• By Rishabh Jain; Ashiyana Nariani, MD, MPH
    Cornea/External Disease, Pediatric Ophth/Strabismus

    Review of: Aftermarket effects of cenegermin for neurotrophic keratopathy in pediatric patients

    Hatcher J, Soifer M, Morales N, et al. The Ocular Surface, July 2021

    Investigators examined the use of cenegermin eyedrops for neurotrophic keratopathy (NK) in children.

    Study design

    This retrospective study was conducted at Duke University Eye Center, Vanderbilt Eye Institute and the University of Chicago Medical Center. Eight children (ages 2–18 years) received 6 daily drops of cenegermin for 8 weeks; 5 patients completed the full regimen. The mean time from diagnosis of NK to the beginning of cenegermin therapy was 11 months. The study captured demographics and health factors including age, NK stage and severity, ocular disease and surgical history, diagnostic testing, esthesiometry measurements, visual acuity and stromal scarring.


    Five patients had clinical improvement due to cenegermin, with 2 experiencing improvement in visual acuity. However, none of the patients had improvement in corneal sensation, which the authors attributed to inconsistent documentation between the various medical centers involved.

    Of the 3 patients who discontinued treatment, one had progression in corneal thinning that led to ulceration and descemetocele formation, requiring emergent keratoplasty. Another patient discontinued therapy after experiencing ocular pain, nightmares and difficulty sleeping. Therapy was accidently terminated in the third patient.


    The study has a small sample size of only 8 patients.

    Clinical significance

    To date, this is the largest clinical series of pediatric NK patients receiving cenegermin treatment. Although the drug has been FDA-approved for patients 2 years or older, aftermarket trials of cenegermin do not include many patients younger than 50 years and there are no major trials focused on pediatric cohorts. Findings from this study suggest that cenegermin is safe and effective for a wide range of etiologies, including congenital NK, ocular trauma, herpes simplex virus infection and base skull surgery.