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. 2023 Apr;12(4):e220136.
doi: 10.57264/cer-2022-0136. Epub 2023 Feb 17.

Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease

Nanhua Zhang  1   2 , Chunyan Liu  1 , Steven J Steiner  3 , Richard B Colletti  4 , Robert Baldassano  5 , Shiran Chen  1 , Stanley Cohen  6   7 , Michael D Kappelman  8 , Shehzad Saeed  9 , Laurie S Conklin  10 , Richard Strauss  10 , Sheri Volger  10 , Eileen King  1   2 , Kim Hung Lo  10
Affiliations

Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease

Nanhua Zhang et al. J Comp Eff Res. 2023 Apr.

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.

Keywords: 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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Conflict of interest statement

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Figures

Figure 1.
Figure 1.. Study design diagram.
CD: Crohn's disease; CI: Confidence interval; ICN: ImproveCareNow; sPCDAI: Short Pediatric Crohn's Disease Activity Index.
See this image and copyright information in PMC

References

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