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

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; Kim Hung Lo

doi:10.57264/cer-2022-0136

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

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

Authors

Nanhua Zhang^{1

2}, Chunyan Liu¹, Steven J Steiner³, Richard B Colletti⁴, Robert Baldassano⁵, Shiran Chen¹, Stanley Cohen^{6

7}, Michael D Kappelman⁸, Shehzad Saeed⁹, Laurie S Conklin¹⁰, Richard Strauss¹⁰, Sheri Volger¹⁰, Eileen King^{1

2}, Kim Hung Lo¹⁰

Affiliations

¹ Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
² University of Cincinnati, Cincinnati, OH 45229, USA.
³ Riley Hospital for Children/Indiana University School of Medicine, Indianapolis, IN 46202, USA.
⁴ University of Vermont College of Medicine, Department of Pediatrics, Burlington, VT 05405, USA.
⁵ The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
⁶ GI Care For Kids, Atlanta, GA 30342, USA.
⁷ Morehouse School of Medicine, Atlanta, GA 30310, USA.
⁸ University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
⁹ Dayton Children's Hospital/Wright State University, Dayton, OH 45404, USA.
¹⁰ Janssen Research & Development, LLC, Spring House, PA 19477, USA.

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.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Child
Crohn Disease* / drug therapy
Humans
Inflammatory Bowel Diseases*
Remission Induction
Reproducibility of Results
Treatment Outcome