Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease-Reference-Cited by-同舟云学术

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

Published:2023-02-17 Issue: Volume: Page:
ISSN:2042-6305
Container-title:Journal of Comparative Effectiveness Research
language:en
Short-container-title:J. Comp. Eff. Res.

Author:

Zhang Nanhua¹²^ORCID,Liu Chunyan¹^ORCID,Steiner Steven J³^ORCID,Colletti Richard B⁴^ORCID,Baldassano Robert⁵^ORCID,Chen Shiran¹^ORCID,Cohen Stanley⁶⁷^ORCID,Kappelman Michael D⁸^ORCID,Saeed Shehzad⁹^ORCID,Conklin Laurie S¹⁰^ORCID,Strauss Richard¹⁰^ORCID,Volger Sheri¹⁰^ORCID,King Eileen¹²,Lo Kim Hung¹⁰

Affiliation:

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.

Funder

Janssen Research and Development

Publisher

Becaris Publishing Limited

Subject

Health Policy

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