Background and aims: Previously published long-term safety data reported a favorable ustekinumab safety treatment profile for treatment of inflammatory bowel disease (IBD). We present the final cumulative safety data from pooled ustekinumab IBD phase 2/3 clinical studies through 5 years in Crohn's disease (CD) and 4 years in ulcerative colitis (UC).
Methods: In phase 3 studies, patients received a single IV placebo or ustekinumab (130mg or ~6mg/kg) induction dose followed by subcutaneous maintenance doses of placebo or ustekinumab (90mg q8w or q12w). Analyses included all patients who received one dose of study treatment and included patients who were biologic-naïve and patients with a history of biologic failure. Safety outcomes are summarized and presented using number of events per 100 patient-years of follow-up and corresponding 95% confidence interval.
Results: In this final pooled safety analysis, 2575 patients were treated with ustekinumab with 4826 patient-years of follow-up. Rates of key safety events, including MACE and malignancies, were similar between placebo and ustekinumab or not higher for ustekinumab.Opportunistic infections, including tuberculosis, and malignancies were reported infrequently. Rates of key safety events in the IBD group were no higher in the ustekinumab group than in the placebo group for both patients who were biologic naïve or who had previously failed a biologic. No lymphomas or cases of posterior reversible encephalopathy syndrome (PRES; formerly known as reversible posterior leukoencephalopathy syndrome [RPLS] were reported.
Conclusion: The final cumulative ustekinumab safety data through 5 years in CD and 4 years in UC demonstrated favorable safety compared to placebo and continues to support the well-established safety profile across all approved indications.
Keywords: inflammatory bowel disease; safety; ustekinumab.
© The Author(s) 2024. Published by Oxford University Press on behalf of European Crohn’s and Colitis Organisation.