PILOT TRIAL OF TOCILIZUMAB FOR REFRACTORY PATIENTS WITH ADULT ONSET STILL'S DISEASE

Abstract

Background Adult onset Still’s disease (AOSD) is a systemic inflammatory disorder with unknown etiology. As therapeutic agents, traditional disease modifying anti-rheumatic drugs (DMARDs) or biological agents are suggested for patients unresponsive to corticosteroid. Among important cytokines for the AOSD pathogenesis, IL-6 plays a pivotal role. Several case reports have suggested that tocilizumab, a humanized anti-interleukin (IL)-6 receptor antibody was effective for intractable AOSD.Objectives Here, we described the clinical course of refractory Korean patients treated with tocilizumab therapy.Methods The 8 patients who were refractory to DMARDs and/or etarnecept or dependent to corticosteroid were enrolled for tocilizumab therapy after informed contents. Tocilizumab at 8mg/kg was administered every 4 weeks.Results Six females and 2 males were treated with tocilizumab. The mean age was 33.8 (26-47) years old. The frequency of tocilizumab therapy was an average of 6.3 (2-13) times. Almost patients showed complete (50.0%) or partial (37.5%) response to tocilizumab therapy except one patient (Table 1). The clinical symptoms improved in 4 weeks (mean), erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP) improved in 6.3 weeks, respectively. Serum ferritin, IL-18 and IL-6 levels slowly improved within 9.3 weeks, 12.5 weeks and 9.3 weeks, respectively. Three patients discontinued tocilizumab therapy although they showed improvement of clinical symptoms; because of severe headache and chest discomfort at 12 weeks, neutropenia at 8 weeks, and severe hepatotoxicity at 8 weeks, respectively. Other adverse events such as mild dizziness, hair loss, weight gain and transient leucopenia were observed and those were tolerable during follow-up.