Brigham and Women's Hospital, Boston, Massachusetts, USA.
Emory University, Atlanta, Georgia, USA.
JACC Heart Fail. 2022 Dec;10(12):948-959. doi: 10.1016/j.jchf.2022.08.002. Epub 2022 Oct 12.
Clinical trials inform on average efficacy, but individualized risk assessments for outcome prediction are important in guiding treatment implementation.
The authors developed and validated a patient-specific risk score to predict survival at 1 and 2 years after HeartMate 3 (HM3) left ventricular assist device (LVAD) implantation.
The MOMENTUM 3 (Multicenter Study of MagLev Technology in Patients Undergoing Mechanical Circulatory Support Therapy with HeartMate 3) trial includes 2,200 HM3 LVAD patients in the pivotal trial and Continued Access Protocol study (2014-2018). The authors randomly assigned all patients to a derivation cohort (n = 1,540) or validation cohort (n = 660). Univariate mortality predictors were screened for potential model inclusion, stepwise selection was used to build the multivariable Cox proportional hazards regression model, and performance (discrimination and calibration) was evaluated.
Age, prior cardiac surgery (coronary artery bypass grafting [CABG] or valve procedure), lower serum sodium, higher blood urea nitrogen (BUN), small left ventricular size, and right atrial pressure-to-pulmonary capillary wedge pressure (RAP/PCWP) ratio >0.6 were significant risk factors for mortality. Receiver-operating characteristic (ROC) analysis in the validation cohort demonstrated an area under the curve (AUC) of 0.76 (95% CI: 0.70-0.81) at 1 year and 0.71 (95% CI: 0.66-0.77) at 2 years. Calibration between predicted and observed survival of the risk quintiles was high, with Pearson correlation coefficients of 0.986 and 0.994 at 1 and 2 years, respectively. Patients were successfully stratified into tertiles with higher-than-average, average, and lower-than-average survival, and observed mortality risk increased by 2-fold from one tertile to the next.
A practical, easy-to-use HM3 Survival Risk Score with 6 components was developed to accurately predict 1- and 2-year survival after HM3 LVAD implantation. The survival risk score can be used to provide individual survival estimates to facilitate shared decision making when considering HM3 LVAD therapy. (MOMENTUM 3 Trial Portfolio; NCT02224755, NCT02892955).
临床试验平均报告疗效，但预测结局的个体化风险评估对于指导治疗实施很重要。
作者开发并验证了一种预测 HeartMate 3（HM3）左心室辅助装置（LVAD）植入后 1 年和 2 年生存率的患者特异性风险评分。
MOMENTUM 3（多中心磁悬浮技术在接受 HeartMate 3 机械循环支持治疗的患者中的应用研究）试验纳入了 2200 例 HM3 LVAD 患者，包括关键性试验和继续准入方案研究（2014-2018 年）。作者将所有患者随机分配到推导队列（n=1540）或验证队列（n=660）。筛选出单变量死亡率预测因素，用于潜在模型纳入，逐步选择用于构建多变量 Cox 比例风险回归模型，并评估其性能（区分度和校准度）。
年龄、既往心脏手术（冠状动脉旁路移植术或瓣膜手术）、血清钠水平较低、血尿素氮（BUN）水平较高、左心室较小、右心房压与肺毛细血管楔压（RAP/PCWP）比值>0.6 是死亡率的显著危险因素。验证队列中的受试者工作特征（ROC）分析显示，1 年时曲线下面积（AUC）为 0.76（95%CI：0.70-0.81），2 年时 AUC 为 0.71（95%CI：0.66-0.77）。风险五分位数的预测与观察生存率之间的校准度很高，Pearson 相关系数分别为 1 年时 0.986 和 2 年时 0.994。患者成功地分为生存率较高、中等和较低的三分位数，从一个三分位数到下一个三分位数，观察到的死亡率风险增加了 2 倍。
作者开发了一种实用、易于使用的 HM3 生存风险评分，包含 6 个指标，可准确预测 HM3 LVAD 植入后 1 年和 2 年的生存率。该生存风险评分可用于提供个体生存估计，以促进在考虑 HM3 LVAD 治疗时进行共同决策。（MOMENTUM 3 试验组合；NCT02224755，NCT02892955）。
