Butala Neel M, Lalani Christina, Tale Archana, Song Yang, Kolte Dhaval, Baron Suzanne, Strom Jordan, Cohen David J, Yeh Robert W
Division of Cardiology, Department of Medicine, Rocky Mountain Regional VA Medical Center, Aurora, CO (N.M.B.).
Division of Cardiology, Department of Medicine, University of Colorado School of Medicine, Aurora (N.M.B.).
Circ Cardiovasc Interv. 2025 Jan;18(1):e014592. doi: 10.1161/CIRCINTERVENTIONS.124.014592. Epub 2025 Jan 21.
Food and Drug Administration-mandated postmarket studies for transcatheter aortic valve replacement in low-risk populations plan to use passively collected registry data linked to claims for long-term follow-up out to 10 years. Therefore, it is critically important to understand the validity of these claims-based end points. We sought to evaluate the ability of administrative claims with () codes to identify trial-adjudicated end points and reproduce treatment comparisons of aortic valve replacement in the Evolut Low Risk Trial.
We linked Evolut Low Risk trial patients to the Medicare Provider Analysis and Review database. We calculated sensitivity, specificity, positive predictive value, negative predictive value, and agreement statistic of claims to detect clinical end points through 2 years in trial patients. We additionally compared end points across treatment arms using trial-adjudicated outcomes versus claims-based outcomes.
Trial-adjudicated deaths were perfectly identified by claims. Claims had good performance in identifying trial-adjudicated disabling stroke (sensitivity 68.8%, specificity 99.0%, positive predictive value 64.7%, negative predictive value 99.1%, =0.66) and pacemaker placement (sensitivity 85.2%, specificity 98.4%, positive predictive value 90.4%, negative predictive value 97.5%, =0.86), but more modest performance in identifying trial-adjudicated myocardial infarction (=0.46) and vascular complications (=0.45). There was no difference between treatment arms for the primary end point of death or disabling stroke using trial data (hazard ratio, 0.83 [95% CI, 0.41-1.68]) or claims data (hazard ratio, 0.89 [95% CI, 0.43-1.81]; interaction =0.71).
Claims-based end points performed well in ascertaining death, disabling stroke, and pacemaker placement and were able to reproduce principal trial findings. These results support the selective use of claims-based end points for transcatheter aortic valve replacement postmarketing surveillance.
URL: https://www.clinicaltrials.gov; Unique identifier: NCT02701283.
美国食品药品监督管理局要求对低风险人群进行经导管主动脉瓣置换术的上市后研究，计划使用被动收集的登记数据，并将其与索赔数据相链接，以进行长达10年的长期随访。因此，了解这些基于索赔的终点指标的有效性至关重要。我们试图评估带有（）代码的行政索赔识别试验判定终点指标以及重现Evolut低风险试验中主动脉瓣置换术治疗比较结果的能力。
我们将Evolut低风险试验患者与医疗保险提供者分析和审查数据库相链接。我们计算了索赔检测试验患者2年内临床终点指标的敏感性、特异性、阳性预测值、阴性预测值和一致性统计量。我们还使用试验判定的结果与基于索赔的结果比较了各治疗组的终点指标。
索赔能够完美识别试验判定的死亡情况。索赔在识别试验判定的致残性卒中（敏感性68.8%，特异性99.0%，阳性预测值64.7%，阴性预测值99.1%，κ=0.66）和起搏器植入（敏感性85.2%，特异性98.4%，阳性预测值90.4%，阴性预测值97.5%，κ=0.86）方面表现良好，但在识别试验判定的心肌梗死（κ=0.46）和血管并发症（κ=0.45）方面表现一般。使用试验数据（风险比，0.83[95%CI，0.41-1.68]）或索赔数据（风险比，0.89[95%CI，0.43-1.81]；交互作用=0.71）时，各治疗组在死亡或致残性卒中主要终点指标方面没有差异。
基于索赔的终点指标在确定死亡、致残性卒中和起搏器植入方面表现良好，并且能够重现试验的主要结果。这些结果支持在经导管主动脉瓣置换术上市后监测中选择性地使用基于索赔的终点指标。
