Observational studyOrphanet journal of rare diseases2022
Long-term clinical efficacy of topical treatment with recombinant human nerve growth factor in neurotrophic keratopathy: a novel cure for a rare degenerative corneal disease?
Observational study in Orphanet journal of rare diseases, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
15 citing papers in PubMed, 33 citations in OpenAlex.
- Recombinant human nerve growth factor (cenegermin) for moderate-to-severe dry eye: phase II, randomized, vehicle-controlled, dose-ranging trial.BMC ophthalmology · 2024Trial
- Management of ocular surface disease involving inflammation and persistent epithelial defects utilising various treatment modalities in the UK National Health Service (NHS).Eye (London, England) · 2026Article
- VEGF and Neurotrophins Interaction in the Retinal Neurovascular Unit Homeostasis: A Target for Ocular Disease Treatment and Management.Pharmaceutical research · 2026Review
- Challenge of Corneal Ulcer Healing: A Novel Conceptual Framework, the "Triad" of Corneal Ulcer Healing/Corneal Neovascularization/Intraocular Pressure, and Avascular Tendon Healing, for Evaluation of Corneal Ulcer Therapy, Therapy of Neovascularization, Glaucoma Therapy, and Pentadecapeptide BPC 157 Efficacy.Pharmaceuticals (Basel, Switzerland) · 2025Review
- Blood Derivatives in the Therapy of Ocular Surface Diseases.International journal of molecular sciences · 2025Review
- AQP5 Regulates Corneal Nerve Distribution Through NETRIN-1 and SLIT3.Investigative ophthalmology & visual science · 2025Article
- The Role of Nerve Growth Factor on the Ocular Surface: A Review of the Current Experimental Research and Clinical Practices.International journal of molecular sciences · 2025Review
- Corneal Neurotization, Recent Progress, and Future Perspectives.Biomedicines · 2025Review
- Long-term Nerve Regeneration in Diabetic Keratopathy Mediated by a Novel NGF Delivery System.Diabetes · 2025Article
- Efficient Production of Recombinant Human Brain-Derived Neurotrophic Factor inInternational journal of molecular sciences · 2024Article
- Review
- Adsorption of the rhNGF Protein on Polypropylene with Different Grades of Copolymerization.Materials (Basel, Switzerland) · 2023Article
- A, B, C's of Trk Receptors and Their Ligands in Ocular Repair.International journal of molecular sciences · 2022Review
- Bowman's membrane lenticule tuck-in: A new approach for the management of neurotrophic ulcers.Saudi journal of ophthalmology : official journal of the Saudi Ophthalmological SocietyArticle
- Utility of cenegermin for the management of neurotrophic keratopathy after penetrating keratoplasty.Oman journal of ophthalmologyArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundNeurotrophic keratopathy (NK) is a rare, degenerative ocular disease characterized by reduction or loss of corneal sensitivity and development of non-healing corneal epithelial defects and ulcers. Cenegermin, a recombinant human nerve growth factor (rhNGF) eye drop solution, is the first drug approved for the treatment of NK. The aim of our study is to evaluate the long-term efficacy of this innovative topical treatment in patients with NK.
methodsRetrospective, consecutive, observational case series study from a single-center setting (Department of Sense Organs, University Sapienza of Rome, Rome, Italy). 18 patients with diagnosis of stage 2 or 3 NK, treated with Cenegermin 20 mcg/ml eye drops were followed for up to 48 months. Recurrence of lesion during follow-up was evaluated at 12, 24, 36, and 48 months. In addition, corneal sensitivity, Schirmer tear test, and visual acuity (VA) were recorded at baseline, end of treatment, and at 12, 24, 36, and 48 months.
resultsThree patients experienced recurrence of persistent epithelial defects (PEDs) within 12 months and one patient experienced recurrence of a corneal ulcer within 36 months. Corneal sensitivity was significantly improved at all timepoints (P < 0.05). Significant improvements in visual acuity and tear production were seen at the completion of treatment as well as at 12, 24, and 36 months (P < 0.05) when compared to baseline.
conclusionsA single 8-week treatment regimen of Cenegermin eye drops has clinical efficacy that can persist for up to 48 months. The long-term clinical utility of treatment with Cenegermin for NK was demonstrated through the low rate of lesion recurrence along with improvements in corneal sensitivity and tear production.
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