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系统性硬化症相关的手指溃疡中血小板抑制剂的使用:EUSTAR 研究对 DU-VASC 模型的推导和验证。

Use of platelet inhibitors for digital ulcers related to systemic sclerosis: EUSTAR study on derivation and validation of the DU-VASC model.

机构信息

Department of Rheumatology, University Hospital Zurich.

Epidemiology, Biostatistics and Prevention Institute, Department of Biostatistics.

出版信息

Rheumatology (Oxford). 2023 Feb 6;62(SI):SI91-SI100. doi: 10.1093/rheumatology/keac405.

DOI:10.1093/rheumatology/keac405
PMID:35904554
Abstract

OBJECTIVE

To develop and validate the prognostic prediction model DU-VASC to assist the clinicians in decision-making regarding the use of platelet inhibitors (PIs) for the management of digital ulcers in patients with systemic sclerosis. Secondly, to assess the incremental value of PIs as predictor.

METHODS

We analysed patient data from the European Scleroderma Trials and Research group registry (one time point assessed). Three sets of derivation/validation cohorts were obtained from the original cohort. Using logistic regression, we developed a model for prediction of digital ulcers (DUs). C-Statistics and calibration plots were calculated to evaluate the prediction performance. Variable importance plots and the decrease in C-statistics were used to address the importance of the predictors.

RESULTS

Of 3710 patients in the original cohort, 487 had DUs and 90 were exposed to PIs. For the DU-VASC model, which includes 27 predictors, we observed good calibration and discrimination in all cohorts (C-statistic = 81.1% [95% CI: 78.9%, 83.4%] for the derivation and 82.3% [95% CI: 779.3%, 85.3%] for the independent temporal validation cohort). Exposure to PIs was associated with absence of DUs and was the most important therapeutic predictor. Further important factors associated with absence of DUs were lower modified Rodnan skin score, anti-Scl-70 negativity and normal CRP. Conversely, the exposure to phosphodiesterase-5 inhibitor, prostacyclin analogues or endothelin receptor antagonists seemed to be associated with the occurrence of DUs. Nonetheless, previous DUs remains the most impactful predictor of DUs.

CONCLUSION

The DU-VASC model, with good calibration and discrimination ability, revealed that PI treatment was the most important therapy-related predictor associated with reduced DU occurrence.

摘要

目的

开发和验证预后预测模型 DU-VASC,以帮助临床医生在系统性硬化症患者的管理中决策是否使用血小板抑制剂 (PIs) 来治疗手指溃疡。其次,评估 PIs 作为预测因子的增量价值。

方法

我们分析了来自欧洲硬皮病试验和研究组登记处(一次评估)的患者数据。从原始队列中获得了三组推导/验证队列。我们使用逻辑回归为预测手指溃疡 (DUs) 开发了一个模型。计算 C 统计量和校准图以评估预测性能。使用变量重要性图和 C 统计量的减少来确定预测因子的重要性。

结果

在原始队列的 3710 名患者中,有 487 名患有 DUs,90 名接受了 PIs 治疗。对于包含 27 个预测因子的 DU-VASC 模型,我们观察到所有队列的校准和区分度都很好(推导队列的 C 统计量为 81.1%[95%CI:78.9%,83.4%],独立时间验证队列为 82.3%[95%CI:779.3%,85.3%])。PI 暴露与无 DUs 相关,是最重要的治疗预测因子。与无 DUs 相关的其他重要因素是较低的改良 Rodnan 皮肤评分、抗 Scl-70 阴性和正常 CRP。相反,使用磷酸二酯酶-5 抑制剂、前列环素类似物或内皮素受体拮抗剂似乎与 DUs 的发生有关。尽管如此,既往 DUs 仍是 DUs 发生的最具影响力的预测因子。

结论

DU-VASC 模型具有良好的校准和区分能力,表明 PI 治疗是与减少 DUs 发生相关的最重要的治疗相关预测因子。

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