Summary
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.
Plain language summary
Multiple imputation of registry data to estimate clinical remission in pediatric patients with Crohn’s disease
What is this article about? The purpose of this study was to investigate whether multiple imputation can help researchers address the challenge of missing data when estimating effectiveness or how well a treatment works in patients treated in clinical practice rather than in a clinical trial. Multiple imputation is a statistical technique that fills in a patient’s missing data with values based on existing data from other patients with similar characteristics. The ImproveCareNow registry provided a suitable source of “real-world” clinical practice data for studying treatment effectiveness in pediatric patients with Crohn’s disease. However, registries often have partial or missing data, which can cause bias, or the tendency to overestimate or underestimate an outcome. In this study, multiple imputation was used to try to reduce bias caused by missing clinical remission outcome for some patients.
What were the results? The true clinical remission rate was 75.1%. Without multiple imputation, the remission rate was underestimated (72.6%). With multiple imputation, the estimated remission rate (74.8%) was closer to the true remission rate.
What do the study results mean? These results show that multiple imputation can reduce bias and improve validity, reliability, and efficiency when using registry data to estimate remission rates in pediatric patients with Crohn’s disease when some patients are missing remission data.
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