Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease.
Journal of comparative effectiveness research(2023)
摘要
To evaluate the performance of the multiple imputation (MI) method for estimating clinical effectiveness in pediatric Crohn's disease in the ImproveCareNow registry; to address the analytical challenge of missing data. Simulation studies were performed by creating missing datasets based on fully observed data from patients with moderate-to-severe Crohn's disease treated with non-ustekinumab biologics. MI was used to impute sPCDAI remission statuses in each simulated dataset. The true remission rate (75.1% [95% CI: 72.6%, 77.5%]) was underestimated without imputation (72.6% [71.8%, 73.3%]). With MI, the estimate was 74.8% (74.4%, 75.2%). MI reduced nonresponse bias and improved the validity, reliability, and efficiency of real-world registry data to estimate remission rate in pediatric patients with Crohn's disease.
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关键词
ImproveCareNow registry,Short Pediatric Crohn's Disease Activity Index (sPCDAI),clinical remission status,disease-specific patient registries,drug repurposing and relabeling,inflammatory bowel disease,missing data,multiple imputation method,pediatric Crohn's disease,real-world evidence
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