Objective To evaluate long-term efficacy and safety of ustekinumab through 2 years in patients with active systemic lupus erythematosus (SLE). Methods This was a placebo-controlled (Week24) phase 2 study in 102 patients with seropositive active SLE. Patients were randomized to ustekinumab (~6 mg/kg single IV infusion, then 90 mg SC every 8 weeks) or placebo, added to background therapy. Placebo patients initiated ustekinumab (90mg SC Q8W) at Week24. Patients could enter an optional open-label study extension after Week40 (final ustekinumab administration at Week104). Efficacy assessments included SRI-4, SLEDAI-2K, PGA, and CLASI. Observed data are reported for the extension period. The final efficacy assessment was at Week112; safety was monitored through Week120. Results In this subset of patients who entered the study extension, 24 in the ustekinumab group and 14 in the placebo-crossover group completed study treatment. At Week112, 79% and 92%, respectively, had an SRI-4 response, 92% in both groups had ≥4-point improvement from baseline in SLEDAI-2K score, 79% and 93%, respectively, had ≥30% improvement from baseline in PGA, 86% and 91%, respectively, had ≥50% improvement in active joint (pain and inflammation) count, and 79% and 100%, respectively, had ≥50% improvement in CLASI activity score. No deaths, malignancies, opportunistic infections, or tuberculosis cases occurred. Safety events were consistent with the known ustekinumab safety profile. Conclusion In the 46 patients who entered the voluntary extension of this phase 2 study, clinical benefit in global and organ-specific SLE-activity measures was observed with ustekinumab through 2years with no new or unexpected safety findings. clinicaltrials.gov: NCT02349061
Long‐term safety and efficacy of anifrolumab in adults with systemic lupus erythematosus: results of a phase 2 open‐label extension study