遵循SPIRIT和CONSORT标准对随机对照试验中使用替代终点的扩展指南解读

doi:10.12114/j.issn.1007-9572.2025.0082

中国全科医学 ›› 2026, Vol. 29 ›› Issue (15): 2107-2112.DOI: 10.12114/j.issn.1007-9572.2025.0082

• 科研方法·工具 • 上一篇

遵循SPIRIT和CONSORT标准对随机对照试验中使用替代终点的扩展指南解读

周慧¹, 唐欲博¹, 陈杰¹, 唐可京¹^,², 元刚³^,^*(), 姚弥⁴^,⁵^,^*()

1．510080　广东省广州市，中山大学附属第一医院药学部
2．510080　广东省广州市，中山大学附属第一医院呼吸与危重症
3．510080　广东省广州市，中山大学附属第一医院老年医学科
4．100034　北京市，北京大学第一医院全科医学科
5．100191　北京市，北京大学医学部全科医学学系

收稿日期:2025-01-10 修回日期:2025-05-15 出版日期:2026-05-20 发布日期:2026-04-14
通讯作者: 元刚, 姚弥
作者贡献：
周慧负责论文的撰写和修订，并对文章负责；唐欲博负责指导文章的结构；陈杰负责提出指导意见；唐可京负责文章的审核；元刚和姚弥负责提出文章思路、指导文章撰写、论文修订，并确认了论文最终版，对文章负责。

Interpretation for the Reporting of Surrogate Endpoints in Randomized Controlled Trial Protocols: Based on SPIRIT and CONSORT Extension Checklist

ZHOU Hui¹, TANG Yubo¹, CHEN Jie¹, TANG Kejing¹^,², YUAN Gang³^,^*(), YAO Mi⁴^,⁵^,^*()

1. Department of Pharmacy, the First Affiliated Hospital of Sun Yat-sen University, Guangzhou 510080, China
2. Division of Pulmonary and Critical Care Medicine, the First Affiliated Hospital of Sun Yat-sen University, Guangzhou 510080, China
3. Department of Geriatrics, the First Affiliated Hospital of Sun Yat-sen University, Guangzhou 510080, China
4. Department of General Practice, Peking University First Hospital, Beijing 100034, China
5. Department of General Practice, Peking University Health Science Center, Beijing 100191, China

Received:2025-01-10 Revised:2025-05-15 Published:2026-05-20 Online:2026-04-14
Contact: YUAN Gang, YAO Mi

摘要/Abstract

摘要： 随机对照试验正在越来越广泛地使用替代终点作为主要结局的评价指标，虽然其具有加快试验进程、减少样本量等诸多优势，但是，并非所有的替代终点都能很好地预测干预效果。因此，使用替代终点作为主要结局指标的规范性和后续验证问题受到人们的重视和思考。近期发布的《基于SPIRIT和CONSORT标准的替代终点扩展指南》对使用替代终点的随机对照试验制订了最低参照标准，旨在提高这些试验方案或报告的科学性及规范性。两个扩展指南的制订符合国际指南编写流程，这些条目在优化试验方案/报告质量的同时也有助于减少临床资源的浪费。本文将两个扩展指南一同对比解读，以期为广大研究者提供参考，引起相关从业者对替代终点及其背后诸多问题的关注。

关键词: 随机对照试验, 替代终点, 替代指标, 主要结局, 临床研究

Abstract:

Surrogate endpoints are frequently used to substitute for primary outcomes in randomized controlled trials to shorter trial duration, reduce sample size and etc. But some surrogate endpoints would increase uncertainty of the intervention's true effect and unreliable in revealing intervention harms. We do need better reporting of trials that rely on surrogate endpoints. The SPIRIT-Surrogate and CONSORT-Surrogate provides the minimum reporting requirements for trials that use surrogate endpoints. Those extension checklists help to improve transparency, usefulness of trial findings, and ultimately reduce research waste. We interpreted these two guidelines with the aim of providing reference for practitioners and drawing their attention to surrogate endpoints.

Key words: Randomized controlled trial, Surrogate endpoints, Surrogate indicators, Primary outcomes, Clinical study

中图分类号:

R 311

周慧,唐欲博,陈杰,等. 遵循SPIRIT和CONSORT标准对随机对照试验中使用替代终点的扩展指南解读[J]. 中国全科医学, 2026, 29(15): 2107-2112. DOI: 10.12114/j.issn.1007-9572.2025.0082.
ZHOU Hui,TANG Yubo,CHEN Jie, et al. Interpretation for the Reporting of Surrogate Endpoints in Randomized Controlled Trial Protocols: Based on SPIRIT and CONSORT Extension Checklist[J]. Chinese General Practice, 2026, 29(15): 2107-2112. DOI: 10.12114/j.issn.1007-9572.2025.0082.

