Real-world Evidence for the Effectiveness and Safety of Ustekinumab Treatment in Children with Crohn’s Disease: A Retrospective Cohort Study using the ImproveCareNow Registry Data (REALITI Study)

Project start date and end date: 2021 - 2023


Ustekinumab is a fully human immunoglobulin G1 kappa monoclonal antibody which binds with high affinity to the p40 submit common to both interleukin (IL)-12 and IL-23. While ustekinumab is approved for the treatment of adult patients with moderate to severe Crohn’s disease, it does not have market authorization for use in children. Nevertheless, there is a substantial amount of off-label use of ustekinumab. In fact, a recent data query of the ICN Registry found over 900 patients with Crohn’s disease ages ≥2 to <26 years at 75 ICN sites were treated with ustekinumab.


The primary objective of this study is to evaluate the clinical effectiveness of ustekinumab (Stelara) by determining the clinical remission rate in pediatric patients with moderately to severely active Crohn’s Disease (CD) in the ImproveCareNow (ICN) registry who were treated with ustekinumab.

Research Topics & Methods:

This study is a historical, single-arm, non-interventional, observational study designed to provide evidence for the effectiveness and safety of ustekinumab treatment in pediatric patients (age 2 to <18) and young adults (age 18 to <26) with moderately to severely active Crohn’s disease.

Patients in this study have all been diagnosed with Crohn’s disease (CD) and enrolled in the ICN pediatric IBD patient registry. Health care, including CD therapies provided to the patients, and data collection has occurred prospectively during routine clinical practice, with no pre-defined research protocol for randomization or blinding to treatment assignment or outcome evaluation. The treatment decision to prescribe ustekinumab was made outside the context of this study.

The study includes two methods of data collection:

  • Chart Reviews
    • A retrospective chart review of all eligible patients enrolled in ICN treated with ustekinumab will be conducted at the participating sites to obtain data not collected in the ICN database. Seventy-two ICN centers with eligible patients were invited to participate.
  • Endoscopy Assessment
    • As part of the comprehensive chart review, endoscopic reports will be collected, de-identified, and assigned to external central reviewers blinded to whether the reports were collected prior to or after ustekinumab treatment. The endoscopy information will be evaluated and scored using the Simplified Endoscopic Mucosal Assessment for Crohn’s Disease (SEMA-CD).


This study will generate knowledge about the use of ustekinumab in the management of pediatric Crohn’s Disease.  The results of this study will be included as part of a Janssen submission to the FDA for the approval of the use of ustekinumab in pediatric patients, as they plan a phase 3 clinical trial of ustekinumab. In addition, the inclusion of real world evidence from ICN could help facilitate the approval of ustekinumab and other new drugs for  IBD, making new IBD treatments accessible to pediatric patients sooner, enabling better outcomes for our patients. If the ICN-Janssen REALITI Study is successful, other new  IBD  drugs could be studied in a similar way, having a major impact on the health of our patients as well as on the sustainability of ICN.


Janssen Pharmaceuticals has provided funding to ICN to perform this study; however, it is important to note that this is an observational study with a  retrospective chart review and not an interventional study.



Steve Steiner, MD, Riley Children’s Hospital
Shehzad Saeed, MD, ImproveCareNow & Dayton Children’s Hospital

For more information, please contact:
Clinical Professor, Department of Pediatrics, Division of Pediatric Gastroenterology
Interim Director, Susan B. Meister Child Health Evaluation and Research Center
1500 E. Medical Center Drive
MPB D5200, Box SPC5718
Ann Arbor, MI 48109-5718