1. data to estimate clinical remission in pediatric inflammatory bowel disease
- Author
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Nanhua Zhang, Chunyan Liu, Steven J Steiner, Richard B Colletti, Robert Baldassano, Shiran Chen, Stanley Cohen, Michael D Kappelman, Shehzad Saeed, Laurie S Conklin, Richard Strauss, Sheri Volger, Eileen King, and Kim Hung Lo
- Subjects
clinical remission status ,disease-specific patient registries ,drug repurposing and relabeling ,improvecarenow registry ,inflammatory bowel disease ,missing data ,multiple imputation method ,pediatric crohn’s disease ,real-world evidence ,short pediatric crohn’s disease activity index (spcdai) ,Public aspects of medicine ,RA1-1270 - Abstract
Aim: 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. Materials & methods: 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. Results: 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%). Conclusion: 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.
- Published
- 2023
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