Division of Cardiology, Department of Pediatrics, University of Pittsburgh School of Medicine, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
The Heart Center, Nationwide Children's Hospital, Columbus, Ohio, USA.
Catheter Cardiovasc Interv. 2021 Aug 1;98(2):E262-E274. doi: 10.1002/ccd.29657. Epub 2021 Mar 29.
We compared 5-year outcomes of transcatheter pulmonary valve (TPV) replacement with the Melody TPV in the post-approval study (PAS) and the investigational device exemption (IDE) trial.
As a condition of approval of the Melody TPV after the IDE trial, the Food and Drug Administration required that a PAS be conducted to evaluate outcomes of TPV replacement in a "real-world" environment. The 5-year outcomes of the PAS have not been published, and the IDE and PAS trials have not been compared.
The cohorts comprised all patients catheterized and implanted at 5 IDE sites and 10 PAS sites. Differences in trial protocols were detailed. Time-related outcomes and valve-related adverse events were compared between the two trials with Kaplan-Meier curves and log-rank testing.
167 patients (median age, 19 years) were catheterized and 150 underwent TPV replacement in the IDE trial; 121 were catheterized (median age, 17 years) and 100 implanted in the PAS. Freedom from hemodynamic dysfunction (p = .61) or any reintervention (p = .74) over time did not differ between trials. Freedom from stent fracture (p = .003) and transcatheter reintervention (p = .010) were longer in PAS, whereas freedom from explant (p = .020) and TPV endocarditis (p = .007) were shorter. Clinically important adverse events (AEs) were reported in 14% of PAS and 7.2% of IDE patients (p = .056); the incidence of any particular event was low in both.
Hemodynamic and time-related outcomes in the PAS and IDE trials were generally similar, confirming the effectiveness of the Melody TPV with real-world providers. There were few significant complications and limited power to identify important differences in AEs. The lack of major differences in outcomes between the two studies questions the usefulness of mandated costly post-approval studies as part of the regulatory process for Class III medical devices.
我们比较了经导管肺动脉瓣（TPV）置换术在批准后研究（PAS）和研究性器械豁免（IDE）试验中使用 Melody TPV 的 5 年结果。
作为 Melody TPV 在 IDE 试验后获得批准的条件之一，美国食品和药物管理局要求进行 PAS，以评估 TPV 置换在“真实世界”环境中的结果。PAS 的 5 年结果尚未公布，并且 IDE 和 PAS 试验尚未进行比较。
该队列包括在 5 个 IDE 地点和 10 个 PAS 地点进行导管化和植入的所有患者。详细比较了试验方案的差异。使用 Kaplan-Meier 曲线和对数秩检验比较了两项试验之间的时间相关结局和瓣膜相关不良事件。
167 例患者（中位数年龄 19 岁）接受 IDE 试验的导管化治疗，150 例接受 TPV 置换；121 例接受导管化治疗（中位数年龄 17 岁），100 例接受 PAS 植入。随着时间的推移，两组之间血流动力学功能障碍（p=0.61）或任何再干预（p=0.74）的无失败率没有差异。支架断裂（p=0.003）和经导管再干预（p=0.010）的无失败率在 PAS 中较长，而移植物（p=0.020）和 TPV 心内膜炎（p=0.007）的无失败率较短。PAS 患者中有 14%和 IDE 患者中有 7.2%报告了临床重要不良事件（AE）（p=0.056）；在两组中，任何特定事件的发生率都较低。
PAS 和 IDE 试验中的血流动力学和时间相关结局通常相似，证实了 Melody TPV 在真实世界提供者中的有效性。不良事件发生率较低，很少有严重并发症，难以确定不良事件的重要差异。两项研究之间的结局没有重大差异，这对作为 III 类医疗器械监管过程一部分的强制性昂贵批准后研究的有效性提出了质疑。
