End Points and Clinical Trial Design in Pulmonary Arterial Hypertension
End Points and Clinical Trial Design in Pulmonary Arterial Hypertension
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DOI：
10.1016/j.jacc.2009.04.007
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发表时间：
2009-06-30
影响因子：
24
通讯作者：
Seeger, Werner
中科院分区：
文献类型：
--
作者：
McLaughlin, Vallerie V.;Badesch, David B.;Seeger, Werner
新的和新兴的治疗方法可能为肺动脉高压患者提供益处。将在随机临床试验中与现有的联合疗法比较其疗效和安全性。需要确定这些试验的适当终点：包括运动试验、至临床恶化时间的复合终点和血流动力学标志物，包括先进的成像模式和生物标志物。生活质量问卷是有用的和重要的次要终点;肺动脉高压特异性问卷目前正在开发中。各种试验设计的优点和缺点，包括安慰剂对照的单药治疗或添加试验，非劣效性研究和退出试验也进行了讨论。（J Am科尔心脏病学杂志2009;54：S97-107）（C）2009年美国心脏病学会基金会
New and emerging therapies might provide benefit in patients with pulmonary arterial hypertension. Their efficacy and safety will be compared with existing combination therapies in randomized clinical trials. Appropriate end points for these trials need to be identified: these will include exercise testing, the composite end point of time to clinical worsening, and hemodynamic markers, including advanced imaging modalities and biomarkers. Quality-of-life questionnaires are useful and important secondary end points; pulmonary arterial hypertension-specific questionnaires are currently being developed. Advantages and disadvantages of various trial designs, including placebo-controlled monotherapy or add-on trials, noninferiority studies, and withdrawal trials are also discussed. (J Am Coll Cardiol 2009;54:S97-107) (C) 2009 by the American College of Cardiology Foundation