An External Control Arm for the Oral Factor XIa Inhibitor Asundexian Phase 2 Trial in Atrial Fibrillation (PACIFIC-AF) Using Electronic Health Records.
IF 3
Q2 CARDIAC & CARDIOVASCULAR SYSTEMS
引用次数: 0
Abstract
Introduction: The aim of this study was to assess the applicability of an external control arm (ECA) approach in the clinical development of the oral factor XIa inhibitor asundexian for stroke prevention in patients with atrial fibrillation (AF), using prospectively collected data from the phase 2 PACIFIC-AF trial (NCT04218266) and real-world individual-level data from patients with AF treated with apixaban in the Optum® de-identified Electronic Health Record data set (Optum® EHR) 2013-2019.
Methods: To build ECAs, real-world patients meeting trial eligibility criteria were matched to patients enrolled in PACIFIC-AF. The primary outcome was the composite of International Society on Thrombosis and Haemostasis-defined major bleeding or clinically relevant non-major bleeding. Event rates were compared between PACIFIC-AF and ECAs at 85 days of trial duration and projected up to 2 years.
Results: Overall, 160,153 real-world patients met PACIFIC-AF eligibility criteria and were matched to patients from the PACIFIC-AF apixaban arm on 101 variables, with matching ratios of 1:10, 1:5, and 1:1. At day 85, the number of events for the primary outcome was 92 (3.68%) in the 1:10 ECA (2500 patients) and 6 (2.40%) in the PACIFIC-AF apixaban arm (250 patients), with incidence rates of 16.67 (90% confidence interval [CI] 13.92-19.63) and 11.10 (90% CI 4.83-19.45) per 100 person-years, respectively.
Conclusions: ECAs matching the PACIFIC-AF apixaban arm could be built from EHRs with concordant event rates for key trial endpoints. The ECA approach enabled the determination of event rates for treatment duration up to 2 years, thereby informing the asundexian pivotal phase 3 trial in AF.
使用电子健康记录治疗心房颤动（PACIFIC-AF）的口服XIa因子抑制剂Asundexian 2期临床试验的外部控制臂
本研究的目的是评估外部对照组（ECA）方法在口服XIa因子抑制剂assundexian预防房颤（AF）患者卒中临床开发中的适用性，使用前瞻性收集的2期PACIFIC-AF试验（NCT04218266）数据和2013-2019年Optum®去识别电子健康记录数据集（Optum®EHR）中阿哌沙班治疗的房颤患者的真实个体水平数据。方法：建立ECAs，将符合试验资格标准的现实世界患者与入组的PACIFIC-AF患者相匹配。主要终点是国际血栓和止血学会定义的大出血或临床相关的非大出血的综合指标。在85天的试验期间比较PACIFIC-AF和eca的事件发生率，并预计长达2年。结果：总体而言，160153名真实世界的患者符合PACIFIC-AF的资格标准，并在101个变量上与PACIFIC-AF阿哌沙班组的患者匹配，匹配比例为1:10,1:5和1:1。在第85天，主要结局的事件数在1:10 ECA组（2500例患者）中为92(3.68%)，在PACIFIC-AF阿哌沙班组（250例患者）中为6(2.40%)，发生率分别为16.67（90%可信区间[CI] 13.92-19.63）和11.10 (90% CI 4.83-19.45) / 100人年。结论：与PACIFIC-AF阿哌沙班组相匹配的eca可以从关键试验终点事件发生率一致的电子病历中构建。ECA方法能够确定治疗时间长达2年的事件发生率，从而为房颤的非连续性关键3期试验提供信息。
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来源期刊
Cardiology and Therapy
CARDIAC & CARDIOVASCULAR SYSTEMS-
CiteScore
5.30
自引率
0.00%
发文量
38
审稿时长
6 weeks
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