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.
Highlights
Younger gestational age (GA) and lower birth weight (BW) are the most well-known risk factors associated with the development and severity of Retinopathy of prematurity (ROP) [6], and major clinical studies have demonstrated a gradual decline in GA and BW [7]
Because insulin-like growth factor 1 (IGF-1) levels in the serum and vitreous fluid are associated with the severity of retinopathy in diabetic retinopathy, Smith et al used oxygen-induced retinopathy (OIR) to analyze IGF-1 function and demonstrated that IGF-1 is required for pathological angiogenesis in ischemic retinal disease [240]
In our previous study using exhaustive genetic analysis, we showed that macrophage inflammatory protein-1β (MIP-1β) expression was significantly increased in the retinas of OIR mice [300,301]
Summary
Retinopathy of prematurity (ROP) is a representative vision-threatening disease in childhood that is characterized by abnormal retinal vessel growth at the boundary of the vascularized and avascular peripheral retinas of preterm infants [1,2]. Improved management of preterm infants, including respiratory support and postnatal nutrition intervention, has gradually reduced the incidence of ROP [4]. This improved maternal and neonatal management has resulted in an increase in the survival rate of extremely preterm infants with a high risk of developing ROP [5]. Clinical studies using optical coherence tomography (OCT), OCT angiography, and electroretinogram techniques have shown that the more severe the ROP, the more impaired the development of the macular structure, foveal vascular formation, and synapse. Preventing the progression of ROP is very important to protect the lifelong quality of vision of preterm infants
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