Pretreatment Histopathological Features are Associated with Ustekinumab Efficacy in Ulcerative Colitis: A...

Biologic therapy options for ulcerative colitis (UC) have increased in recent years. Although predicting treatment response may support personalized treatment strategies, the predictive value of histopathological features in UC remains unclear. This exploratory study aimed to evaluate the association between ustekinumab effectiveness and pre-treatment histopathological findings.

This single-center, retrospective, observational pilot study included patients with moderate-to-severe UC who were administered ustekinumab at the Kitasato University Hospital between April 2020 and March 2024. The endpoints were the relationship between pre-treatment histopathological findings, assessed using the Geboes score, and clinical efficacy at weeks 8 and 52, stratified by prior exposure to advanced therapies.

The study enrolled 33 patients. Among advanced therapy-naïve (AT-naïve) patients, responders at week 8 were significantly more likely to have low neutrophilic infiltration in the lamina propria than non-responders (66.7% vs. 0%, p = 0.03). Among advanced therapy-exposed (AT-exposed) patients, the degree of neutrophilic or eosinophilic infiltration in colonic tissue at week 8 did not differ significantly between responders and non-responders. Among AT-naïve patients, those who achieved clinical remission at week 52 tended to have mild eosinophilic infiltration in the lamina propria compared with non-responders (80.0% vs. 33.3%, p = 0.07). Among AT-exposed patients, the degree of neutrophilic or eosinophilic infiltration in colonic tissue at week 52 did not differ significantly between responders and non-responders.

In moderate-to-severe UC, pre-treatment mucosal histopathological assessment may serve as a treatment response marker. This information can guide development of personalized treatment strategies.