Development and Validation of a Clinical Decision Support Tool to Predict Disease Progression in Crohn's Disease Treated with Ustekinumab.

Background/Objectives: Ustekinumab (UST) is an effective and safe drug for treating Crohn's disease (CD), but data on disease progression after UST treatment is limited. This study aimed to develop a clinical decision support tool (CDST) to identify Chinese patients with CD less likely to experience disease progression during UST treatment. Methods: A multicenter, retrospective observational study was conducted among Chinese patients with CD who started UST treatment between 1 May 2020 and 20 October 2022. Baseline characteristics, defined as the measurements taken closest to, and prior to, the first dose of UST, were collected. Disease progression, defined as CD-related surgery, hospitalization, and complications, was evaluated by week 52 (±4 weeks). Predictors were identified using logistic regression, and a UST-specific CDST (UST-CDST) was developed. The UST-CDST was then internally and externally validated using the area under the receiver operating characteristic curve (AUC). Results: Among 602 enrolled patients, 533 were included in the analysis. Four factors were suggestively associated with disease progression: prior biologics usage, baseline disease severity, baseline C-reactive protein, and baseline hemoglobin. The prediction model demonstrated an AUC of 0.88 in internal validation and 0.66 in external validation. The UST-CDST effectively stratified patients into low-risk or high-risk groups for disease progression within one year. Conclusions: A UST-CDST was developed and validated to identify patients with CD less or more likely to experience disease progression around week 52 under UST therapy. The scoring system promises to facilitate clinical decision-making and personalized treatment.