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多中心研究的未开发潜力:对心血管风险预测模型的综述揭示了分析不当和报告差异巨大的问题。

Untapped potential of multicenter studies: a review of cardiovascular risk prediction models revealed inappropriate analyses and wide variation in reporting.

作者信息

Wynants L, Kent D M, Timmerman D, Lundquist C M, Van Calster B

机构信息

1Department of Development and Regeneration, KU Leuven, Herestraat 49, box 7003, 3000 Leuven, Belgium.

5Department of Epidemiology, CAPHRI Care and Public Health Research Institute, Maastricht University, PO Box 9600, 6200 MD Maastricht, The Netherlands.

出版信息

Diagn Progn Res. 2019 Feb 22;3:6. doi: 10.1186/s41512-019-0046-9. eCollection 2019.

Abstract

BACKGROUND

Clinical prediction models are often constructed using multicenter databases. Such a data structure poses additional challenges for statistical analysis (clustered data) but offers opportunities for model generalizability to a broad range of centers. The purpose of this study was to describe properties, analysis, and reporting of multicenter studies in the Tufts PACE Clinical Prediction Model Registry and to illustrate consequences of common design and analyses choices.

METHODS

Fifty randomly selected studies that are included in the Tufts registry as multicenter and published after 2000 underwent full-text screening. Simulated examples illustrate some key concepts relevant to multicenter prediction research.

RESULTS

Multicenter studies differed widely in the number of participating centers (range 2 to 5473). Thirty-nine of 50 studies ignored the multicenter nature of data in the statistical analysis. In the others, clustering was resolved by developing the model on only one center, using mixed effects or stratified regression, or by using center-level characteristics as predictors. Twenty-three of 50 studies did not describe the clinical settings or type of centers from which data was obtained. Four of 50 studies discussed neither generalizability nor external validity of the developed model.

CONCLUSIONS

Regression methods and validation strategies tailored to multicenter studies are underutilized. Reporting on generalizability and potential external validity of the model lacks transparency. Hence, multicenter prediction research has untapped potential.

REGISTRATION

This review was not registered.

摘要

背景

临床预测模型通常使用多中心数据库构建。这种数据结构给统计分析带来了额外挑战(聚类数据),但为模型推广到广泛的中心提供了机会。本研究的目的是描述塔夫茨PACE临床预测模型注册中心多中心研究的特性、分析和报告情况,并说明常见设计和分析选择的后果。

方法

从塔夫茨注册中心随机选取50项在2000年后发表的多中心研究进行全文筛选。模拟示例说明了与多中心预测研究相关的一些关键概念。

结果

多中心研究参与中心数量差异很大(范围为2至5473个)。50项研究中有39项在统计分析中忽略了数据的多中心性质。在其他研究中,通过仅在一个中心构建模型、使用混合效应或分层回归,或使用中心层面特征作为预测变量来解决聚类问题。50项研究中有23项未描述获取数据的临床环境或中心类型。50项研究中有4项既未讨论所开发模型的可推广性,也未讨论其外部有效性。

结论

针对多中心研究的回归方法和验证策略未得到充分利用。关于模型可推广性和潜在外部有效性的报告缺乏透明度。因此,多中心预测研究具有未开发的潜力。

注册情况

本综述未注册。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ef17/6460661/667e64c63249/41512_2019_46_Fig1_HTML.jpg

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