Efficacy and Vascular Redevelopment of Anti-vascular Endothelial Growth Factor Therapy for Stage 2 early-diagnosed Familial Exudative Vitreoretinopathy

Purpose: To report the efficacy of anti-vascular endothelial growth factor (VEGF) therapy in stage 2 familial exudative vitreoretinopathy (FEVR) diagnosed under 3 months of age, and to present the first evidence of continued peripheral vascular growth and its influencing factors. Methods: This retrospective, consecutive study included 24 eyes (14 patients) between January 1, 2017, and December 1, 2024. Outcome measures were the response to anti-VEGF treatment and the vascular growth assessed by disc-vascularized border/disc-fovea (DB/DF). Results: The median age of patients at treatment was 10.5 days, with a median follow-up duration of 22 months. Nineteen (79%) eyes showed “Regression” and 3 (13%) demonstrated “Persistence”. Among them, six eyes received additional laser photocoagulation. The remaining two (8%) eyes showed “Progression” and underwent lens-sparing vitrectomy. The generalized estimating equations model revealed that retinal vessels continued to grow progressively after treatment (β = 0.0010; 95% CI, 0.0005–0.0014; P < 0.001). The segmented mixed model demonstrated that the relative DB/DF ratio increased at a rate of 0.004 per week (95% CI, 0.0023–0.0056) until 35.4 weeks post-treatment. Conclusion: Anti-VEGF therapy may effectively regress the high vascular activity of stage 2 early-diagnosed FEVR and may promote continued vascular growth toward the periphery.