Propensity score methods for comparative-effectiveness analysis: A case study of direct oral anticoagulants in the atrial fibrillation population.
Propensity score methods for comparative-effectiveness analysis: A case study of direct oral anticoagulants in the atrial fibrillation population.
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DOI：
10.1371/journal.pone.0262293
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发表时间：
2022
期刊：
影响因子：
3.7
通讯作者：
Langhorne P
中科院分区：
文献类型：
--
作者：
Ciminata G;Geue C;Wu O;Deidda M;Kreif N;Langhorne P
探讨在苏格兰直接口服抗凝剂（DOACs）卫生技术评估的背景下，使用真实世界证据（RWE）评估相对有效性时的方法学挑战。我们使用来自处方信息系统（PIS）、苏格兰发病率记录（SMR）和新抗凝患者死亡率记录的关联数据，探索使用倾向评分（PS）匹配、逆概率加权（IPW）和协变量调整的方法学挑战。模型性能通过标准化差异评估。比较了所有doac（包括阿哌沙班、达比加群和利伐沙班）与华法林的临床结果（中风和大出血）和死亡率。患者从首次口服抗凝剂处方到首次临床事件或死亡，随访2年。对治疗切换或停止进行审查。总的来说，每个PS模型都得到了患者协变量的良好平衡。当应用于样本量相对较大的亚组（联合doacs与华法林）时，IPW被发现是评估协变量平衡的最佳方法。IPTW-IPCW方法的治疗效果更大，但仍与其他PS方法估计的治疗效果一致。当应用于样本量较小的亚组（达比加群与华法林）时，结果模型中PS的协变量调整效果良好，因为该方法不需要进一步减少样本量，也不需要修剪或截断极端权值。适当PS方法的选择可能根据数据的特点而有所不同。如果未观察到的混淆假设成立，则应确定和测试多种方法。基于PS的方法可以使用常规收集的关联数据来实施，从而支持卫生技术决策。
To explore methodological challenges when using real-world evidence (RWE) to estimate comparative-effectiveness in the context of Health Technology Assessment of direct oral anticoagulants (DOACs) in Scotland. We used linkage data from the Prescribing Information System (PIS), Scottish Morbidity Records (SMR) and mortality records for newly anticoagulated patients to explore methodological challenges in the use of Propensity score (PS) matching, Inverse Probability Weighting (IPW) and covariate adjustment with PS. Model performance was assessed by standardised difference. Clinical outcomes (stroke and major bleeding) and mortality were compared for all DOACs (including apixaban, dabigatran and rivaroxaban) versus warfarin. Patients were followed for 2 years from first oral anticoagulant prescription to first clinical event or death. Censoring was applied for treatment switching or discontinuation. Overall, a good balance of patients’ covariates was obtained with every PS model tested. IPW was found to be the best performing method in assessing covariate balance when applied to subgroups with relatively large sample sizes (combined-DOACs versus warfarin). With the IPTW-IPCW approach, the treatment effect tends to be larger, but still in line with the treatment effect estimated using other PS methods. Covariate adjustment with PS in the outcome model performed well when applied to subgroups with smaller sample sizes (dabigatran versus warfarin), as this method does not require further reduction of sample size, and trimming or truncation of extreme weights. The choice of adequate PS methods may vary according to the characteristics of the data. If assumptions of unobserved confounding hold, multiple approaches should be identified and tested. PS based methods can be implemented using routinely collected linked data, thus supporting Health Technology decision-making.
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影响因子：
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