Concordance between target trial emulation and randomised controlled trials: systematic review and meta-analysis.

Methods

Systematic reviewand meta-analysis.

系统评价和荟萃分析。

data_sources

Medline, EMBASE, Scopus, PsycINFO, and Web of Science (2010 to 2025).

检索了Medline、EMBASE、Scopus、PsycINFO和Web of Science数据库（2010年至2025年）。

study_selection

Observational studies that explicitly aimed to emulate target randomised controlled trials of a health intervention were included.

纳入了明确旨在模仿目标健康干预措施的随机对照试验的观察性研究。

data_extraction

Data were extracted on study domain, target population, intervention, comparator, and main outcomes for each emulation and corresponding randomised controlled trial.

从每个模拟研究和相应的随机对照试验中提取了研究领域、目标人群、干预措施、对照组和主要结果的数据。

Methods

For 21 predefined emulation design features, concordance was assessed using Pearson correlation coefficients, standardised difference agreement, and ratio of ratios, with subgroup and regression analyses of study characteristics and concordance.

使用皮尔逊相关系数、标准化差异一致性以及比率比率对21个预定义的模拟设计特征的一致性进行了评估，同时对研究特征和一致性进行了亚组和回归分析。

Results

Among 107 target trial emulation-randomised controlled trialpairs, the Pearson correlation coefficient was 0.59 (95% confidence interval(CI) 0.45 to 0.70), standardised difference agreement was 79% (85/107), and summary ratio of ratios was 0.96 (95% CI 0.92 to 1.01; I2=36%).

在107对目标试验模拟-随机对照试验配对中，皮尔逊相关系数为0.59（95%置信区间（CI）0.45至0.70），标准化差异一致性为79%（85/107），总结比率之比为0.96（95% CI 0.92至1.01；I2=36%）。

In 63 pairs with closer emulation of trial design, a higher level of agreement was observed, with a Pearson correlation coefficient of 0.83 (0.73 to 0.89) and standardised difference agreement of 87% (55/63).

在63对更接近试验设计模拟的配对中，观察到更高的一致性水平，皮尔逊相关系数为0.83（0.73至0.89），标准化差异一致性为87%（55/63）。

In subgroup analysis, trial emulations tended to systematically underestimate treatment effects from randomised controlled trials for specific outcomes related to venous thromboembolism and major adverse cardiovascular events, while overestimating those for respiratory outcomes, with pooled ratio of ratios of 0.76 (0.58 to 1.00; I2=40%), 0.91 (0.86 to 0.96; I2=32%), and 1.20 (1.03 to 1.40; I2=40%), respectively.

在亚组分析中，试验模拟倾向于系统性地低估随机对照试验中与静脉血栓栓塞和主要不良心血管事件相关的特定结果的治疗效果，同时高估呼吸结果的治疗效果，合并的比率比为0.76（0.58至1.00；I2=40%）、0.91（0.86至0.96；I2=32%）和1.20（1.03至1.40；I2=40%）。

Moreover, emulations based on claims data tended to underestimate the treatment effects (ratio of ratios 0.90, 0.82 to 0.99; I2=38%).

此外，基于索赔数据的模拟倾向于低估治疗效果（比率比为0.90，95%置信区间0.82至0.99；I2=38%）。

In univariate regression analyses, poorer target trial emulation-randomised controlled trialconcordance was associated with emulation designs with imbalanced baseline population characteristics, treatment started in hospital, and lower outcome emulation quality.

在单变量回归分析中，较差的目标试验模拟-随机对照试验一致性与基线人群特征不平衡的模拟设计、在医院开始治疗以及较低的结局模拟质量有关。

Conclusions

Current target trial emulations achieve moderate concordance when replicating their corresponding randomised controlled trials.

当前的目标试验模拟在复制其相应的随机对照试验时实现了中等程度的一致性。

Concordance could be improved by better emulation design, including better baseline and outcome emulation and leveraging multisource linked databases.

通过改进模拟设计，包括更佳的基线和结果模拟以及利用多源链接数据库，可以提升一致性。

systematic_review_registration

PROSPERO CRD42024619811.

PROSPERO CRD42024619811。