The interleukin-6 receptor antibody tocilizumab was effective for the treatment of adult-onset Still’s disease, but the primary efficacy end point was not met, according to results of a study published in the Annals of the Rheumatic Diseases.
Researchers conducted a multicenter, double-blind, phase 3, randomized controlled trial investigating the efficacy and safety of tocilizumab in 27 patients with adult-onset Still’s disease who were nonresponsive to glucocorticoids. Study participants were randomly assigned to receive tocilizumab 8 mg/kg administered intravenously every 2 weeks for a total of 12 weeks or placebo. The primary outcome measure was a 50% improvement in American College of Rheumatology criteria (ACR50) 4 weeks after starting therapy. Investigators also measured patient’s systemic response score and recorded any adverse events that occurred during treatment.
After statistical analysis, ACR50 response criteria at 4 weeks was met in 61.5% of patients in the tocilizumab group compared with 30.8% in the placebo group. However, the result was not statistically significant (95% CI, 31.6%-86.1%; P =.238). The least squares mean for difference in systemic feature score in the tocilizumab group at 12 weeks was −4.1 compared with −2.3 in placebo (P =.003).
Serious adverse events in patients receiving tocilizumab included aseptic necrosis in the hips, anaphylactic shock, and infections.
“The study suggests that tocilizumab is effective in adult-onset Still’s disease, although the primary endpoint was not met and solid conclusion was not drawn,” the researchers wrote.
Further studies are needed to fully establish whether tocilizumab is safe and effective for adults with glucocorticoid refractory Still’s disease.
Please see manuscript for a full list of author disclosures.
Kaneko Y, Kameda H, Ikeda K, et al. Tocilizumab in patients with adult-onset still’s disease refractory to glucocorticoid treatment: a randomised, double-blind, placebo-controlled phase III trial [published online October 2, 2018]. Ann Rheum Dis. doi: 10.1136/annrheumdis-2018-213920