皮下efgartigimod PH20在一般化肌痛性肌痛症:一个第三阶段随机非劣势性研究 (ADAPT-SC) 和一个长期开放标签延伸研究 (ADAPT-SC+) 的中间分析
James F Howard1, Tuan Vu2, George Li3
1Department of Neurology, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
概括
皮下efgartigimod PH20在一般化肌痛性肌痛症 (gMG) 患者中显示出与静脉注射efgartigimod相比的非劣质性. 长期研究证实了其安全性,耐受性和在改善日常生活活动方面的一致有效性.
科学领域:
- 神经学 神经学
- 免疫学 免疫学 免疫学
背景情况:
- 一般化肌痛性肌痛症 (gMG) 是一种慢性自身免疫神经肌肉疾病.
- 目前对gMG的治疗有局限性,需要新的治疗方法.
研究的目的:
- 为了评估皮下 (SC) efgartigimod PH20与静脉注射 (IV) efgartigimod在gMG患者中的非劣势性.
- 通过一项开放式扩展研究 (ADAPT-SC+) 评估SC efgartigimod PH20的长期安全性,耐受性和疗效.
主要方法:
- ADAPT-SC研究:随机选择的参与者接受了SCefgartigimodPH20 (1000毫克) 或IVefgartigimod (10毫克/公斤) 在每周4次的管理.
- 主要终点:第四周总免疫球蛋白G (IgG) 与基线的百分比变化.
- 二级终点:评估MG-ADL和QMG响应率和平均得分变化.
主要成果:
- 达到了主终点,显示了SC和IVefgartigimod之间的总IgG降低的非劣势性.
- 早在第一周就观察到GMG症状的临床上有意义的改善,到第4周效果最大.
- ADAPT-SC+证明了持续的安全性,耐受性和MG-ADL得分的持续改善,随着持续的SC efgartigimod PH20治疗.
结论:
- 在治疗gMG时,SCefgartigimod PH20的效果与IVefgartigimod的效果无差.
- Efgartigimod PH20 SC为广泛的gMG患者群体提供了有利的长期安全性和有效性概况.
- 这些发现支持SC efgartigimod PH20作为一种可行的治疗方案,用于gMG管理.
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