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Purpose: To determine the efficacy and safety of nerve growth factor cenegermin for treatment of limbal stem cell deficiency (LSCD) associated with neurotrophic keratopathy (NK).

Methods: Patients diagnosed with LSCD with NK who previously failed conventional treatment were enrolled in this prospective open-label pilot study. All participants were treated with cenegermin for 8 weeks. The primary aim was to determine if there is reduction in the area of abnormal epithelium following treatment. Other factors evaluated were corneal sensation, visual acuity (VA), and LSCD severity.

Results: Six eyes of five patients were included in the study. Cenegermin decreased the area of abnormal corneal epithelium in five of six eyes, measuring 73% of total corneal area at the initial visit and 48% at the final visit (P=0.036). Corneal sensation increased in all patients, Cochet-Bonnet aesthesiometry measured 14.7mm at the initial visit and 26.7mm at final visit (P=0.009). Visual acuity showed overall improvement, with initial logMAR VA of 1.67 and final logMAR VA of 1.19 (P=0.045). VA improved in four out of six eyes. LSCD scoring improved using the Aravena scoring system, though this recovery was not statistically significant (P=0.14). One patient with a persistent epithelial defect at baseline showed resolution with cenegermin. No patient withdrew from the study due to adverse side effects.

Conclusions: Cenegermin improved cornea epithelial condition, visual acuity, and corneal sensation in patients with LSCD and NK who had failed prior treatment. Further studies are necessary to better understand the anatomical changes and to confirm our results with a larger randomized control trial.