Author(s)Lynda Charters

Key Takeaways

• A pediatric subgroup from a pivotal phase 3 trial randomized 2:1 showed epithelium-on oxygen-enriched CXL improved Kmax at 6 months versus sham, indicating disease stabilization.
• Visual outcomes favored treatment, with mean BSCVA improving by 7.9 ETDRS letters and six eyes gaining ≥15 letters, while sham eyes declined overall.

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Phase 3 shows epi-on oxygen CXL flattens pediatric keratoconus, improves vision, and stays well tolerated—now commercially available.

(Image Credit: AdobeStock)

An approved noninvasive epithelium-on, oxygen-enriched corneal cross-linking (CXL) therapy for keratoconus, Epioxa (Glaukos Corporation), improved the corneal curvature and the best spectacle-corrected visual acuity (BSCVA) in a subset of pediatric patients in a phase 3 trial (NCT 03442751).

Michael B. Raizman, MD, from Ophthalmic Consultants of Boston and Tufts University School of Medicine, Boston, described the treatment at the 51^st annual meeting of the American Association for Pediatric Ophthalmology and Strabismus, March 18-22, in Boston.

Keratoconus progresses more rapidly in pediatric patients,^1,2 making early intervention critical to preserve visual function. Raizman and colleagues evaluated the efficacy and safety of epithelium-on oxygen-enriched corneal CXL in a sub-group analysis of 30 eyes of 22 pediatric patients aged 13 to 17 years. In this sub-group analysis of a pivotal Phase 3, prospective, multicenter, randomized, sham/placebo controlled trial, patients with keratoconus were randomized 2:1 to receive epithelium-on oxygen-enriched cross-linking (n=19) or sham/placebo (n=11).

The primary study endpoint was the mean change in the maximal corneal curvature (Kmax) from baseline to month 6. Investigators also evaluated the BSCVA and the development of any adverse events.

How did the treatment fare in the results?

Raizman reported that at month 6, the mean Kmax improved from baseline by 0.5 diopter (D) in CXL-treated eyes and worsened by 1.5 D in sham/placebo eyes, with a between-group difference of -2.0 D.

The mean BSCVA improved by 7.9 Early Treatment Diabetic Retinopathy Study (ETDRS) letters and worsened by 2.4 ETDRS letters in the sham/placebo group. He shared that six of the active-treatment eyes gained 15 or more letters of BSCVA versus zero in the sham/placebo group.

No serious adverse events occurred; the most common treatment-related ocular adverse events were conjunctival hyperemia, photophobia, hyperemia, and corneal opacity, with the majority being mild in severity.

The treatment is now commercially available.

Raizman emphasized that this CXL treatment is well-tolerated and had an acceptable safety profile in this subset of pediatric patients with keratoconus. He also underscored the importance of early detection of keratoconus and intervention with the goal of preserving visual function.

“These findings confirm what we suspected—that untreated pediatric patients can experience rapid advancement of the disease. Young patients are more likely to have rapid vision loss, if not treated. This demonstrates the importance of early intervention to stabilize the disease. Having a safe and effective non-invasive FDA-approved epithelium-on cross-linking therapy available may encourage more patients to be treated early, protecting vision for the long term in these young patients,” Raizman stated.

Raizman is a consultant to Glaukos Corporation. He has no other financial interest in Glaukos or in any other products. He does not accept fees for lectures, presentations, publications, or travel and does not participate in speaker’s bureaus.

References

1. Anitha V, Vanathi M, Raghavan A, Rajaraman R, Ravindran M, Tandon R. Pediatric keratoconus – Current perspectives and clinical challenges. Indian J Ophthalmol. 2021;69(2):214-25. doi:10.4103/ijo.IJO_1263_20.
2. Ferdi AC, Nguyen V, Gore DM, Allan BD, Rozema JJ, Watson SL. Keratoconus natural progression: a systematic review and meta-analysis of 11 529 eyes. Ophthalmology. 2019;126:935-45. doi:10.1016/j.ophtha.2019.02.029

First published – Phase 3 data show Epioxa improves corneal curvature and vision in pediatric keratoconus | Ophthalmology Times – Clinical Insights for Eye Specialists