A Randomized, Controlled Trial of Cyclosporine A Cationic Emulsion in Pediatric Vernal Keratoconjunctivitis: The VEKTIS Study

BACKGROUND: Vernal keratoconjunctivitis (VKC) is a chronic, allergic, and potentially severe ocular disease affecting children and adolescents that can lead to impaired quality of life (QoL) and loss of vision. PURPOSE: This study evaluated the efficacy and safety of an investigational therapy for severe VKC, cyclosporine A cationic emulsion (CsA CE), an oil-in-water emulsion with increased bioavailability versus conventional CsA formulations. DESIGN: VEKTIS is a phase 3, multicenter, double-masked, vehicle-controlled trial. PARTICIPANTS: Pediatric patients (4-18 years) with active severe VKC (grade of 3 or 4 on the Bonini severity scale) and severe keratitis (corneal fluorescein staining [CFS] score of 4 or 5 on the modified Oxford scale). METHODS: 169 patients were randomized to CsA CE 0.1% (1 mg/mL) eye drops four times daily (QID, high-dose), CsA CE twice daily (BID, low-dose) + vehicle BID, or vehicle QID for 4 months. MAIN OUTCOME MEASURES: The primary endpoint was a mean composite score that reflected CFS, rescue medication use (dexamethasone 0.1% QID), and corneal ulceration over the 4 months. RESULTS: Differences in least-squares means versus vehicle for the primary endpoint were statistically significant for both the high-dose (0.76, P=0.007) and the low-dose group (0.67, P=0.010), with treatment effect mainly driven by CFS score. Significant differences were found between both active treatment groups and vehicle for use of rescue medication. VKC symptoms and patient QoL (assessed by visual analogue scale and the Quality of Life in Children with Vernal Keratoconjunctivitis [QUICK] questionnaire) improved in all 3 groups, with significant improvements for high-dose CsA CE versus vehicle. CONCLUSIONS: The efficacy of high-dose CsA CE in improving keratitis, symptoms, and QoL for those with severe VKC was demonstrated in these study patients. In addition, in this study cohort, CsA CE was well-tolerated.