PURPOSE: To assess the safety and efficacy of cyclosporine A cationic emulsion (CsA
CE) 0.1% eye drops in pediatric patients with severe active vernal keratoconjunctivitis
(VKC). DESIGN: Multicenter, double-masked, randomized controlled trial 8-month safety
analysis. METHODS: Of 169 patients (age range, 4-17 years) initially randomized in
the 4-month VErnal KeratoconjunctiviTIs Study (VEKTIS), 142 entered the 8-month follow-up
period during which CsA CE patients remained on their original regimen (CsA CE 4 times
daily [QID, high-dose] or CsA CE twice daily [BID, low-dose] + vehicle BID) and vehicle
patients were allocated to one of these 2 active regimens. Main outcome measures were
safety, including treatment-emergent adverse events, and efficacy, including corneal
fluorescein staining (CFS) score. RESULTS: Improvements in CFS score, rescue medication
use, key VKC symptoms (photophobia, tearing, itching, and mucous discharge), and quality
of life (QoL) assessed by QUICK questionnaire observed with CsA CE compared with vehicle
during the 4-month evaluation period remained stable during the 8-month follow-up
period, with the high-dose regimen continuing to provide greater benefits in most
efficacy measures. CsA CE was well tolerated. Treatment-related treatment-emergent
adverse events during the 12-month study were reported in 15 (20.8%) and 11 (15.7%)
of the CsA CE high-dose and low-dose patients, respectively, most commonly instillation
site pain (13.9% and 7.1%, respectively). Laboratory data, vital signs, slit lamp
examination, best-corrected distance visual acuity, and intraocular pressure raised
no safety concerns. CONCLUSIONS: Improvements in keratitis, symptoms, and QoL achieved
after CsA CE treatment for 4 months remained stable over the 8-month follow-up period.
CsA continued to maintain a favorable safety profile. (C) 2019 The Authors. Published
by Elsevier Inc.
