Cyclosporine A cationic emulsion (CsA CE) appears more effective than vehicle alone in treating pediatric patients with vernal keratoconjunctivitis, according to a study funded by the developer, Santen.
In a paper online December 16 in Ophthalmology, Dr. Dominique Bremond-Gignac of Paris V Descartes University, in France, and colleagues note that this potentially debilitating orphan disease more commonly affect boys than girls and is managed using off-label treatments. A Santen CsA emulsion is approved in Europe for severe keratitis in adults with dry eye disease.
Therapy for vernal keratoconjunctivitis
To investigate whether the agent might also provide effective therapy for vernal keratoconjunctivitis, 169 children and adolescents with a mean age of about 9 years about 80% of whom were boys were randomized to treatment with the emulsion or to vehicle alone.
They were given either one drop of the emulsion 0.1% (1 mg/mL) four times daily (high dose), or one drop twice daily along with one drop of the vehicle twice daily (low dose), or one drop of the vehicle four times a day.
At four months, the response was significantly greater in the high-dose group (57.1%) and the low-dose group (61.1%) than in the vehicle group (34.5%). Quality of life improved in all groups but was significantly higher in the high-dose group at most time points.
The most treatment-related adverse event was mild or moderate and the incidence was similar across groups, although instillation-site pain was greatest in the high-dose group. Best corrected distance visual acuity improved in all groups with the greatest gain being seen in the high-dose group.
Differences in least-squares means versus vehicle for the primary endpoint, a mean composite score that reflected corneal fluorescein staining (CFS), rescue medication use and corneal ulceration over the course of the study were significant for both doses. This was driven mainly by CFS score. The greatest improvement in the composite score was seen in the first month indicating that treatment benefits occurred rapidly.
Given these favorable findings, the researchers are further assessing the safety of the treatment over an eight-month follow-up period. The study was sponsored by Santen and Santen personnel were involved in most aspects of the study including data interpretation. Dr. Bremond-Gignac and other authors have relationships with Santen.