Safety of Ustekinumab in Inflammatory Bowel Disease: Pooled Safety Analysis of Results from Phase 2/3 Studies

Inflamm Bowel Dis. 2020 Sep 23;izaa236. doi: 10.1093/ibd/izaa236. Online ahead of print.

William J Sandborn 1, Brian G Feagan 2, Silvio Danese 3, Christopher D O'Brien 4, Elyssa Ott 5, Colleen Marano 4, Thomas Baker 5, Yiying Zhou 4, Sheri Volger 4, Ilia Tikhonov 4, Christopher Gasink 5, Bruce E Sands 6, Subrata Ghosh 7


Author information

  • 1University of California San Diego, La Jolla, CA, USA.
  • 2Robarts Clinical Trials, Robarts Research Institute, Western University, London, Ontario, Canada.
  • 3Humanitas Clinical Research Center-IRCCS and Humanitas University, Department of Biomedical Sciences, Milan, Italy.
  • 4Janssen Research & Development, LLC, Spring House, PA, USA.
  • 5Janssen Scientific Affairs, LLC, Horsham, PA, USA.
  • 6Dr. Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 7Institute of Translational Medicine and NIHR Biomedical Research Center, University of Birmingham, United Kingdom.


Background: Ustekinumab is currently approved globally in Crohn's disease (CD) and psoriatic diseases. Recent phase 3 data demonstrate safety/efficacy in ulcerative colitis (UC). Crohn's disease and UC phase 3 programs had similar study designs, facilitating integrated safety analyses.

Methods: Data from 6 ustekinumab phase 2/3 CD and UC studies were pooled, and safety was evaluated through 1 year. Patients received 1 placebo or ustekinumab (generally 130 mg or ~6 mg/kg) intravenous induction, then subcutaneous (90 mg) maintenance every 8/12 weeks. Analyses incorporated all patients who received ≥1 ustekinumab dose. Safety outcomes are presented as percentages of patients (induction) and as number of patients with events per 100 patient-years of follow-up (through 1 year). For key safety events, 95% confidence intervals (CIs) are provided, as appropriate. Hazard ratios with 95% CIs from time-to-event analyses for serious adverse events and serious infections were also performed.

Results: Through 1 year, 2574 patients received ustekinumab (1733 patient-years of follow-up). The number of patients with adverse events per 100 patient-years (placebo 165.99 [95% CI, 155.81-176.67] vs ustekinumab 118.32 [95% CI, 113.25-123.55]), serious AEs (27.50 [95% CI, 23.45-32.04] vs 21.23 [95% CI, 19.12-23.51]), infections (80.31 [95% CI, 73.28-87.84] vs 64.32 [95% CI, 60.60-68.21]), serious infections (5.53 [95% CI, 3.81-7.77] vs 5.02 [95% CI, 4.02-6.19]), and malignancies excluding nonmelanoma skin cancer (0.17 [95% CI, 0.00-0.93] vs 0.40 [95% CI, 0.16-0.83]) were similar between placebo and ustekinumab.

Conclusions: The safety profile of ustekinumab across the pooled inflammatory bowel disease population through 1 year was favorable and generally comparable to placebo. These data are consistent with the established safety profile of ustekinumab across indications.

Clinicaltrials.gov numbers: NCT00265122; NCT00771667; NCT01369329; NCT01369342; NCT01369355; NCT02407236.

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