首都医科大学学报 ›› 2025, Vol. 46 ›› Issue (2): 191-196.doi: 10.3969/j.issn.1006-7795.2025.02.003

• 临床研究与方法学应用进展 • 上一篇    下一篇

胜出率法在脑血管病临床试验中的应用

田若冰1，胥芹1,2,3，周全1，田雪1,2,3，李欣亚1，郑曼琪1，王安心1,2,3*   

  1. 1.首都医科大学附属北京天坛医院北京市神经外科研究所流行病学研究室，北京 100070；2.首都医科大学附属北京天坛医院国家神经系统疾病临床医学研究中心，北京 100070；3.首都医科大学临床流行病学与临床试验学系，北京 100070
  • 收稿日期:2024-12-02 出版日期:2025-04-21 发布日期:2025-04-14
  • 通讯作者: 王安心 E-mail:wanganxin@bjtth.org
  • 基金资助:
    北京市自然科学基金-海淀原始创新联合基金项目（L222123）。

Application of the win ratio method to clinical trials in cerebrovascular disease

Tian Ruobing1, Xu Qin1,2,3, Zhou Quan1, Tian Xue1,2,3, Li Xinya1, Zheng Manqi1, Wang Anxin1,2,3*   

  1. 1.Department of Epidemiology, Beijing Neurosurgical Institute, Beijing Tiantan Hospital, Capital Medical University, Beijing 100070, China ；2.China National Clinical Research Center for Neurological Diseases, Beijing Tiantan Hospital, Capital Medical University, Beijing 100070, China; 3.Department of Clinical Epidemiology and Clinical Trial, Capital Medical University, Beijing 100070, China
  • Received:2024-12-02 Online:2025-04-21 Published:2025-04-14
  • Supported by:
    This study was supported by  Natural Science Foundation of Beijing-Haidian Original Innovation Joint Fund Project（L222123）.

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摘要： 目的  介绍胜出率法的概念和原理，并结合脑血管病临床试验的案例进行分析。 方法  基于氯吡格雷联合阿司匹林用于急性非致残性脑血管事件高危人群-II研究，依据对预后影响的关键因素将结局事件依次定义为：① 90 d内死亡的时间；② 90 d内缺血性卒中复发的时间；③ 90 d内中重度出血的时间。以氯吡格雷联合阿司匹林为参照组，采用胜出率法分析替格瑞洛联合阿司匹林的胜出率比值(winning ratio, Rw)，Bootstrap法估计Rw的95%置信区间(confidence interval, CI)，并与竞争风险模型计算的风险比(hazard ratio, HR)进行比较。结果  当仅考虑死亡事件时，胜出率法提示替格瑞洛组显著优于氯吡格雷组，Rw值为2.00 (95% CI: 1.52~2.47)，在逐步纳入缺血性卒中复发和中重度出血后，胜出率法计算所得的Rw值为1.29 (95% CI: 1.25~1.57)，Fine-Gray竞争风险回归模型所得HR值为0.78 (95% CI: 0.65~0.95)，两种方法均表明替格瑞洛组疗效优于氯吡格雷组。结论  胜出率法在分析具有复合终点的临床试验时，可以对多种结局变量划分优先顺序后进行分析，显示胜出率法的优势及其在脑血管病研究中的应用前景。

关键词: 胜出率, 脑血管病, Cox比例风险回归, 复合终点数据, 临床试验

Abstract: Objective  To introduce the concepts and principles of the win ratio method and to analyze it in the context of a case study of a clinical trial in cerebrovascular disease. Methods  Based on the study of clopidogrel with aspirin in high risk patients with acute non disabling cerebrovascular events 2, and key prognostic factors, the outcome events were defined sequentially as ① time to death within 90 d, ② time to recurrence of ischemic stroke within 90 d, ③ time to moderate-to-severe hemorrhage within 90 d. Using clopidogrel combined with aspirin as the reference group, the winning ratio (Rw) of ticagrelor combined with aspirin was analyzed by the win ratio method, and the 95% confidence interval (CI) of Rw was estimated by the Bootstrap method and compared with the hazard ratio (HR) calculated by the competing risk model. Results  When only fatal events were considered, the win ratio method suggested that the ticagrelor group was significantly better than the clopidogrel group, Rw=2.00 (95%CI:1.52-2.47), and after stepwise inclusion of  ischemic stroke and moderate-to-severe hemorrhage recurrence, the win ratio method yielded a value of 1.29(95% CI:1.25-1.57), and the HR value from Fine and Gray competing risk regression was 0.78 (95% CI:0.65-0.95), both of which indicated that the efficacy of the ticagrelor group was superior to that of the clopidogrel group. Conclusion  The win ratio method can be used to analyze clinical trials with composite endpoints after prioritizing multiple outcome variables, showing the advantages of win ratio and its promising application in cerebrovascular disease research.

Key words: win ratio, cerebrovascular disease, cox proportional risk regression, composite end point data, clinical trial

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