Centre for Inherited Cardiovascular Diseases, Department of Cardiology, Great Ormond Street Hospital, London, United Kingdom.
Institute of Cardiovascular Sciences, University College London, London, United Kingdom.
JAMA Cardiol. 2019 Sep 1;4(9):918-927. doi: 10.1001/jamacardio.2019.2861.
Sudden cardiac death (SCD) is the most common mode of death in childhood hypertrophic cardiomyopathy (HCM), but there is no validated algorithm to identify those at highest risk.
To develop and validate an SCD risk prediction model that provides individualized risk estimates.
DESIGN, SETTING, AND PARTICIPANTS: A prognostic model was developed from a retrospective, multicenter, longitudinal cohort study of 1024 consecutively evaluated patients aged 16 years or younger with HCM. The study was conducted from January 1, 1970, to December 31, 2017.
The model was developed using preselected predictor variables (unexplained syncope, maximal left-ventricular wall thickness, left atrial diameter, left-ventricular outflow tract gradient, and nonsustained ventricular tachycardia) identified from the literature and internally validated using bootstrapping.
A composite outcome of SCD or an equivalent event (aborted cardiac arrest, appropriate implantable cardioverter defibrillator therapy, or sustained ventricular tachycardia associated with hemodynamic compromise).
Of the 1024 patients included in the study, 699 were boys (68.3%); mean (interquartile range [IQR]) age was 11 (7-14) years. Over a median follow-up of 5.3 years (IQR, 2.6-8.3; total patient years, 5984), 89 patients (8.7%) died suddenly or had an equivalent event (annual event rate, 1.49; 95% CI, 1.15-1.92). The pediatric model was developed using preselected variables to predict the risk of SCD. The model's ability to predict risk at 5 years was validated; the C statistic was 0.69 (95% CI, 0.66-0.72), and the calibration slope was 0.98 (95% CI, 0.59-1.38). For every 10 implantable cardioverter defibrillators implanted in patients with 6% or more of a 5-year SCD risk, 1 patient may potentially be saved from SCD at 5 years.
This new, validated risk stratification model for SCD in childhood HCM may provide individualized estimates of risk at 5 years using readily obtained clinical risk factors. External validation studies are required to demonstrate the accuracy of this model's predictions in diverse patient populations.
心脏性猝死（SCD）是儿童肥厚型心肌病（HCM）最常见的死亡方式，但目前尚无经过验证的算法来识别风险最高的患者。
开发并验证一种 SCD 风险预测模型，以提供个体化的风险评估。
设计、地点和参与者：这是一项从 1970 年 1 月 1 日至 2017 年 12 月 31 日进行的回顾性、多中心、纵向队列研究中开发的预后模型，纳入了 1024 例连续评估的年龄在 16 岁及以下的 HCM 患者。
该模型使用从文献中筛选出的预先选定的预测变量（不明原因的晕厥、最大左心室壁厚度、左心房直径、左心室流出道梯度和非持续性室性心动过速）进行开发，并通过自举法进行内部验证。
SCD 或同等事件（心搏骤停、适当的植入式心律转复除颤器治疗或与血流动力学受损相关的持续性室性心动过速）的复合结局。
在纳入的 1024 例患者中，699 例为男性（68.3%）；平均（四分位距[IQR]）年龄为 11（7-14）岁。中位随访时间为 5.3 年（IQR，2.6-8.3；总患者年数为 5984 年），89 例（8.7%）患者发生猝死或出现同等事件（年发生率为 1.49%；95%CI，1.15-1.92）。该儿科模型使用预先选定的变量来预测 SCD 风险。验证了该模型预测 5 年风险的能力；C 统计量为 0.69（95%CI，0.66-0.72），校准斜率为 0.98（95%CI，0.59-1.38）。在植入性心律转复除颤器的患者中，每植入 10 例，有 6%或以上的 5 年 SCD 风险，可能有 1 例患者在 5 年内免于 SCD。
这项针对儿童肥厚型心肌病 SCD 的新的、经过验证的风险分层模型，可能使用易于获得的临床危险因素，提供 5 年的个体化风险估计。需要进行外部验证研究，以证明该模型在不同患者人群中的预测准确性。
