Ustekinumab for Intestinal Behçet's Disease: The First Cohort with a Novel Therapy

Evidence-based treatments for intestinal Behçet's disease (BD) remain limited. There is an unmet need for the development of effective therapies for refractory cases. We aimed to evaluate the safety and efficacy of ustekinumab, an anti-interleukin (IL)-12/23 monoclonal antibody, for intestinal BD treatment.

A retrospective cohort study was conducted on intestinal BD patients who received ≥ 1 dose of ustekinumab and had ≥ 3-month follow-up. The primary outcome was clinical response at 3 months. Secondary outcomes included clinical remission at 3, 6, and 12 months, endoscopic/radiologic/ultrasound responses and remissions, mucosal healing, and safety profiles.

Sixteen patients were enrolled, among whom 87.5% and 68.8% achieved clinical response and clinical remission at 3 months. At 6 months, 72.7% (8/11) of patients achieved clinical response and 45.5% (5/11) achieved clinical remission. In addition, 80.0% (8/10) had endoscopic response, 70.0% (7/10) achieved mucosal healing, 87.5% (7/8) had radiologic response, and 55.6% (5/9) had ultrasound response, respectively. At 12 months, 85.7% (6/7) patients achieved both clinical response and remission, with 100% endoscopic response, 83.4% mucosal healing, 100% (5/5) ultrasound response, 40.0% (2/5) ultrasound remission, 66.7% (2/3) radiologic response and remission. All five steroid users at baseline discontinued corticosteroids by 6 months. Only 18.8% (3/16) experienced self-limiting adverse events, and no patient discontinued treatment due to adverse events.

Ustekinumab appears to be an effective, well-tolerated treatment for intestinal BD, particularly for refractory cases.