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多变量预测模型的外部验证:方法学实施和报告的系统评价。

External validation of multivariable prediction models: a systematic review of methodological conduct and reporting.

机构信息

Centre for Statistics in Medicine, Botnar Research Centre, University of Oxford, Windmill Road, Oxford OX3 7LD, UK.

出版信息

BMC Med Res Methodol. 2014 Mar 19;14:40. doi: 10.1186/1471-2288-14-40.

Abstract

BACKGROUND

Before considering whether to use a multivariable (diagnostic or prognostic) prediction model, it is essential that its performance be evaluated in data that were not used to develop the model (referred to as external validation). We critically appraised the methodological conduct and reporting of external validation studies of multivariable prediction models.

METHODS

We conducted a systematic review of articles describing some form of external validation of one or more multivariable prediction models indexed in PubMed core clinical journals published in 2010. Study data were extracted in duplicate on design, sample size, handling of missing data, reference to the original study developing the prediction models and predictive performance measures.

RESULTS

11,826 articles were identified and 78 were included for full review, which described the evaluation of 120 prediction models. in participant data that were not used to develop the model. Thirty-three articles described both the development of a prediction model and an evaluation of its performance on a separate dataset, and 45 articles described only the evaluation of an existing published prediction model on another dataset. Fifty-seven percent of the prediction models were presented and evaluated as simplified scoring systems. Sixteen percent of articles failed to report the number of outcome events in the validation datasets. Fifty-four percent of studies made no explicit mention of missing data. Sixty-seven percent did not report evaluating model calibration whilst most studies evaluated model discrimination. It was often unclear whether the reported performance measures were for the full regression model or for the simplified models.

CONCLUSIONS

The vast majority of studies describing some form of external validation of a multivariable prediction model were poorly reported with key details frequently not presented. The validation studies were characterised by poor design, inappropriate handling and acknowledgement of missing data and one of the most key performance measures of prediction models i.e. calibration often omitted from the publication. It may therefore not be surprising that an overwhelming majority of developed prediction models are not used in practice, when there is a dearth of well-conducted and clearly reported (external validation) studies describing their performance on independent participant data.

摘要

背景

在考虑是否使用多变量(诊断或预后)预测模型之前,必须在未用于开发模型的数据中评估其性能(称为外部验证)。我们批判性地评估了多变量预测模型的外部验证研究的方法学实施和报告。

方法

我们对 2010 年在 PubMed 核心临床期刊上发表的描述一种或多种多变量预测模型的某种形式的外部验证的文章进行了系统回顾。研究数据由两名研究人员独立提取,内容包括设计、样本量、缺失数据处理、对原始预测模型开发研究的参考以及预测性能衡量标准。

结果

共确定了 11826 篇文章,并对其中 78 篇进行了全面审查,这些文章描述了对 120 个预测模型的评估。这些模型都是在未用于开发模型的参与者数据中进行评估的。33 篇文章描述了预测模型的开发及其在单独数据集上的性能评估,45 篇文章仅描述了对另一个数据集上已发表预测模型的评估。57%的预测模型以简化评分系统的形式呈现和评估。16%的文章未报告验证数据集的结局事件数量。54%的研究未明确提及缺失数据。67%的研究未报告模型校准情况,而大多数研究都评估了模型区分度。报告的性能衡量标准通常不清楚是针对完整回归模型还是简化模型。

结论

描述多变量预测模型某种形式的外部验证的研究绝大多数报告质量较差,关键细节经常未呈现。验证研究的设计较差,对缺失数据的处理和承认不当,预测模型的关键性能衡量标准之一即校准,也经常在出版物中被省略。因此,当缺乏关于独立参与者数据中模型性能的精心设计和明确报告(外部验证)研究时,开发的预测模型在实践中未得到广泛应用,也就不足为奇了。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fec7/3999945/b2ba1456af49/1471-2288-14-40-1.jpg

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