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与安慰剂相比,兰德鲁马布对预防遗传性血管发作的作用:随机临床试验
Aleena Banerji1, Marc A Riedl2, Jonathan A Bernstein3
1Division of Rheumatology, Allergy, and Immunology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston.
JAMA
|November 28, 2018
概括
在3期试验中,兰德鲁马布显著降低了遗传性血管发作. 这种单克隆抗体为遗传性血管 (HAE) 患者提供了有前途的预防疗法.
科学领域:
- 免疫学
- 药理学
背景情况:
- 目前对遗传性血管 (HAE) 的长期预防治疗存在局限性.
- 这是一种罕见的遗传性疾病,其特征是经常出现胀.
研究的目的:
- 评估针对血卡利克林的单克隆抗体lanadelumab在预防HAE发作的有效性.
- 评估lanadelumab作为HAE的预防治疗的安全性和耐受性.
主要方法:
- 这是一项3期随机双盲,安慰剂对照试验,涉及125名HAE型I或II患者.
- 患者接受皮下治疗 (150毫克每4周,300毫克每4周,或300毫克每2周) 或安慰剂26周.
主要成果:
- 与安慰剂相比,lanadelumab 在所有测试方案中显著降低了每月HAE发作率 (P < 0. 001).
- 在lanadelumab组中最常见的不良反应是注射部位反应和头.
- 有90. 4%的患者完成了26周的研究.
结论:
- 在降低HAE发作频率方面,皮下使用的lanadelumab具有显著的有效性.
- 对于遗传性血管患者来说,lanadelumab 是一种潜在的新预防性治疗选择.
- 需要进一步的研究来确定长期的安全性和疗效.
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