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在临床试验中重新考虑复合终点:患者和试验者的见解

Joshua M Stolker¹, John A Spertus², David J Cohen²

¹From Saint Louis University, St. Louis, MO (J.M.S., K.K.J.); University of Missouri-Kansas City, Kansas City (J.A.S., D.J.C., E.B., B.B.L., P.S.C.); and Saint Luke's Mid America Heart and Vascular Institute; Kansas City, MO (D.J.C., P.G.J., P.S.C.). jstolker@yahoo.com.

|September 10, 2014

概括

患者和临床试验者对复合终点的评价不同. 患者优先考虑心肌梗塞和中风,而试验人员专注于死亡,表明复合终点可能不反映真实偏好.

科学领域:

心血管医学心血管医学
临床试验设计临床试验设计
患者报告的结果

背景情况:

在临床试验中经常使用复合终点来增加统计能力.
在复合终点中,个体成分的相对重要性可能在患者和研究人员之间有很大差异.

研究的目的:

研究和比较心血管患者和临床试验人员如何权衡复合终点的单个组件.
确定当前的复合终点权重是否准确地反映了患者和临床医生的优先事项.

主要方法:

对785名心血管患者和164名临床试验作者进行了调查,调查了5种不良事件 (死亡,心肌梗塞,中风,冠状动脉再血管化,心痛住院) 的感知重要性.
分配了"支出权重",并计算了与死亡相对的终点比率,以量化感知到的严重程度.
分析了患者和试验组之间的权重差异,并根据人口统计和临床因素进行调整.

主要成果:

与死亡 (权重5) 相比,患者对心肌梗塞 (比率1.12) 和中风 (比率1.08) 赋予了类似或更大的权重.
临床试验参与者优先考虑死亡 (权重8) 超过心肌梗塞 (比率0.63) 和中风 (比率0.53).
两组人都认为再血管化和住院比死亡的严重程度要少得多.

结论:

对于复合终点的单个组件,患者和试验者的偏好之间存在显著的差异.
当前的复合终点方法可能不能充分代表患者或试验者的值.
未来的试验设计应考虑纳入患者报告的结果措施,以更好地与患者优先级协调复合终点.

关键词:

心血管疾病心血管疾病临床试验临床试验临床试验临床试验临床试验终点确定终点的确定.患者的偏好,患者的偏好.以患者为中心的护理.统计统计统计统计统计

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