Department of Cardiology, Children's Hospital, Boston, MA, USA.
Am J Transplant. 2012 May;12(5):1240-8. doi: 10.1111/j.1600-6143.2011.03932.x. Epub 2012 Feb 2.
We sought to develop and validate a quantitative risk-prediction model for predicting the risk of posttransplant in-hospital mortality in pediatric heart transplantation (HT). Children <18 years of age who underwent primary HT in the United States during 1999-2008 (n = 2707) were identified using Organ Procurement and Transplant Network data. A risk-prediction model was developed using two-thirds of the cohort (random sample), internally validated in the remaining one-third, and independently validated in a cohort of 338 children transplanted during 2009-2010. The best predictive model had four categorical variables: hemodynamic support (ECMO, ventilator support, VAD support vs. medical therapy), cardiac diagnosis (repaired congenital heart disease [CHD], unrepaired CHD vs. cardiomyopathy), renal dysfunction (severe, mild-moderate vs. normal) and total bilirubin (≥ 2.0, 0.6 to <2.0 vs. <0.6 mg/dL). The C-statistic (0.78) and the Hosmer-Lemeshow goodness-of-fit (p = 0.89) in the model-development cohort were replicated in the internal validation and independent validation cohorts (C-statistic 0.75, 0.81 and the Hosmer-Lemeshow goodness-of-fit p = 0.49, 0.53, respectively) suggesting acceptable prediction for posttransplant in-hospital mortality. We conclude that this risk-prediction model using four factors at the time of transplant has good prediction characteristics for posttransplant in-hospital mortality in children and may be useful to guide decision-making around patient listing for transplant and timing of mechanical support.
我们旨在开发和验证一种用于预测儿科心脏移植(HT)后院内死亡风险的定量风险预测模型。使用器官采购与移植网络数据,确定了 1999 年至 2008 年期间在美国接受初次 HT 的<18 岁儿童(n=2707)。使用队列的三分之二(随机样本)开发风险预测模型,在其余三分之一中进行内部验证,并在 2009-2010 年接受移植的 338 名儿童队列中进行独立验证。最佳预测模型有四个分类变量:血流动力学支持(ECMO、呼吸机支持、VAD 支持与药物治疗)、心脏诊断(修复型先天性心脏病[CHD]、未修复型 CHD 与心肌病)、肾功能不全(严重、轻度-中度与正常)和总胆红素(≥2.0、0.6 至<2.0 与<0.6mg/dL)。模型开发队列中的 C 统计量(0.78)和 Hosmer-Lemeshow 拟合优度(p=0.89)在内部验证和独立验证队列中得到复制(C 统计量 0.75、0.81 和 Hosmer-Lemeshow 拟合优度 p=0.49、0.53),表明对移植后院内死亡率有可接受的预测。我们得出结论,这种在移植时使用四个因素的风险预测模型对儿童移植后院内死亡率具有良好的预测特征,可能有助于指导患者列入移植名单和机械支持的时机。