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

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

Author information

¹Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.

²University of Cincinnati, Cincinnati, OH 45229, USA.

³Riley Hospital for Children/Indiana University School of Medicine, Indianapolis, IN 46202, USA.

⁴University of Vermont College of Medicine, Department of Pediatrics, Burlington, VT 05405, USA.

⁵The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.

⁶GI Care For Kids, Atlanta, GA 30342, USA.

⁷Morehouse School of Medicine, Atlanta, GA 30310, USA.

⁸University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.

⁹Dayton Children's Hospital/Wright State University, Dayton, OH 45404, USA.

¹⁰Janssen 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.