To evaluate the long-term efficacy and safety of Ustekinumab (UST) in a large cohort of patients with perianal fistulizing Crohn's disease (pfCD), identify independent predictors of fistula healing, and develop a clinical nomogram for individualized outcome prediction.
Approach:
Study Design: Multicenter retrospective cohort study conducted across seven tertiary referral hospitals in China, enrolling 163 pfCD patients treated with UST from 2020 to 2023.
Endpoints: Primary endpoints included fistula response and clinical remission rates, with univariate/multivariate logistic regression used to identify healing predictors.
Key Findings:
Fistula response rate was 88.3% and clinical remission rate was 70.1% at final follow-up.
Significant improvements in inflammatory biomarkers (CRP and ESR) and hemoglobin levels were observed.
Definitive surgery and fewer prior biologics were independent predictors of healing.
The developed nomogram showed good discriminative ability (AUC = 0.852 for 24-week remission).
UST was well-tolerated with an adverse event rate of 7.4%.
MRI-confirmed healing was modest at 14.2%.
Interpretation:
UST demonstrated clinically meaningful efficacy in managing pfCD.
Limitations:
Retrospective design may introduce bias.
Limited long-term follow-up data.
Conclusion:
UST demonstrated significant efficacy and safety outcomes in the management of pfCD, and the nomogram aids in individualized treatment decision-making.
