Phase 2 STELABEC Trial Supports Ustekinumab for the Treatment of Behçet Disease

The safety and efficacy of ustekinumab was determined in a phase 2 trial for the treatment of recurrent oral ulcers in Behçet disease.

Evidence supports the efficacy and safety of ustekinumab for the treatment of recurrent oral ulcers in Behçet disease (BD), according to study data published in Journal of the American Academy of Dermatology.

The current prospective, open-label, phase 2 study (STELABEC; Identifier: NCT02648581) included patients with BD with recurrent oral or genital ulcers (≥2 episodes within previous 3 months) that were refractory to colchicine. Patients received subcutaneous injections of ustekinumab 90 mg at baseline and at weeks 2, 4, and 16. Patients with BD that responded to treatment by week 24 received additional injections at weeks 28 and 40. Patients were followed-up for a total of 52 weeks.

The primary endpoint was change in number of oral ulcers at week 24. Secondary endpoints included the percentage of patients achieving partial and complete response at weeks 24 and 52. Complete response was defined as the absence of oral or genital ulcers; partial response was defined as a 50% or greater decrease in the number of oral ulcers from baseline.

A total of 15 patients with BD were enrolled in the study who received at least 1 ustekinumab injection. Mean age was 35.5±9.5 years; mean disease duration was 7.6±6.5 years; and 60% were men. At week 24, clinical response was observed in 11 patients: 9 (60%) with complete response and 2 with partial response. Of the 4 patients with treatment failure at week 24, 3 discontinued ustekinumab due to relapse or lack of efficacy and 1 was lost to follow-up. The mean number of oral ulcers per patient decreased from 2.1±1.3 at baseline to 0.3±0.5 at week 24 (P =.0017). Mean pain on the visual analog scale (0-100) decreased from 49.6±35.6 to 3.0±6.71 (P =.0005).

At week 52, treatment response was maintained in 10 patients, among whom 9 had a complete response. The mean number of oral ulcers at week 52 was 0.3±0.8 (P =.0002) vs baseline).

The most common side effects were gastrointestinal symptoms (33%), headache (33%), pruritus (27%), and upper respiratory tract infection (27%). None of the patients discontinued treatment due to adverse events.

Study limitations included the open-label, nonrandomized design, and the small study cohort.

Researchers concluded, “…ustekinumab is effective for the treatment of [patients with] BD with recurrent oral ulcers refractory to colchicine with a very good safety profile but larger randomized controlled trials are needed to confirm these findings.”

Disclosure: This research was supported by grants from Janssen. Please see the original reference for a full list of authors’ disclosures.


London J, Régent A, Dion J, et al. Efficacy and safety of ustekinumab in Behcet’s disease: results from the prospective phase 2 STELABEC trial. J Am Acad Dermatol. Published online December 2, 2021. doi:10.1016/j.jaad.2021.11.045