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治疗系统性红斑狼的巴里西替尼:双盲,随机,安慰剂对照的第二阶段试验
Daniel J Wallace1, Richard A Furie2, Yoshiya Tanaka3
1Division of Rheumatology, Cedars-Sinai Medical Center, University of California at Los Angeles, Los Angeles, CA, USA.
Lancet (London, England)
|July 26, 2018
概括
在第二阶段试验中,巴里西提尼布4mg显著改善了全身性红斑狼的症状. 这种Janus激酶抑制剂是狼患者的新口服疗法.
科学领域:
- 关节病学
- 免疫学
- 药理学
背景情况:
- 系统性红斑狼 (SLE) 存在重大未满足的医疗需求.
- 作为一种选择性的Janus激酶 (JAK) 1和JAK2抑制剂,巴里西替尼对SLE的潜在治疗益处进行了研究.
- 雅克抑制剂代表了自身免疫性疾病的新疗法.
研究的目的:
- 评估巴里西替尼在活跃的全身性红斑狼患者中的疗效和安全性.
- 确定巴里西替尼治疗SLE症状的最佳剂量,特别是关节炎和皮疹.
主要方法:
- 一个双盲,随机,安慰剂控制,多中心的第二阶段研究.
- 314名患有激活性SLE的患者被分配给每天一次的巴里西提尼布 (2 mg或4 mg) 或安慰剂,持续24周.
- 主要终点:根据系统性红斑性狼活动指数-2000 (SLEDAI- 2K) 的数据,关节炎或皮疹在第24周消失.
主要成果:
- 4毫克巴里西提尼可显著改善关节炎或皮疹的SLEDAI-2K分辨率 (67%与安慰剂相比,p=0. 0414).
- 2毫克巴里西替尼呈现改善趋势,但没有达到统计显著性 (58%与安慰剂相比,p=0. 39).
- 安全性与之前的巴里西提尼研究一致;与安慰剂相比,巴里西提尼组的严重不良事件和感染率更高.
结论:
- 4毫克巴里西提尼在治疗常规疗法控制不良的患者中显示出显著的SLE症状,特别是关节炎和皮疹.
- 巴里西提尼的安全性与之前的研究一致,支持其作为SLE口服治疗的潜力.
- 这项研究支持对巴里西提尼布在SLE治疗中的进一步研究.
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