PwMS中在24周内MSWS-12量表上实现临床意义改善的比例更高（44.3%对33.0%；P < 0.001）。PR-氨吡啶治疗的MSWS-12有反应者在TUG速度、BBS评分、MSIS-29 PHYS评分、EQ-5D效用指数和VAS评分方面与PR-氨吡啶治疗的MSWS-12无反应者及安慰剂相比，从基线有更大改善。基于基线特征的亚组分析显示PR-氨吡啶的效果具有一致性。
MOBILE和ENHANCE的汇总分析证实了先前的证据，即PR-氨吡啶治疗可在行走、活动能力和平衡、自我报告的MS身体影响以及生活质量方面带来具有临床意义的改善，并且对广泛的PwMS有效。
