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Updated: Nov 20, 2025
Retinal Pathophysiological Evaluation in a Rat Model
Published on: May 6, 2022
Translational Research in Retinopathy of Prematurity: From Bedside to Bench and Back Again
Mitsuru Arima1,2, Yuya Fujii1, Koh-Hei Sonoda1
1Department of Ophthalmology, Graduate School of Medical Sciences, Kyushu University, Fukuoka 8128582, Japan.
Insights
Retinopathy of prematurity (ROP) is a major cause of childhood blindness. While anti-VEGF therapy is effective, new, safe treatments are needed for this premature infant disease.
Area of Science:
- Ophthalmology
- Neonatology
- Vascular Biology
Background:
- Retinopathy of prematurity (ROP) is a significant cause of childhood blindness in preterm infants.
- Oxygen levels are strongly linked to ROP development, leading to the use of oxygen-induced retinopathy (OIR) animal models.
- Anti-vascular endothelial growth factor (VEGF) agents are now a first-line treatment for ROP.
Purpose of the Study:
- To review the evolution of retinopathy of prematurity research from basic science to clinical application.
- To discuss the efficacy and limitations of current anti-VEGF therapies for ROP.
- To identify novel therapeutic targets for ROP.
Main Methods:
- Review of existing literature on ROP pathogenesis and treatment.
- Analysis of clinical trial data for anti-VEGF agents.
- Discussion of emerging biotechnological advancements in ROP research.
Main Results:
- Anti-VEGF therapy is effective and minimally invasive for ROP but has long-term safety concerns and recurrence risks.
- There is a clear need for novel, safe, and minimally invasive treatments for ROP.
- Biotechnology advancements are driving translational research for new ROP therapies.
Conclusions:
- Current anti-VEGF treatments for retinopathy of prematurity have limitations, highlighting the need for alternative therapies.
- Ongoing research focuses on identifying and developing novel molecular targets for ROP.
- Future ROP therapies aim to be safe, minimally invasive, and address unmet medical needs.
Abstract:
Retinopathy of prematurity (ROP), a vascular proliferative disease affecting preterm infants, is a leading cause of childhood blindness. Various studies have investigated the pathogenesis of ROP. Clinical experience indicates that oxygen levels are strongly correlated with ROP development, which led to the development of oxygen-induced retinopathy (OIR) as an animal model of ROP. OIR has been used extensively to investigate the molecular mechanisms underlying ROP and to evaluate the efficacy of new drug candidates. Large clinical trials have demonstrated the efficacy of anti-vascular endothelial growth factor (VEGF) agents to treat ROP, and anti-VEGF therapy is presently becoming the first-line treatment worldwide. Anti-VEGF therapy has advantages over conventional treatments, including being minimally invasive with a low risk of refractive error. However, long-term safety concerns and the risk of late recurrence limit this treatment. There is an unmet medical need for novel ROP therapies, which need to be addressed by safe and minimally invasive therapies. The recent progress in biotechnology has contributed greatly to translational research. In this review, we outline how basic ROP research has evolved with clinical experience and the subsequent emergence of new drugs. We discuss previous and ongoing trials and present the candidate molecules expected to become novel targets.
