Cenegermin treatment for pediatric neurotrophic keratopathy: a systematic review.

Objective: This systematic review evaluates the efficacy and safety of cenegermin in pediatric neurotrophic keratopathy (NK), assessing treatment outcomes, predictors of efficacy, and adverse effects.

Methods: Adhering to PRISMA guidelines, we reviewed studies from Embase, MEDLINE, HealthSTAR, and other databases up to July 28, 2024. Studies were included if they involved pediatric patients (≤18 years) treated with cenegermin for NK. Two reviewers independently screened and extracted data, assessing the risk of bias using Joanna Briggs Institute tools.

Results: Of the 20 articles reviewed, 8 case reports or series met inclusion criteria, encompassing 21 pediatric cases (33% female), with an average age of 5.87 ± 3.05 years. Etiologies included congenital conditions, radiotherapy, and infections. Cenegermin was administered as a 0.002% drop 6 times daily for 8 weeks. Treatment was typically initiated after the failure of conventional methods. Improvements in corneal transparency were observed in all cases, with visual acuity improved in 4 out of 6 studies reporting this metric. Factors associated with improvement in corneal transparency were the severity of preexisting corneal scarring and ocular surface instability.

Conclusions: Cenegermin is a viable treatment option for pediatric NK, showing efficacy in improving corneal transparency and, to a lesser extent, visual acuity. However, its adoption is constrained by limited pediatric data, cost, and potential side effects. Further research is needed to establish long-term safety and effectiveness, and to optimize treatment protocols for this vulnerable population.