Abstract
Purpose:
To assess the efficacy and safety of tocilizumab, a monoclonal antibody against the IL-6 receptor, in the treatment of JIA-associated uveitis refractory to other modes of systemic immunosuppression.
Methods:
We received IRB and FDA approval to recruit 5 patients into a 16 month open-label study of the effectiveness of monthly tocilizumab 8-10 mg/kg infusions for the treatment of refractory juvenile idiopathic arthritis (JIA)-associated uveitis. Patients characterized as refractory had failed treatment with corticosteroids and at least one standard immunosuppressive. Initial treatment outcome was ascertained at 16 weeks after study initiation by ability to control anterior chamber intraocular inflammation and without need to increase adjunctive systemic immunosuppressive therapy and utilizing no more than two drops of prednisolone acetate daily. Secondary outcome measures included visual acuity, macular edema as measured by ocular coherence tomography (OCT), and counts of swollen or painful joints. Patients who meet criteria for clinical success at 16 weeks will be allowed to complete up to 52 additional weeks in the study with final treatment outcome ascertained at week 68.
Results:
Two patients have enrolled in this study at the time of this abstract submission. One met the primary endpoint of control of inflammation with steroid tapering by week 16, and has continued in the study. The second patient failed to gain control of bilateral anterior chamber inflammation with high dose corticosteroids two tocilizumab infusion, and was withdrawn from the study at week 8. Interestingly, both patients had OCT-documented macular edema at study outset and had resolution of macular edema on study therapy.
Conclusions:
Our preliminary results suggest that tocilizumab may be effective for the treatment of uveitic macular edema in patients with refractory JIA-associated uveitis, and was beneficial in achieving and maintaining control of inflammation in one of two early study enrollees. No treatment-limiting toxicity was noted. Enrollment is ongoing and further study is required to define patient populations who may derive the most benefit from tocilizumab therapy for JIA-associated uveitis.