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

Abstract

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.

Effectiveness and Safety of Ustekinumab in Pediatric Ulcerative Colitis: A Multi-center Retrospective Study from the Pediatric IBD Porto Group of ESPGHAN

“…Ustekinumab has a generally favorable safety profile [9, 10, 12-14, 16, 25, 29]. Worsening of UC, nasopharyngitis, and upper respiratory tract infection are the most frequently reported adverse events [10], while discontinuation of the therapy due to adverse events is infrequent [11,13,15,29]. The safety profile of ustekinumab in our study is similar to those reported for the adult population.…”

Ustekinumab in the Treatment of Inflammatory Bowel Diseases: Evolving Paradigms

“…Overall, although limited by the small sample sizes enrolled and the short follow-up time, these studies provided further reliable evidence for the effectiveness and safety of UST. A systematic review of 13 RW studies [ 89 ] noted that clinical remission and induction were achieved in 24% to 61% and in 47% to 77% of patients, respectively. Moreover, clinical remission was achieved in 33% to 79% of cases at the 52-week follow-up, whereas steroid-free remission was reported in six studies and ranged from 14% to 67%.…”

“…In UC, a systematic review of RW data found that patients with AEs ranged from 2.6% to 32% [ 89 ]. In another recent systematic review with a meta-analysis of RW, the incidence rates (iRs) of colectomy, mild and severe AEs, and serious infections were 4.8, 7.9, 0.8, and 0.3 per 100 patient-years, respectively [ 91 ].…”

“…Other treatments are usually recommended for refractory patients or those with specific contraindications to anti-TNF-α drugs [ 108 ]. The real-life data for the use of UST in this setting are still scarce and limited by small sample sizes and large heterogeneity of therapy protocols/outcome definitions [ 9 , 10 , 11 , 12 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 , 21 , 35 , 45 , 47 , 48 , 55 , 61 , 62 , 63 , 68 , 69 , 70 , 71 , 72 , 74 , 75 , 76 , 77 , 78 , 79 , 80 , 81 , 82 , 83 , 84 , 85 , 86 , 87 , 88 , 89 , 91 , 92 , 93 , 94 , 95 , 96 , 97 , 98 , 99 , 100 , 101 , …”

A Narrative Review of Cytokine Networks: Pathophysiological and Therapeutic Implications for Inflammatory Bowel Disease Pathogenesis

“…Accumulating evidence has shown the efficacy of ustekinumab, a human IgG1 monoclonal antibody targeting the shared p40 subunit of IL-12/23 for the treatment of CD and UC patients both in randomized clinical trials and real-life experiences [309][310][311][312][313][314][315][316][317]. Ustekinumab is a human IgG1 monoclonal antibody currently approved for the treatment of psoriasis, psoriatic arthritis, and moderate-severe CD and UC [318][319][320]. In a substantial real-world Israeli cohort study involving non-naïve-to-biological-treatment CD patients, ustekinumab demonstrated effectiveness and safety in inducing clinical remission.…”