Canadian VIGOUR Centre, University of Alberta, Edmonton, Alberta, Canada.
Department of Medicine, University of Alberta, Edmonton, Alberta, Canada.
JAMA Cardiol. 2019 Nov 1;4(11):1122-1128. doi: 10.1001/jamacardio.2019.3604.
Pragmatic trials test interventions using designs that produce results that may be more applicable to the population in which the intervention will be eventually applied.
To investigate how pragmatic or explanatory cardiovascular (CV) randomized clinical trials (RCT) are, and if this has changed over time.
Six major medical and CV journals, including New England Journal of Medicine, Lancet, JAMA, Circulation, European Heart Journal, and Journal of the American College of Cardiology.
All CV-related RCTs published during 2000, 2005, 2010, and 2015 were identified and included.
Included RCTs were assessed by 2 independent adjudicators with expertise in RCT and CV medicine.
The outcome measure was the level of pragmatism evaluated using the Pragmatic Explanatory Continuum Index Summary (PRECIS)-2 tool, which uses a 5-point ordinal scale (ranging from very pragmatic to very explanatory) across 9 domains of trial design, including eligibility, recruitment, setting, organization, intervention delivery, intervention adherence, follow-up, primary outcome, and analysis.
Of 616 RCTs, the mean (SD) PRECIS-2 score was 3.26 (0.70). The level of pragmatism increased over time from a mean (SD) score of 3.07 (0.74) in 2000 to 3.46 (0.67) in 2015 (P < .001 for trend; Cohen d relative effect size, 0.56). The increase occurred mainly in the domains of eligibility, setting, intervention delivery, and primary end point. PRECIS-2 score was higher for neutral trials than those with positive results (P < .001) and in phase III/IV trials compared with phase I/II trials (P < .001) but similar between different sources of funding (public, industry, or both; P = .38). More pragmatic trials had more sites, larger sample sizes, longer follow-ups, and mortality as the primary end point.
The level of pragmatism increased moderately over 2 decades of CV trials. Understanding the domains of current and future clinical trials will aid in the design and delivery of CV trials with broader application.
实用临床试验采用可能更适用于干预措施最终应用人群的设计来检验干预措施。
研究心血管（CV）随机临床试验（RCT）的实用性或解释性如何，以及这种情况是否随时间而变化。
包括《新英格兰医学杂志》、《柳叶刀》、《美国医学会杂志》、《循环》、《欧洲心脏杂志》和《美国心脏病学会杂志》在内的 6 大医学和 CV 期刊。
确定并纳入了 2000 年、2005 年、2010 年和 2015 年发表的所有与 CV 相关的 RCT。
由 2 名具有 RCT 和 CV 医学专业知识的独立裁判进行评估。
使用实用解释连续体指数综合（PRECIS）-2 工具评估实用性水平，该工具在 9 个试验设计领域使用 5 点序数量表（范围从非常实用到非常解释性），包括资格、招募、设置、组织、干预措施的提供、干预措施的依从性、随访、主要结局和分析。
在 616 项 RCT 中，PRECIS-2 评分的平均值（标准差）为 3.26（0.70）。实用性水平随时间推移而增加，从 2000 年的平均（标准差）评分 3.07（0.74）增加到 2015 年的 3.46（0.67）（趋势 P<.001；Cohen d 相对效应大小为 0.56）。增加主要发生在资格、设置、干预措施的提供和主要终点领域。中性试验的 PRECIS-2 评分高于阳性结果试验（P<.001），III/IV 期试验高于 I/II 期试验（P<.001），但不同资金来源（公共、工业或两者兼有）之间的评分无差异（P=.38）。更实用的试验具有更多的试验地点、更大的样本量、更长的随访时间和死亡率作为主要终点。
在 20 年的 CV 试验中，实用性水平适度提高。了解当前和未来临床试验的领域将有助于设计和提供更广泛应用的 CV 试验。
