Effectiveness and Persistency of Ustekinumab Treatment for Ulcerative Colitis: A Phoenix retrospective Cohort Study

Abstract

Background: Real-world data regarding ustekinumab (UST) for ulcerative colitis (UC) particularly in biologics-naïve patients is currently limited. This study aimed to elucidate the real-world effectiveness and safety of UST for UC.

Methods: Overall, 150 patients with UC treated with UST from March 2020 to January 2023 were enrolled across 7 referral hospitals. To assess the clinical efficacy and persistence of UST, retrospective analyses were conducted from weeks 8 to 56. Predictive factors concerning the response and persistence of UST were examined through univariate and multivariate analyses.

Results: Of the 150 patients, 125 received UST for remission induction, including 36% biologics-naïve. The response and remission rates were 72.8% and 56.0% at week 8 and 73.2% and 63.4% at week 56, respectively. Biologics-naïve patients represented higher response and remission rates at week 8 (84.4% and 73.3%) than those with biologics exposure (66.2% and 46.2%). Patients with prior antitumor necrosis factor (anti-TNF) and vedolizumab (VDZ) exposure had relatively lower response and remission rates (34.5% and 24.1%, respectively). The 1-year cumulative persistence rate was 84.0%. Multivariate analysis revealed that the chronic continuous type and prior anti-TNF and VDZ exposure were negative predictive factors for week 8 responsiveness. Clinical response at week 8 was a predictor of 1-year persistence. Adverse event incidence remained notably low at 6.4%.

Conclusions: This study highlights the safety and effectiveness of UST as an induction and maintenance therapy for UC. Chronic continuous type and previous anti-TNF and VDZ exposure negatively contributed to short-term effectiveness, whereas short-term effectiveness provided good persistency.

Keywords: bio-failure; bio-naïve; persistency; ulcerative colitis; ustekinumab.

Graphical Abstract

Figure 1.

Clinical response and remission rates of ustekinumab (UST) for ulcerative colitis (UC) in overall population (A) at weeks 8, 16, 32, and 56, and in the (B) classification with previous use of biologics and Janus kinase inhibitors (JAKs; biologics [bio]-naïve/bio-failure) and (C) classification with the number of previous use of biologic agents (bio 1 agent/bio ≥ 2 agents).

Figure 2.

Corticosteroid (CS)-free clinical remission rate of UST for UC at weeks 8, 16, 32, and 56 in the (A) overall population and (B) classification with previous use of biologics and JAKis (bio-naïve/bio-failure). (The clinical remission rate as control is shown on the left side).

Figure 3.

Clinical response and remission rates of UST for UC at week 8 in the classification with previous use of antitumor necrosis factor (anti-TNF), vedolizumab (VDZ), and tofacitinib (TOF).

Figure 4.

Cumulative persistence rate of UST (A) in the overall population, (B) classification with the presence of clinical response at week 8, (C) classification with previous exposure to biologics (bio-naïve and bio-failure), and (D) classification with previous exposure to both anti-TNF and VDZ.