图/表 1

参考文献 39

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SPIRIT标准或CONSORT标准的所属分类	替代终点扩展条目	条目所属的替代终点扩展指南
摘要和试验概述介绍、背景或目的	1.说明主要结局指标是一个替代终点；及干预措施对应的目标结局中哪项效果的评估是被替代的	SPIRIT标准和CONSORT标准的替代终点扩展指南
结局	2.说明使用替代终点作为主要结局的实际理由或科学依据	SPIRIT标准和CONSORT标准的替代终点扩展指南
	3.描述其他被考虑的替代终点以及选择当前替代终点的原因	SPIRIT替代终点扩展指南
	4.描述所选替代终点的理由：a替代终点的充分性证据（或证据不足）；b有证据（或证据不足）表明有效性与所使用的环境（如干预、疾病、人群）有关	SPIRIT标准和CONSORT标准的替代终点扩展指南
样本量	5.明确样本量是否经过验证，能够在使用该替代终点的最小效应情况下预测目标结局获益	SPIRIT标准和CONSORT标准的替代终点扩展指南
伦理、患者及公众的安排	6.说明是否以及如何通知受试者将被入组，并在（入组）注册前告知该试验旨在使用替代终点评估干预效果	SPIRIT标准和CONSORT标准的替代终点扩展指南
结果/结局或评估	7.如果主要结局是一个包含替代终点的复合终点，则需要报告干预措施对所有事件（指标）的影响	CONSORT标准替代终点扩展指南
危害和讨论	8.需要评价使用替代终点的情况下，该试验设计（包括样本量和随访期）是否充分捕捉到了目前干预措施的潜在危害	SPIRIT标准和CONSORT标准的替代终点扩展指南
统计方法和讨论	9.说明计划进行哪些后续分析/研究以验证当前关于目标结局的发现	SPIRIT标准和CONSORT标准的替代终点扩展指南
讨论和解释	10.需要在使用替代性主要结局的背景下对试验结果进行解释，包括干预措施的效果对目标结果已知的有效性评价，和被评估的干预措施对受试者潜在的风险获益	CONSORT标准替代终点扩展指南
传播和数据获取	11.如果试验中将收集替代终点和目标结果的数据，请说明数据公开访问的安排，以便未来进行二次研究	SPIRIT标准和CONSORT标准的替代终点扩展指南

SPIRIT标准或CONSORT标准的所属分类	替代终点扩展条目	条目所属的替代终点扩展指南
摘要和试验概述介绍、背景或目的	1.说明主要结局指标是一个替代终点；及干预措施对应的目标结局中哪项效果的评估是被替代的	SPIRIT标准和CONSORT标准的替代终点扩展指南
结局	2.说明使用替代终点作为主要结局的实际理由或科学依据	SPIRIT标准和CONSORT标准的替代终点扩展指南
	3.描述其他被考虑的替代终点以及选择当前替代终点的原因	SPIRIT替代终点扩展指南
	4.描述所选替代终点的理由：a替代终点的充分性证据（或证据不足）；b有证据（或证据不足）表明有效性与所使用的环境（如干预、疾病、人群）有关	SPIRIT标准和CONSORT标准的替代终点扩展指南
样本量	5.明确样本量是否经过验证，能够在使用该替代终点的最小效应情况下预测目标结局获益	SPIRIT标准和CONSORT标准的替代终点扩展指南
伦理、患者及公众的安排	6.说明是否以及如何通知受试者将被入组，并在（入组）注册前告知该试验旨在使用替代终点评估干预效果	SPIRIT标准和CONSORT标准的替代终点扩展指南
结果/结局或评估	7.如果主要结局是一个包含替代终点的复合终点，则需要报告干预措施对所有事件（指标）的影响	CONSORT标准替代终点扩展指南
危害和讨论	8.需要评价使用替代终点的情况下，该试验设计（包括样本量和随访期）是否充分捕捉到了目前干预措施的潜在危害	SPIRIT标准和CONSORT标准的替代终点扩展指南
统计方法和讨论	9.说明计划进行哪些后续分析/研究以验证当前关于目标结局的发现	SPIRIT标准和CONSORT标准的替代终点扩展指南
讨论和解释	10.需要在使用替代性主要结局的背景下对试验结果进行解释，包括干预措施的效果对目标结果已知的有效性评价，和被评估的干预措施对受试者潜在的风险获益	CONSORT标准替代终点扩展指南
传播和数据获取	11.如果试验中将收集替代终点和目标结果的数据，请说明数据公开访问的安排，以便未来进行二次研究	SPIRIT标准和CONSORT标准的替代终点扩展指南

遵循SPIRIT和CONSORT标准对随机对照试验中使用替代终点的扩展指南解读

Interpretation for the Reporting of Surrogate Endpoints in Randomized Controlled Trial Protocols: Based on SPIRIT and CONSORT Extension Checklist

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