在用免疫检查点抑制剂治疗期间治疗肌性恶化的eculizumab
Laura Fionda1,2, Elena Rossini3,4, Antonio Lauletta3,4
1Neuromuscular and Rare Disease Centre, Sant'Andrea Hospital, Via di Grottarossa 1035-1039, 00189, Rome, Italy. laura.fionda@uniroma1.it.
概括
在接受 pembrolizumab 治疗癌症的患者中,eculizumab 有效治疗了严重肌痛性肌痛症的恶化. 这种方法允许继续治疗癌症并改善MG症状,而无需停止药物治疗.
科学领域:
- 神经免疫学 神经免疫学
- 在瘤学瘤学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 像 pembrolizumab 这样的免疫检查点抑制剂 (ICI) 可以引发自身免疫性疾病,包括肌痛性硬化症 (MG).
- 在ICI治疗期间MG恶化是一个挑战,因为停止癌症治疗可能对瘤结果有害.
- 作为补充剂抑制剂的eculizumab为某些自身免疫性疾病提供了有针对性的治疗方法.
研究的目的:
- 评估eculizumab在治疗肌痛性硬化症 (MG) 恶化中的疗效和安全性,在接受 pembrolizumab 免疫检查点抑制剂 (ICI) 治疗的患者.
- 为了确定eculizumab是否可以在不需要停止治疗结直肠癌的pembrolizumab的情况下管理MG爆发.
主要方法:
- 一个单臂的前性病例研究,研究了一名76岁的男性,在接受 pembrolizumab治疗结直肠癌期间,他患有全局抗乙胆受体 (AChR) 阳性MG (MGFA 类 IVB).
- 该患者经历了一种严重的MG恶化,耐药于类固醇和静脉注射免疫球蛋白 (IVIg).
- 在继续服用 pembrolizumab 的同时启动了 eculizumab;在 18 周内使用标准化 MG 尺度 (MGFA,MG-ADL,QMG,MGC,MG-QOL-15) 评估了患者的结果.
主要成果:
- 患者在经过18周的eculizumab治疗后,在所有评估的MG尺度上表现出显著和渐进的改善,达到MGFAIIIB级.
- 关键的改善包括MG-ADL降低40%,MG复合降低36%,QMG得分降低约30%.
- 腹筋症状显著改善,MG-ADL和MG复合物减少了70%左右,QMG减少了40%. 埃奎利祖马布耐受性很好,癌症的进展得到了很好的控制.
结论:
- 埃奎利祖马布 (eculizumab) 是一种潜在的基于机制的治疗方法,用于治疗抗编程细胞死亡蛋白1 (PD-1) 药物 (如 pembrolizumab) 的患者的MG恶化.
- 这种治疗策略允许继续关键的ICI治疗,避免对癌症治疗造成不利的中断.
- 在更大的案例系列中进行进一步的调查是有必要的,以验证这些发现.
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