Vu Tuan H, Mantegazza Renato, Annane Djillali, Katsuno Masahisa, Meisel Andreas, Nicolle Michael W, Bril Vera, Aguzzi Rasha, Frick Glen, Howard James F
University of South Florida Morsani College of Medicine, Tampa, Florida, USA.
Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Eur J Neurol. 2025 Apr;32(4):e70158. doi: 10.1111/ene.70158.
Ravulizumab, an anti-complement C5 monoclonal antibody, was efficacious with acceptable safety in the randomized controlled period (RCP) and interim open-label extension (OLE) periods of the CHAMPION MG phase 3 trial in adults with anti-acetylcholine receptor antibody-positive (AChR-Ab+) generalized myasthenia gravis (gMG). Here, we report final results from the OLE.
Patients who completed the 26-week RCP could enter the OLE and receive ravulizumab for up to 4 years. Efficacy and safety were assessed throughout the OLE.
Among all ravulizumab-treated patients (n = 169; median [range] ravulizumab treatment, 759.0 [14.0, 1265.0] days), 161 entered the OLE (ravulizumab-ravulizumab: n = 78; placebo-ravulizumab: n = 83). Sustained improvements were observed in Myasthenia Gravis Activities of Daily Living (MG-ADL) total scores (ravulizumab-ravulizumab, least squares mean [95% CI] change from RCP baseline at week 164: -4.0 [-5.3, -2.8]; p < 0.0001; placebo-ravulizumab, change from OLE baseline after 138 weeks of treatment: -2.1 [-3.3, -0.9]; p = 0.0005). One hundred and forty-one out of 160 (88.1%) patients achieved a ≥ 2-point improvement in MG-ADL total score, and 59/141 (41.8%) achieved a score of 0 or 1; once achieved, 32/59 (54.2%) sustained this status for > 50% of their remaining time in the study. Similar improvements were observed in Quantitative Myasthenia Gravis and Myasthenia Gravis Quality of Life-15 revised scores, and Neurological Quality of Life Fatigue subscale scores. Clinical deterioration event rates were reduced in the OLE versus placebo in the RCP. Corticosteroid usage was reduced in the OLE. Ravulizumab was well tolerated; no meningococcal infections were reported.
Ravulizumab demonstrated clinically meaningful and durable efficacy and safety in adults with AChR-Ab+ gMG.
ravulizumab是一种抗补体C5单克隆抗体，在抗乙酰胆碱受体抗体阳性（AChR-Ab+）的成人全身性重症肌无力（gMG）的CHAMPION MG 3期试验的随机对照期（RCP）和中期开放标签扩展期（OLE）中疗效显著且安全性可接受。在此，我们报告OLE的最终结果。
完成26周RCP的患者可进入OLE，并接受ravulizumab治疗长达4年。在整个OLE期间评估疗效和安全性。
在所有接受ravulizumab治疗的患者中（n = 169；ravulizumab治疗的中位[范围]时间为759.0 [14.0, 1265.0]天），161例进入OLE（ravulizumab-ravulizumab组：n = 78；安慰剂-ravulizumab组：n = 83）。重症肌无力日常生活活动（MG-ADL）总分持续改善（ravulizumab-ravulizumab组，第164周时相对于RCP基线的最小二乘均值[95%CI]变化：-4.0 [-5.3, -2.8]；p < 0.0001；安慰剂-ravulizumab组，治疗138周后相对于OLE基线的变化：-2.1 [-3.3, -0.9]；p = 0.0005）。160例患者中有141例（88.1%）MG-ADL总分改善≥2分，59/141例（41.8%）达到0分或1分；一旦达到，32/59例（54.2%）在研究剩余时间的>50%内维持该状态。在定量重症肌无力和重症肌无力生活质量-15修订评分以及神经生活质量疲劳子量表评分方面也观察到类似的改善。与RCP中的安慰剂相比，OLE中的临床恶化事件发生率降低。OLE中皮质类固醇的使用减少。Ravulizumab耐受性良好；未报告脑膜炎球菌感染。
Ravulizumab在AChR-Ab+ gMG成人患者中显示出具有临床意义的持久疗效和安全性。
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