Recombinant human nerve growth factor in pediatric neuropathic keratopathy: a focus on corneal opacities, amblyopia, and visual outcomes

Abstract

To evaluate the effects of topical recombinant human nerve growth factor (rhNGF) in pediatric neurotrophic keratopathy (NK), with specific attention to corneal opacities, amblyopia, and visual outcomes. Methods: This retrospective study included 10 pediatric patients with NK refractory to conventional therapy who received topical rhNGF for 8 weeks. The cohort had complex multisystem comorbidities and frequently required bedside examinations with limited cooperation; therefore, standardized imaging, quantitative grading, and clinic-based photography were often not feasible. Results: Mean age was 5.1 ± 4.8 years, and 80% of patients were within the amblyogenic period. Mean follow-up was 71.7 ± 45.6 weeks. All patients presented with persistent epithelial defects and corneal opacities, central in 80% of cases. Baseline best-corrected visual acuity was 0.75 ± 0.23 logMAR. Complete epithelial healing was achieved in 9 of 10 patients (90%), with clinician-assessed qualitative improvement in corneal opacity appearance in 6 patients (60%) within 8 weeks. Baseline best-corrected visual acuity did not significantly improve at week 8 or at final follow-up. Six patients demonstrated asymmetric astigmatism greater than +1.50 diopters. One patient with advanced stage 3 NK did not achieve epithelial healing and had limited follow-up because of international referral. Conclusions: Topical rhNGF was effective and well tolerated in pediatric NK, producing rapid epithelial healing and qualitative improvement in corneal opacity appearance. Limited visual recovery likely reflects preexisting central stromal scarring and amblyopia, emphasizing the importance of early NK recognition and timely rhNGF initiation to preserve visual potential during childhood visual development and long-term clinical decision making globally care.