Composite Endpoint Outcomes of the STOP-Uveitis Study: Evaluating the Safety, Tolerability, and Efficacy of Tocilizumab in Patients with Noninfectious Uveitis.

Abstract

Purpose : To report the efficacy of two different doses of intravenous (IV) infusions of Tocilizumab (TCZ), in eyes with non-infectious intermediate, posterior or pan-uveitis using a composite endpoint scoring system.

Methods : STOP-Uveitis is a randomized, multi-center clinical trial evaluating the role of intravenous infusions of TCZ in patients with non-infectious uveitis. Patients were randomized (1:1) into one of 2 treatment groups (Group 1:4mg/kg and Group 2:8mg/kg). Infusions were given every 4 weeks in both groups until month 6 (primary endpoint). Efficacy outcome was ascertained by a composite endpoint scoring system consisting of: 1) visual acuity; 2) intraocular inflammation as assessed by vitreous haze; 3) central retinal thickness; 4) posterior segment inflammatory outcome assessed by fluorescein angiography, and 5) tapering of steroid dose. Each component of the grading system was assigned one of three possible outcomes: (+1) improvement, (-1) worsening, or (0) no-change based on specific criteria (Table 1). A positive clinical response was defined by improvement in at least one parameter and worsening in none. A negative clinical response was defined as worsening of any parameter. A stable clinical response was defined as neither improvement nor worsening in any of the parameters. Number of patients achieving successful clinical response was calculated and compared between the study groups. Statistical significance was set at p<0.05.

Results : Thirty-seven patients were enrolled in STOP-Uveitis. At month 6, 31 (83.8%) subjects demonstrated a positive clinical response (Group-1=14 (77.8%) and Group-2=17 (89.5%)). Three (8.1%) subjects (Only in Group-1) met the criteria for treatment failure. Three (8.1%) subjects showed a stable clinical response (Group-1=1 and Group-2=2). The difference in clinical response between the two groups was not significant (p>0.05). Figure 1 demonstrates the percentage of patients showing outcomes of various components of the composite endpoint scoring system.

Conclusions : Both high and low dose IV TCZ were effective in either improving or maintaining stability in various components of the composite end point scoring system. A composite scoring system as used in this study may be a better measure to assess efficacy outcomes as compared to only vitreous haze score.

This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.

 

Table 1

 

Figure 1