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Cenegermin treatment for pediatric neurotrophic keratopathy: a systematic review
Daiana R Pur1, Fady Sedarous2, Asim Ali3
1Department of Surgery, Division of Ophthalmology, McMaster University, Hamilton, ON, Canada.
Insights
Cenegermin shows promise for treating pediatric neurotrophic keratopathy (NK), improving corneal transparency and vision. Further research is needed to confirm its long-term safety and effectiveness in children.
Area of Science:
- Ophthalmology
- Regenerative Medicine
Background:
- Neurotrophic keratopathy (NK) is a degenerative corneal disease affecting vision.
- Pediatric NK cases are rare and pose unique treatment challenges.
Purpose of the Study:
- To systematically review the efficacy and safety of cenegermin for treating pediatric NK.
- To identify predictors of treatment success and adverse effects.
Main Methods:
- PRISMA-guided systematic review of Embase, MEDLINE, and HealthSTAR databases up to July 2024.
- Inclusion of studies on pediatric patients (≤18 years) treated with cenegermin for NK.
- Data extraction and risk of bias assessment by two independent reviewers.
Main Results:
- Eight case reports/series involving 21 pediatric patients were analyzed.
- Cenegermin (0.002% drops, 6x daily for 8 weeks) improved corneal transparency in all cases.
- Visual acuity improved in 4/6 studies; corneal scarring and ocular surface instability predicted better outcomes.
Conclusions:
- Cenegermin is a potential treatment for pediatric NK, enhancing corneal transparency and visual acuity.
- Limited pediatric data, cost, and side effects hinder widespread adoption.
- Further research is essential to establish long-term safety, efficacy, and optimal protocols for children.
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.
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