用于治疗慢性免疫血小板缺血症 (RAISE) 的埃尔特伦博巴格:为期6个月的随机化第三阶段研究
Gregory Cheng1, Mansoor N Saleh, Claus Marcher
1Chinese University of Hong Kong, Shatin, NT, Hong Kong SAR, China. gcheng@cuhk.edu.hk
Lancet (London, England)
|August 27, 2010
概括
与安慰剂相比,Eltrombopag显著增加了慢性免疫血栓塞缩症患者的应答率. 这种口服血栓形成素受体激动剂为管理这种疾病提供了有益的治疗选择.
科学领域:
- 血液学 血液学 血液学
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 慢性免疫性血小板缺血 (ITP) 是一种自身免疫性疾病,其特征是血小板数量较低.
- 埃尔特罗姆波帕格是一种口服的血栓形成素受体激动剂,用于治疗血栓细胞缩症.
研究的目的:
- 在6个月的时间内,比较每日ELTROMBOPAG与安慰剂在慢性ITP患者的疗效.
- 为了评估在接受eltrombopag的患者中实现治疗性血小板计数的几率.
主要方法:
- 第三阶段,双盲,安慰剂对照研究,涉及先前治疗慢性ITP的成年人.
- 随机分配 (2:1) 给eltrombopag (每天50毫克) 或安慰剂,结合标准护理.
- 主要终点:在6个月内治疗反应的几率 (血小板数量50,000-400,000/μL);治疗意图分析.
主要成果:
- 与安慰剂 (28%) 相比,eltrombopag组的响应率显著更高 (79%).
- 埃尔特罗姆波帕格的反应概率为8.2 (p<0.0001).
- 较少的eltrombopag患者需要救援治疗 (18%vs. 40%),并减少了更多的并发药物 (59%vs. 32%).
结论:
- 埃尔特朗博巴格在治疗慢性ITP方面是有效的,特别是在对脊髓切除术或先前治疗无反应的患者中.
- 必须权衡eltrombopag的潜在益处与相关风险,包括血栓栓塞事件和肝酶升高.
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