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Update on Anti-Vascular Endothelial Growth Factor Safety for Retinopathy of Prematurity
Ana Bety Enríquez1, Robert L Avery2, Caroline R Baumal1
1Department of Ophthalmology, New England Eye Center, Tufts University School of Medicine, Boston, MA.
Insights
Anti-vascular endothelial growth factor (anti-VEGF) agents offer a simpler treatment for retinopathy of prematurity (ROP). This review examines the current evidence on the safety of anti-VEGF for ROP, addressing concerns about long-term outcomes.
Area of Science:
- Ophthalmology
- Neonatology
- Pediatric Medicine
Background:
- Retinopathy of prematurity (ROP) is a major cause of childhood blindness in preterm infants.
- Current standard treatment, peripheral retinal ablation, has limitations including specialized requirements and suboptimal outcomes.
- Anti-vascular endothelial growth factor (anti-VEGF) agents are emerging as an alternative treatment for ROP.
Purpose of the Study:
- To review current evidence on the systemic and ocular safety of anti-VEGF treatment for retinopathy of prematurity.
- To address the growing use of off-label anti-VEGF agents in ROP management.
- To evaluate potential long-term outcomes associated with anti-VEGF therapy in ROP.
Main Methods:
- Systematic review of recent scientific literature.
- Analysis of studies reporting on anti-VEGF treatment for retinopathy of prematurity.
- Evaluation of data concerning both systemic and ocular safety profiles.
Main Results:
- Increased worldwide use of anti-VEGF agents for ROP treatment.
- Continued off-label application of anti-VEGF therapy.
- Limited data on long-term safety and potential adverse outcomes.
Conclusions:
- Anti-VEGF agents represent a promising, simpler alternative for ROP treatment.
- Further research is crucial to fully understand the long-term safety and efficacy of anti-VEGF in ROP.
- Addressing the knowledge gaps in safety is essential for informed clinical practice.
Abstract:
Retinopathy of prematurity (ROP) is a neovascular retinal disorder that occurs in infants born prematurely. Nowadays, ROP constitutes a leading cause of childhood blindness worldwide and for decades the standard of care has involved peripheral retinal ablation. However, this type of treatment requires the use of specialized equipment by well-trained physicians, has been associated with poor structural and visual outcomes in some preterm infants, and despite its adequate application, some cases of ROP may continue to progress. Therefore, the need for simpler and more efficient strategies made anti-vascular endothelial growth factor (anti-VEGF) medications an appealing option for treatment. Recently, the use of anti-VEGF agents for ROP has increased worldwide; nevertheless, this practice remains off-label, and there is a lack of information regarding its safety profile and the possibility of unfavorable long-term outcomes causes the utmost concern. This review updates the recent evidence regarding the systemic and ocular safety of anti-VEGF treatment for ROP.

