This study evaluated the efficacy and safety of an investigational cationic emulsion cyclosporine A (CsA CE) for severe vernal keratoconjunctivitis (VKC). The novel CsA CE is an oil-in-water emulsion with increased bioavailability compared with conventional CsA formulations.
Study design
This phase 3, multicenter, double-masked, vehicle-controlled trial evaluated 169 pediatric patients with active severe VKC and severe keratitis. Participants were randomized to 3 groups: CsA CE 0.1% (1 mg/ml) eye drops 4 times daily (high dose), CsA CE twice daily (low dose) plus vehicle twice daily or vehicle 4 times daily for 4 months.
The primary endpoint was a mean composite score that reflected corneal fluorescein staining (CFS), rescue medication use (dexamethasone 0.1% 4 times daily) and corneal ulceration over the 4-month study period.
Outcomes
Both the high-dose (P=0.007) and low-dose (P=0.010) groups performed significantly better than vehicle, and the treatment effect appeared to be mainly driven by the CFS score.
Significant differences were found between both active treatment groups and the vehicle with regards to the use of rescue medication. Vernal keratoconjunctivitis symptoms and patient quality of life, assessed by visual analog scale and the Quality of Life in Children with Vernal Keratoconjunctivitis questionnaire, improved in all 3 groups. The most significant improvements were observed in the high-dose CsA CE, compared with vehicle.
Limitations
The study enrolled only patients with severe VKC. Moreover, the modified Oxford scoring system used to assess CFS data was developed for use in patients with dry eye disease, rather than VKC.
Clinical significance
This novel cationic emulsion of cyclosporine A led to a significant reduction of VKC signs during a 4-week treatment period, particularly in patients with severe keratitis. The findings suggest this therapy is potentially beneficial for this patient population.