Efficacy, Effectiveness, and Safety of Ustekinumab for the Treatment of Ulcerative Colitis: A Systematic Review

Lay Summary This systematic review concluded that for the treatment of ulcerative colitis, ustekinumab has not only demonstrated efficacy in randomized controlled trials (and their extension studies) but also effectiveness and safety in observational studies from real clinical practice. Objective This study aimed to evaluate ustekinumab efficacy, effectiveness, and safety as a treatment for ulcerative colitis in adult patients. Methods A systematic review of the efficacy, effectiveness, and safety of ustekinumab in ulcerative colitis was carried out. The search was conducted via PubMed, Embase, and the Cochrane library. Two reviewers independently assessed the quality of studies and extracted study data. Results Of the 892 studies identified, 17 were included: 1 randomized controlled trial (RCT), 3 long-term extensions, and 13 observational studies. In the randomized clinical trial evaluating efficacy at week 8, clinical remission was achieved in 16% of patients, whereas clinical response was achieved in 51% and 62% of patients who received intravenous ustekinumab at a dose of 130 mg and 6 mg/kg, respectively. At 3 years' follow-up, symptomatic remission was achieved in 68% of patients. On the other hand, the effectiveness of ustekinumab was evaluated in 13 observational studies. In these studies, clinical remission at induction was achieved in 24% to 61% of cases, whereas clinical response at induction was achieved in 47% to 77% of cases. Moreover, clinical remission was achieved in 33% to 79% of cases at 52 weeks of follow-up. The adverse events ranged from 2.6% to 77% of all the studies that reported safety data. Adverse events leading to discontinuation ranged from 2.6% to 8.1%, and serious adverse events were uncommon and ranged from 3.7% to 6.0%. Conclusions Ustekinumab has demonstrated efficacy (in RCTs), effectiveness (in real clinical practice), and safety for the treatment of ulcerative colitis.