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根据三种预测模型评估原发性血小板增多症患者的血栓风险:一项外部验证研究。

Risk of thrombosis in essential thrombocythemia according to three prediction models: an external validation study.

机构信息

Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hatyai, Songkhla, Thailand.

Department of Pathology, Faculty of Medicine, Prince of Songkla University, Hatyai, Songkhla, Thailand.

出版信息

J Thromb Thrombolysis. 2023 Apr;55(3):527-535. doi: 10.1007/s11239-023-02769-y. Epub 2023 Jan 18.

Abstract

BACKGROUND

Thrombosis is a major complication of essential thrombocythemia (ET). There are three well-known prediction models for thrombotic risk in ET patients. However, only few external validation studies for the performance of these models in Asian populations have been conducted. Thus, we aimed to evaluate the performance of these models for predicting the risk of thrombosis in Thai patients with ET.

METHODS

We retrospectively evaluated the clinical characteristics and thrombotic risk of 149 Thai ET patients in a university hospital in Southern Thailand between 2002 and 2019. Thrombotic risk variables were evaluated using Cox proportional hazard regression. The Brier score, calibration plot, and Harrel concordance index (C-index) were used to evaluate the performance of the three models.

RESULTS

With a median follow-up of 5.2 years, there were a total of 28 thrombotic events in 26 patients. Age > 60 years was a significant prognostic factor for thrombosis in the multivariate Cox regression analysis. The Brier scores were 0.251, 0.273, and 0.276 in the conventional, IPSET-thrombosis, and revised IPSET-thrombosis models, respectively. The conventional model had optimal calibration and good discrimination (C-index, 0.67; 95%CI:0.55-0.79). The IPSET thrombosis (C-index 0.33; 95%CI:0.20-0.49) and revised IPSET thrombosis (C-index 0.31; 95%CI:0.18-0.44) models showed poor discrimination.

CONCLUSION

The conventional model, which is based on age and history of thrombosis, is the best model to predict thrombotic risk in Thai ET patients. Further studies with a larger number of patients with thrombotic events are needed to validate the IPSET-thrombosis and revised IPSET-thrombosis models.

摘要

背景

血栓形成是原发性血小板增多症(ET)的主要并发症。有三个著名的 ET 患者血栓形成风险预测模型。然而,只有少数关于这些模型在亚洲人群中的表现的外部验证研究。因此,我们旨在评估这些模型在预测泰国 ET 患者血栓形成风险方面的表现。

方法

我们回顾性评估了 2002 年至 2019 年间泰国南部一所大学医院的 149 例泰国 ET 患者的临床特征和血栓形成风险。使用 Cox 比例风险回归评估血栓形成风险变量。使用 Brier 评分、校准图和 Harrel 一致性指数(C 指数)评估三个模型的性能。

结果

中位随访 5.2 年后,26 例患者共发生 28 例血栓形成事件。年龄>60 岁是多变量 Cox 回归分析中血栓形成的显著预后因素。常规模型、IPSET-thrombosis 模型和修订后的 IPSET-thrombosis 模型的 Brier 评分分别为 0.251、0.273 和 0.276。常规模型具有最佳的校准和良好的区分度(C 指数,0.67;95%CI:0.55-0.79)。IPSET 血栓形成(C 指数 0.33;95%CI:0.20-0.49)和修订后的 IPSET 血栓形成(C 指数 0.31;95%CI:0.18-0.44)模型显示出较差的区分度。

结论

基于年龄和血栓形成史的常规模型是预测泰国 ET 患者血栓形成风险的最佳模型。需要进一步进行更多血栓形成事件患者的研究来验证 IPSET-thrombosis 和修订后的 IPSET-thrombosis 模型。

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