Investigative Ophthalmology & Visual Science Cover Image for Volume 61, Issue 7
June 2020
Volume 61, Issue 7
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ARVO Annual Meeting Abstract  |   June 2020
Efficacy and Tolerability of Cenegermin for Stage 1 Neurotrophic Keratopathy
Author Affiliations & Notes
  • Leyla Yavuz Saricay
    Department of Ophthalmology, Center for Translational Ocular Immunology,Tufts Medical Center, Tufts School of Medicine, Boston, Massachusetts, United States
    Department of Ophthalmology, Cornea Service New England Eye Center,Tufts Medical Center, Tufts School of Medicine, Boston, Massachusetts, United States
  • Betul Bayraktutar
    Department of Ophthalmology, Center for Translational Ocular Immunology,Tufts Medical Center, Tufts School of Medicine, Boston, Massachusetts, United States
    Department of Ophthalmology, Cornea Service New England Eye Center,Tufts Medical Center, Tufts School of Medicine, Boston, Massachusetts, United States
  • Jonathan Lilley
    Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelpia, Pennsylvania, United States
  • Mina Massaro-Giordano
    Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelpia, Pennsylvania, United States
  • Pedram Hamrah
    Department of Ophthalmology, Center for Translational Ocular Immunology,Tufts Medical Center, Tufts School of Medicine, Boston, Massachusetts, United States
    Department of Ophthalmology, Cornea Service New England Eye Center,Tufts Medical Center, Tufts School of Medicine, Boston, Massachusetts, United States
  • Footnotes
    Commercial Relationships   Leyla Yavuz Saricay, None; Betul Bayraktutar, None; Jonathan Lilley, None; Mina Massaro-Giordano, Celularity (C), Dompe (C), Lynthera (C), PRN (I), Verily (C); Pedram Hamrah, Dompe (C), Dompe (S), Neutoptiks (C), Noveome (C), Noveome (S)
  • Footnotes
    Support  NIH-1R61NS113341-01, Research to prevent blindness RPBChallenge Grant ,Massachusetts Lions Eye Research Fund,Inc.,Tufts Institutional Support.
Investigative Ophthalmology & Visual Science June 2020, Vol.61, 374. doi:
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    • Get Citation

      Leyla Yavuz Saricay, Betul Bayraktutar, Jonathan Lilley, Mina Massaro-Giordano, Pedram Hamrah; Efficacy and Tolerability of Cenegermin for Stage 1 Neurotrophic Keratopathy. Invest. Ophthalmol. Vis. Sci. 2020;61(7):374.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Topical recombinant human nerve growth factor (rhNGF, cenegermin-bkbj, Oxervate 0.002%, Dompé U.S. Inc) has been approved by FDA in all stages of neurotrophic keratopathy (NK). Only results for stage II and III NK have been reported so far. Our aim was to evaluate its efficacy and tolerability in patients with stage 1 NK in an initial case series.

Methods : Patients with stage 1 NK with diffuse corneal fluorescein staining (CFS) and decreased corneal sensation, who did not respond to conventional therapy and were treated with rhNGF were included (n=14). Demographic features, best corrected visual acuity (BCVA), CFS using Oxford scale, nerve density analyzed by in vivo confocal microscopy (HRT3/RCM) (n=5) at baseline and after treatment, and side effects of rhNGF treatment were recorded. Nerve density of patients was compared to 22 age- and sex-matched healthy reference controls.

Results : Fourteen eyes of 11 patients were included. The mean age was 64.9±13 years in patients and 55.6±9.0 years in reference controls (p=0.07). The etiologies of NK included Sjögren's syndrome (33%), herpes simplex keratitis (13%), laser-assisted in situ keratomileusis (6%), and penetrating keratoplasty (6%), with 40% being multifactorial. Thirteen eyes were treated for 8 weeks and 1 patient for 16 weeks with rhNGF six times a day. The mean BCVA was 20/30 and the mean CFS score was 3.6±1.0 at baseline. Following treatment, mean BCVA improved to 20/25 (p=0.02) and the mean CFS score decreased to 1.1±0.8 (p<0.001). The mean total, main and branch nerve densities at baseline were 5,274.4±4,013.6mm/mm2, 3,563.3±2,513.8 and 1,711.1±1,743.3 in NK patients and 23,886.1±3,514.8, 10,798±1,877.0 and 13,088±2,723.0 in reference controls, respectively (p<0.001). Following treatment, the mean total, trunk and branch nerve densities of patients increased to 8,459.7±3,037.2 (p=0.13), 4,905.9±1,400.5 (p=0.12), and 3,553.7±1,843.7 (p=0.11), but did not reach statistical significance. Ten patients reported mild to moderate discomfort and 1 patient discontinued rhNGF temporarily, but resumed thereafter.

Conclusions : Topical rhNGF improved ocular surface findings and vision in stage 1 NK patients. While nerve densities increased after treatment, it was not significant in our small pilot study. Topical rhNGF seems to be an effective and well-tolerated treatment for epithelial healing in stage 1 NK, but may result in ocular discomfort while on therapy.

This is a 2020 ARVO Annual Meeting abstract.

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