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·918· http: //www.chinagp.net E-mail: zgqkyx@chinagp.net.cn March 2023, Vol.26 No.8
developed by foreign scholars,yet the former one has been found to have limited predictive value in Chinese HCM patients.
Objective To develop a risk prediction model for thrombotic events in Chinese patients with HCM. Methods A retrospective
cohort study design was used. Five hundred and thirty-seven HCM patients who admitted to West China Hospital of Sichuan
University from 2010 to 2018 were recruited. Post-discharge health status was collected by use of telephone follow-up or checking
the treatment status recorded in the electronic medical record system once every 6 to 12 months until a composite thrombotic event
(defined as the endpoint event) or death occurred or the determined thrombotic risk assessment day of this study(2019-12-31).
Univariate and multvariate Cox regression analyses were applied to build a thrombotic risk prediction model,and its internal
validation was tested in a resample using the bootstrapping technique. Results Due to data missing,24 cases were excluded,
and the other 513 cases were finally included. During a median follow-up of 4.2 years(IQR:1.3-6.2 years),thrombotic events
occurred in 42 cases(8.18%),with an annual morbidity rate of 2.10%〔95%CI(1.47%,2.73%)〕. By multivariate Cox
regression analysis,age,prior thrombotic event and left ventricular ejection fraction(LVEF) were identified(P<0.05) and
used for constructing the formula of SAAE score (S=prior stroke and other thrombotic events,A=atrial fibrillation,A=age,
E=LVEF) for predicting thrombotic events after being weighted based on the regression coefficient. Internal validation suggested
that SAAE score could discriminate thrombotic events in the whole population{Harrell's C-index=0.773〔95%CI(0.688,
0.858)〕},with a calibration slope of 1.006,and could well discriminate 1-year,3-year and 5-year thrombotic events
(Harrell's C-index=0.790,0.799,0.735),with a good calibration ability. In addition,the SAAE score also performed well
in distinguishing thrombotic events in patients with or without atrial fibrillation{Harrell's C-index=0.669〔95%CI(0.548,
0.791)〕,0.647〔95%CI(0.498,0.795)〕},with good calibration ability. Besides that,SAAE score could partially
discriminate 1-year,3-year and 5-year thrombotic events in these two groups,with certain calibration ability. For three groups
(whole study population,patients with/without atrial fibrillation),SAAE score could discriminate the risk of thrombotic events(either
low,moderate or high) excellently. For the whole study population,SAAE score was better than HCM Risk-CVA score in
distinguishing thrombotic events(P=0.013). Decision curve analysis showed the net benefit of SAAE score was better than HCM
Risk-CVA score at different prediction time points(1,3 and 5 years). Conclusion This thrombotic events risk prediction
model developed by us for Chinese HCM patients,namely SAAE score,could well stratify the risk of thrombotic events.
【Key words】 Cardiomyopathy,hypertrophic;Thromboembolism;Stroke;Atrial fibrillation;Age;Ejection
fraction;Score models
肥 厚 型 心 肌 病(hypertrophic cardiomyopathy, 事件风险预测模型,对于指导临床决策具有重要的临床
HCM)是一种以左心室壁肥厚为特征的疾病 [1-2] 。 意义。鉴于此,本研究拟构建适合中国 HCM 患者的 TE
HCM 常出现各种临床表现并伴随多种并发症,其中血 事件风险预测模型。
栓栓塞(thromboembolism,TE)事件发生率相对较高, 1 对象与方法
既往文献提示在 HCM 患者中,TE 事件的年发病率约为 1.1 研究对象 本研究系回顾性队列研究,收集
1.0%,明显高于一般人群 [3-4] ;如患者合并心房颤动, 2010—2018 年在四川大学华西医院就诊的 537 例 HCM
则 TE 事件年发病率更高 [5-7] 。作为 HCM 的一个重要 患者病例资料。本研究遵循《赫尔辛基宣言》的原则,
并发症,TE 事件可导致 HCM 患者预后不良及生存率降 并经四川大学华西医院生物医学研究伦理委员会批准
低 [2,5-6,8-10] 。 (批准文号:2019-1147)。由于为回顾性研究,委员
因此,如能在 HCM 患者中识别易发生 TE 事件的 会批准知情同意豁免。
高风险患者,则可以采取相应的预防措施,一定程度上 1.2 HCM 诊断标准、相关定义与基线数据收集 根据
降低 TE 事件发生的可能性。关于如何识别高危患者, 2014 年欧洲心脏病学协会指南 [2] ,HCM 的诊断是基于
相对于单个预测指标,在临床实践中通常使用风险预 左心室壁肥厚,并需排除负荷增加如高血压、主动脉瓣
测模型帮助临床决策,从而为患者做出合理的治疗选 狭窄和先天性主动脉瓣下隔膜等引起的左心室壁肥厚,
择 [11-12] ,因风险预测模型综合了某个疾病不同的病理 并排除了其他可能导致心肌肥厚的疾病,如已知的代谢
生理特征,可整体评估患者的预后。目前针对 HCM 患 疾病(如 Anderson-Fabry 病)或肥厚型心肌病综合征(如
者 TE 事件的风险预测,仅国外学者构建了两个模型: Noonan 综合征)等。成年人 HCM 的诊断标准为 [2] 左
HCM Risk-CVA [9] 及 French HCM score [13] 。既往研究 心室舒张末期室壁厚度≥ 15 mm,或者有明确家族史者
发现 HCM Risk-CVA 模型对于中国 HCM 患者的临床价 左心室舒张末期室壁厚度≥ 13 mm。本研究使用超声心
值较为有限 [14] 。因此,构建适合中国 HCM 患者的 TE 动图评估左心室壁肥厚等指标,严格按照目前国际通用