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Updated: Jul 18, 2026

Monitoring Dynamic Growth of Retinal Vessels in Oxygen-Induced Retinopathy Mouse Model
Published on: April 2, 2021
Timed topical dexamethasone eye drops improve mitochondrial function to prevent severe retinopathy of prematurity
Hitomi Yagi1,2, Myriam Boeck1,3, Mariya Petrishka-Lozenska4
1Department of Ophthalmology, Boston Children's Hospital, Harvard Medical School, 3 Blackfan Circle, CLS 18, Boston, MA, 02115, USA.
Insights
Topical dexamethasone eye drops, given early, can prevent severe retinopathy of prematurity (ROP) in preterm infants. This treatment shows promise for preventing ROP neovascularization and associated vision loss.
Area of Science:
- Ophthalmology
- Neonatology
- Molecular Biology
Background:
- Pathological neovascularization in retinopathy of prematurity (ROP) leads to vision impairment in preterm infants.
- Current ROP treatments are reactive, costly, and carry risks of severe complications.
Purpose of the Study:
- To evaluate the preventative efficacy of early topical dexamethasone for retinopathy of prematurity.
- To investigate the mechanisms underlying dexamethasone's effects on retinal neovascularization.
Main Methods:
- Administered topical 0.1% dexamethasone eye drops to high-risk preterm infants and in a mouse model of ROP (oxygen-induced retinopathy - OIR).
- Assessed neovascularization and measured retinal mitochondrial gene and inflammatory marker expression in OIR.
- Investigated the role of mitochondrial ATP synthetase by blocking its function.
Main Results:
- Early topical dexamethasone prevented neovascularization in infants and suppressed it by 30% in OIR.
- Treatment timing was critical; pre-neovascularization treatment was effective, while later treatment showed limited efficacy or exacerbated disease.
- Dexamethasone increased mitochondrial gene expression and decreased inflammatory markers in OIR, with effects reversed by blocking ATP synthetase.
Conclusions:
- Optimally timed topical dexamethasone is a potential preventative strategy for ROP.
- Dexamethasone's efficacy involves modulation of mitochondrial function and inflammatory pathways.
- This approach offers a simple, preventative clinical option for severe ROP.
Abstract:
Pathological neovascularization in retinopathy of prematurity (ROP) can cause visual impairment in preterm infants. Current ROP treatments which are not preventative and only address late neovascular ROP, are costly and can lead to severe complications. We showed that topical 0.1% dexamethasone eye drops administered prior to peak neovessel formation prevented neovascularization in five extremely preterm infants at high risk for ROP and suppressed neovascularization by 30% in mouse oxygen-induced retinopathy (OIR) modeling ROP. In contrast, in OIR, topical dexamethasone treatment before any neovessel formation had limited efficacy in preventing later neovascularization, while treatment after peak neovessel formation had a non-statistically significant trend to exacerbating disease. Optimally timed topical dexamethasone suppression of neovascularization in OIR was associated with increased retinal mitochondrial gene expression and decreased inflammatory marker expression, predominantly found in immune cells. Blocking mitochondrial ATP synthetase reversed the inhibitory effect of dexamethasone on neovascularization in OIR. This study provides new insights into topical steroid effects in retinal neovascularization and into mitochondrial function in phase II ROP, and suggests a simple clinical approach to prevent severe ROP.

