Registry-Based Randomized Clinical Trials

doi:10.3969/j.issn.1673-5765.2022.10.018

Abstract

Abstract: A registry-based randomized controlled trial (RCT) is a clinical trial utilizing the existing high-quality registry platform for case records, data collection, randomized utilization, and follow-up. This article introduced the basic concept, historical origin, design ideas of registry-based RCT, comparison with traditional RCTs, applicable scenarios, cases analysis, statistical methods, report standards and so on.

Key words: Registry; Randomized controlled trial; Clinical study; Real-world data

摘要： 基于登记/注册研究的随机对照试验是一种利用现有的高质量登记/注册平台进行病例记录、数据收集、随机化应用以及随访的临床试验。本文简要介绍基于登记/注册研究的随机对照试验的基本概念、历史渊源、设计思路与传统随机对照试验的比较、适用场景、案例解读、统计方法及报告规范等内容，以期帮助临床研究者认识了解、掌握此类研究设计。

关键词: 登记; 随机对照试验; 临床研究; 真实世界数据

ZHOU Qi, YANG Kaixuan, JIANG Yingyu, GU Hongqiu. Registry-Based Randomized Clinical Trials[J]. Chinese Journal of Stroke, 2022, 17(10): 1145-1151.

周齐, 杨凯璇, 姜英玉, 谷鸿秋. 基于登记/注册研究的随机对照试验[J]. 中国卒中杂志, 2022, 17(10): 1145-1151.

References

[1] LI G W，SAJOBI T T，MENON B K，et al. Registry-based randomized controlled trials-what are the advantages，challenges，and areas for future research?[J/OL]. J Clin Epidemiol，2016，80：16-24[2022-08-09]. https://doi.org/10.1016/j.jclinepi.2016.08.003.
[2] GLIKLICH R E，LEAVY M B，DREYER N A. Registries for evaluating patient outcomes：a user's guide[M]. Rockville（MD）：Agency for Healthcare Research and Quality，2020.
[3] 杨星月，刘佳，何丽云，等. 病例注册登记研究的应用进展[J]. 中国循证医学杂志，2016，16（12）：4.
[4] JERNBERG T，ATTEBRING M F，HAMBRAEUS K，et al. The Swedish web-system for enhancement and development of evidence-based care in heart disease evaluated according to recommended therapies（SWEDEHEART）[J]. Heart，2010，96（20）：1617-1621.
[5] FROBERT O，LAGERQVIST B，GUDNASON T，et al. Thrombus aspiration in ST-elevation myocardial infarction in Scandinavia（TASTE trial）. A multicenter，prospective，randomized，controlled clinical registry trial based on the Swedish angiography and angioplasty registry（SCAAR）platform. Study design and rationale[J]. Am Heart J，2010，160（6）：1042-1048.
[6] FRÖBERT O，LAGERQVIST B，OLIVECRONA G K，et al. Thrombus aspiration during ST-segment elevation myocardial infarction[J]. N Engl J Med，2013，369（17）：1587-1597.
[7] JAMES S，RAO S V，GRANGER C B. Registry-based randomized clinical trials-a new clinical trial paradigm[J]. Nat Rev Cardiol，2015，12（5）：312-316.
[8] BROWN P，BRUNNHUBER K，CHALKIDOU K，et al. How to formulate research recommendations[J]. BMJ，2006，333（7572）：804-806.
[9] AMIR-BEHGHADAMI M，JANATI A. Population，intervention，comparison，outcomes and study（PICOS）design as a framework to formulate eligibility criteria in systematic reviews[J]. Emerg Med J，2020，37（6）：387.
[10] JAMES S，FRÖBERT O，LAGERQVIST B. Cardiovascular registries：a novel platform for randomised clinical trials[J]. Heart，2012，98（18）：1329.
[11] NYBERG K，HEDMAN P. Swedish guidelines for registry-based randomized clinical trials[J]. Ups J Med Sci，2019，124（1）：33-36.
[12] European Medicines Agency. Guideline on registry-based studies[EB/OL].（2021-10-26）[2022-08-09]. https://www.ema.europa.eu/en/guideline-registry-based-studies-0.
[13] LI Z X，WANG C J，ZHAO X Q，et al. Substantial progress yet significant opportunity for improvement in stroke care in China[J]. Stroke，2016，47（11）：2843-2849.
[14] WANG Y L，LI Z X，ZHAO X Q，et al. Effect of a multifaceted quality improvement intervention on hospital personnel adherence to performance measures in patients with acute ischemic stroke in China：a randomized clinical trial[J]. JAMA，2018，320（3）：245-254.
[15] WANG Y L，LI Z X，XIAN Y，et al. Rationale and design of a cluster-randomized multifaceted intervention trial to improve stroke care quality in China：the GOLDEN BRIDGE-acute ischemic stroke[J/OL]. Am Heart J，2015，169（6）：767-774，e2[2022-08-09]. https://doi.org/10.1016/j.ahj.2015.03.008.
[16] VELENTGAS P，DREYER N A，NOURJAH P，et al. Developing a protocol for observational comparative effectiveness research：a user's guide[M]. Rockville（MD）：Agency for Healthcare Research and Quality（US），2013.
[17] 高培，王杨，罗剑锋，等. 基于真实世界数据评价治疗结局研究的统计分析技术规范[J]. 中国循证医学杂志，2019，19（7）：787-793.
[18] ELDRIDGE S M，CHAN C L，CAMPBELL M J，et al. CONSORT 2010 statement：extension to randomised pilot and feasibility trials[J/OL]. Pilot Feasibility Stud，2016，2：64[2022-08-09]. https://doi.org/10.1186/s40814-016-0105-8.
[19] KWAKKENBOS L，JUSZCZAK E，HEMKENS L G，et al. Protocol for the development of a CONSORT extension for RCTs using cohorts and routinely collected health data[J/OL]. Res Integr Peer Rev，2018，3：9[2022-08-09]. https://doi.org/10.1186/s41073-018-0053-3.
[20] KWAKKENBOS L，IMRAN M，MCCALL S J，et al. CONSORT extension for the reporting of randomised controlled trials conducted using cohorts and routinely collected data（CONSORT-ROUTINE）：checklist with explanation and elaboration[J/OL]. BMJ，2021，373：n857[2022-08-09]. https://doi.org/10.1136/bmj.n857.
[21] CALVERT M，BLAZEBY J，ALTMAN D G，et al. Reporting of patient-reported outcomes in randomized trials：the CONSORT PRO extension. [J]. JAMA，2013，309（8）：814-822.
[22] THORPE K E，ZWARENSTEIN M，OXMAN A D，et al. A pragmatic-explanatory continuum indicator summary（PRECIS）：a tool to help trial designers[J/OL]. CMAJ，2009，180（10）：E47-E57[2022-08-09]. https://doi.org/10.1503/cmaj.090523.