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建立并验证口服补液盐治疗体位性心动过速综合征患儿疗效的多变量预测模型。

Establishment and validation of a multivariate predictive model for the efficacy of oral rehydration salts in children with postural tachycardia syndrome.

机构信息

Department of Pediatrics, Peking University First Hospital, Beijing, China; Department of Cardiology, Beijing Children's Hospital, Beijing, China.

Department of Pediatrics, Peking University First Hospital, Beijing, China.

出版信息

EBioMedicine. 2024 Feb;100:104951. doi: 10.1016/j.ebiom.2023.104951. Epub 2024 Jan 4.

Abstract

BACKGROUND

The therapeutic effectiveness of the empirical and unselected use of oral rehydration salts (ORS) on postural tachycardia syndrome (POTS) is not satisfactory in children. Therefore, looking for suitable predictors of the therapeutic effects of ORS before treatment is extremely necessary to implement individualised treatment for paediatric patients with POTS.

METHODS

A retrospective case-control analysis of 130 patients (aged 5-18 years) who suffered from POTS with a 3-month treatment of ORS was conducted. A nomogram model was developed in the training set (n = 87) to predict the therapeutic response to ORS. Univariate analysis and logistic regression were applied to select the most useful predictors. ROC curves were applied to evaluate the discriminative performance of the nomogram model. The nomogram was then evaluated by calibration curves and the Hosmer-Lemeshow (H-L) test. The results were further validated using 1000 bootstrap resamples. External validation was performed in an independent validation set (n = 43).

FINDINGS

Among the ten variables with significant differences between the responders and non-responders in univariate analysis, five variables were found to be independently associated factors for ORS therapeutic efficacy among POTS children in the further logistic regression, including mean corpuscular haemoglobin concentration (MCHC), mean corpuscular volume (MCV), mean arterial pressure (MAP) at the first minute of the upright position, urine specific gravity (SG), and P-wave voltage peaking ratio (PWP). The nomogram model was established in the training set (AUC 0.926 [95% CI: 0.865-0.988], yielding a sensitivity of 87.8% and a specificity of 86.8%). The calibration curves showed good agreement between the prediction of the nomogram and actual observation in both the training and validation sets. The nomogram also effectively predicted the external validation set (sensitivity 82.1%, specificity 73.3%, and accuracy 79.1%).

INTERPRETATION

We established a feasible and high-precision nomogram model to predict the efficacy of ORS, which would help implement individualised treatment for children with POTS.

FUNDING

This study was supported by National High-Level Hospital Clinical Research Funding (Multi-centre Clinical Research Project of Peking University First Hospital) (2022CR59).

摘要

背景

经验性和非选择性使用口服补液盐(ORS)治疗体位性心动过速综合征(POTS)在儿童中的疗效并不令人满意。因此,在治疗前寻找 ORS 治疗效果的合适预测因子对于实施儿科 POTS 患者的个体化治疗极为必要。

方法

对 130 例(年龄 5-18 岁)接受 ORS 治疗 3 个月的 POTS 患者进行回顾性病例对照分析。在训练集中(n=87)建立了列线图模型以预测 ORS 治疗反应。应用单因素分析和逻辑回归选择最有用的预测因子。ROC 曲线用于评估列线图模型的判别性能。通过校准曲线和 Hosmer-Lemeshow(H-L)检验评估列线图。然后通过 1000 次 bootstrap 重采样进行验证。外部验证在独立验证集中进行(n=43)。

结果

在单因素分析中,反应者和无反应者之间有 10 个变量有显著差异,其中 5 个变量在进一步的逻辑回归中被发现是儿童 POTS 患者 ORS 治疗效果的独立相关因素,包括平均红细胞血红蛋白浓度(MCHC)、平均红细胞体积(MCV)、直立位第一分钟的平均动脉压(MAP)、尿比重(SG)和 P 波电压峰值比(PWP)。在训练集中建立了列线图模型(AUC 0.926 [95%CI:0.865-0.988],灵敏度为 87.8%,特异性为 86.8%)。校准曲线显示,训练集和验证集的列线图预测与实际观察之间具有良好的一致性。该列线图还能有效预测外部验证集(灵敏度 82.1%,特异性 73.3%,准确率 79.1%)。

结论

我们建立了一种可行且高精度的列线图模型来预测 ORS 的疗效,这将有助于对 POTS 患儿实施个体化治疗。

资助

本研究得到国家高水平医院临床研究资助(北京大学第一医院多中心临床研究项目)(2022CR59)的支持。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0b21/10796963/cfbc8d6e1b91/gr1.jpg

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