Long-Term Effectiveness and Persistence of Ustekinumab in Crohn’s Disease Patients: An Observational Retrospective Study

Background/Objectives: The incidence of Crohn’s disease (CD) is increasing, necessitating effective long-term treatments. Ustekinumab (UST) is a biologic drug that blocks interleukins (ILs) 12/23. It has been approved for moderate-to-severe CD, but real-world long-term data in Chinese populations remain limited. Methods: This retrospective, single-center, cohort study included patients with CD who initiated UST between June 2020 and May 2025. The primary outcomes were clinical remission at week 24 and treatment persistence through week 96. Outcomes were calculated based on the number of patients with available data at each time point. Results: Of 110 patients screened, 95 were included; 69.5% were bio-naïve. The clinical remission rate was 86.3% (n = 82/95) at week 24, with 80% (n = 24/30) of those patients maintaining remission at week 96. Biological remission was achieved by 51.5% (n = 17/33) at week 24, with 47.8% (n = 11/23) maintaining it through week 96. No significant difference in efficacy was observed between UST monotherapy and combination therapy with 5-ASA. Multivariate analysis revealed absence of perianal lesion and normal serum albumin were associated with clinical remission at week 24. Endoscopic remission was documented in 39.3% (n = 11/28) of reassessed patients, with a significant reduction in Simple Endoscopic Score for Crohn’s Disease on UST. Analysis of drug survival on UST revealed no statistically significant difference (p = 0.2044) between bio-naïve and non-naïve patients. Adverse events were reported in 9.5% of patients, with none leading to treatment discontinuation. Conclusions: Ustekinumab was associated with significant long-term effectiveness and a favorable safety profile, including in bio-naïve and non-bio-naïve patients.