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

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dc.contributor.authorZhang, Nanhua
dc.contributor.authorLiu, Chunyan
dc.contributor.authorSteiner, Steven J.
dc.contributor.authorColletti, Richard B.
dc.contributor.authorBaldassano, Robert
dc.contributor.authorChen, Shiran
dc.contributor.authorCohen, Stanley
dc.contributor.authorKappelman, Michael D.
dc.contributor.authorSaeed, Shehzad
dc.contributor.authorConklin, Laurie S.
dc.contributor.authorStrauss, Richard
dc.contributor.authorVolger, Sheri
dc.contributor.authorKing, Eileen
dc.contributor.authorLo, Kim Hung
dc.contributor.departmentPediatrics, School of Medicine
dc.date.accessioned2024-02-26T14:18:39Z
dc.date.available2024-02-26T14:18:39Z
dc.date.issued2023
dc.description.abstractAims: 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.
dc.eprint.versionFinal published version
dc.identifier.citationZhang N, Liu C, Steiner SJ, et al. Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease. J Comp Eff Res. 2023;12(4):e220136. doi:10.57264/cer-2022-0136
dc.identifier.urihttps://hdl.handle.net/1805/38649
dc.language.isoen_US
dc.publisherBecaris
dc.relation.isversionof10.57264/cer-2022-0136
dc.relation.journalJournal of Comparative Effectiveness Research
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 Internationalen
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/4.0
dc.sourcePMC
dc.subjectClinical remission status
dc.subjectDisease-specific patient registries
dc.subjectDrug repurposing and relabeling
dc.subjectImproveCareNow registry
dc.subjectInflammatory bowel disease
dc.subjectMissing data
dc.subjectMultiple imputation method
dc.subjectPediatric Crohn's disease
dc.subjectReal-world evidence
dc.subjectShort Pediatric Crohn's Disease Activity Index (sPCDAI)
dc.titleUsing multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease
dc.typeArticle
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