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对临床效用与预测能力指标之间的关系以及人群中平均风险的影响进行评估。

An assessment of the relationship between clinical utility and predictive ability measures and the impact of mean risk in the population.

作者信息

McGeechan Kevin, Macaskill Petra, Irwig Les, Bossuyt Patrick Mm

机构信息

Sydney School of Public Health, The University of Sydney, Sydney, Australia.

出版信息

BMC Med Res Methodol. 2014 Jul 3;14:86. doi: 10.1186/1471-2288-14-86.

Abstract

BACKGROUND

Measures of clinical utility (net benefit and event free life years) have been recommended in the assessment of a new predictor in a risk prediction model. However, it is not clear how they relate to the measures of predictive ability and reclassification, such as the c-statistic and Net Reclassification Improvement (NRI), or how these measures are affected by differences in mean risk between populations when a fixed cutpoint to define high risk is assumed.

METHODS

We examined the relationship between measures of clinical utility (net benefit, event free life years) and predictive ability (c-statistic, binary c-statistic, continuous NRI(0), NRI with two cutpoints, binary NRI) using simulated data and the Framingham dataset.

RESULTS

In the analysis of simulated data, the addition of a new predictor tended to result in more people being treated when the mean risk was less than the cutpoint, and fewer people being treated for mean risks beyond the cutpoint. The reclassification and clinical utility measures showed similar relationships with mean risk when the mean risk was less than the cutpoint and the baseline model was not strong. However, when the mean risk was greater than the cutpoint, or the baseline model was strong, the reclassification and clinical utility measures diverged in their relationship with mean risk.Although the risk of CVD was lower for women compared to men in the Framingham dataset, the measures of predictive ability, reclassification and clinical utility were both larger for women. The difference in these results was, in part, due to the larger hazard ratio associated with the additional risk predictor (systolic blood pressure) for women.

CONCLUSION

Measures such as the c-statistic and the measures of reclassification do not capture the consequences of implementing different prediction models. We do not recommend their use in evaluating which new predictors may be clinically useful in a particular population. We recommend that a measure such as net benefit or EFLY is calculated and, where appropriate, the measure is weighted to account for differences in the distribution of risks between the study population and the population in which the new predictors will be implemented.

摘要

背景

在风险预测模型中评估新的预测指标时,推荐使用临床效用指标(净效益和无事件生存年数)。然而,尚不清楚它们与预测能力和重新分类指标(如c统计量和净重新分类改善(NRI))之间的关系,也不清楚当假定定义高风险的固定切点时,这些指标如何受到人群平均风险差异的影响。

方法

我们使用模拟数据和弗雷明汉数据集,研究了临床效用指标(净效益、无事件生存年数)与预测能力指标(c统计量、二元c统计量、连续NRI(0)、具有两个切点的NRI、二元NRI)之间的关系。

结果

在模拟数据分析中,当平均风险低于切点时,添加新的预测指标往往会导致更多的人接受治疗;而当平均风险超过切点时,接受治疗的人数则会减少。当平均风险低于切点且基线模型不强时,重新分类指标和临床效用指标与平均风险呈现出相似的关系。然而,当平均风险高于切点或基线模型较强时,重新分类指标和临床效用指标与平均风险的关系出现了分歧。尽管在弗雷明汉数据集中女性患心血管疾病的风险低于男性,但女性的预测能力、重新分类指标和临床效用指标均更高。这些结果的差异部分归因于女性与额外风险预测指标(收缩压)相关的更大风险比。

结论

诸如c统计量和重新分类指标等并不能反映实施不同预测模型的后果。我们不建议在评估哪些新的预测指标在特定人群中可能具有临床实用性时使用它们。我们建议计算净效益或EFLY等指标,并在适当情况下对该指标进行加权,以考虑研究人群与将实施新预测指标的人群之间风险分布的差异。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b3b0/4105158/0f5b21205a6f/1471-2288-14-86-1.jpg

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