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Optimal sampling in derivation studies was associated with improved discrimination in external validation for heart
Naotsugu Iwakami1, Toshiyuki Nagai2, Toshiaki A Furukawa3
1Department of Cardiovascular Medicine, National Cerebral and Cardiovascular Center, Osaka, Japan; Department of Research Promotion and Management, National Cerebral and Cardiovascular Center, Osaka, Japan; Department of Health Promotion and Human Behavior, Kyoto University Graduate School of Medicine/Public Health, Kyoto, Japan.
Optimal sampling is crucial for the external validity of heart failure (HF) prognostic models. Ensuring derivation studies accurately reflect the target population improves prediction accuracy for 30-day mortality.
Area of Science:
- Cardiology
- Medical Informatics
- Biostatistics
Background:
- Prognostic models are essential tools in heart failure (HF) management.
- External validity, or generalizability, of these models is often limited.
- Identifying factors that ensure model performance in new populations is critical.
Purpose of the Study:
- To identify key determinants of external validity for prognostic models in heart failure.
- To assess the impact of study characteristics on the performance of HF prognostic models.
Main Methods:
- Systematic literature search for HF prognostic models predicting 30-day mortality.
- Performance evaluation in a cohort of 3,452 acute HF patients.
- Application of critical appraisal tools to assess bias and study characteristics.
Main Results:
- 224 models were identified from 6,354 studies; mean c-statistic was 0.64.
- Optimal sampling, reflecting the gap between study and target populations, was significantly associated with higher model performance (β=0.25, P<0.001).
- This association remained significant after adjusting for study characteristics (β=0.24, P=0.01).
Conclusions:
- Optimal sampling in derivation studies is a primary determinant of external validity for HF prognostic models.
- Improving how study populations represent the intended patient groups enhances model generalizability.
- These findings can guide the development and selection of more reliable prognostic tools for heart failure.

