Abstract
Background Topical NGF eye drops (Cenegermin) were approved in 2015 as an orphan drug for the treatment of neurotrophic keratopathy (NK). The active substance Cenegermin is a recombinant form of human NGF (nerve growth factor).
Objectives Presentation of efficacy and safety of Cenegermin for use in patients in an university real-life setting.
Material and Methods Retrospective study at the Köln University Eye Hospital from 2017 to 2019 with n = 11 eyes. Average follow-up was 13.5 ± 7.1 months. Reasons for treatment were neurotrophic keratopathy stage II and III, clinically mostly in combination with corneal neovascularization.
Results Seven eyes with a NK II and 4 eyes with a NK III with a median observation period of 13.6 months (range 1.2 – 20.3 months) from a total of 11 patients were included. The median patient age was 42.8 ± 23.6 years (range 18 – 75 years). Before the start of therapy, the median erosion area measured 3.1 ± 1.4 × 1.9 ± 1.1 mm and the median ulcer area had a size of 2.3 ± 1.1 × 2.1 ± 0.8 mm. After the start of therapy with Cenegermin (application 6×/day), the epithelial defect closed in all 11 study eyes (100%) within 4 – 12 weeks (mean: 49 d ± 9 d). In 9 out of 10 patients (90%) pre-existing corneal neovascularization regressed significantly (p < 0.001). Before the start of therapy, a value in the Luneau test of 2.9 ± 1.9 (minimum 1/6, maximum 4/6) was found and rose to a median value of 4.2 ± 1.7 (minimum 2/6, maximum 6/6) after 18 months (p = 0.015). Therapy with Cenegermin had no long-term effect on intraocular pressure: the pressure ranged between
13.2 ± 4.1 mmHg (minimum 8 mmHg, maximum 21 mmHg). Under therapy with NGF eye drops, 67% of the patients, after an initial decrease, showed a long-term improvement in visual acuity (BSCVA) from 0.72 ± 0.31 to 0.46 ± 0.29 logMAR after 18 months (p = 0.005). Relapses in form of a corneal erosion in the long-term follow-up were observed in only one eye. One patient had to stop therapy because of local pain, no other local and systemic side effects were observed.
Conclusion This real-life series on the use of Cenegermin in a university context shows a good effectiveness of the substance for epithelial closure in various underlying diseases (100% within 12 weeks). There is a long-term improvement in vision and corneal sensitivity. Larger real-life cohorts with various underlying diseases should follow.
Key words
neurotrophic keratopathy - cornea - orphan drugs - Cenegermin - Oxervate
