Efficacy And Vascular Redevelopment Of Anti-Vascular Endothelial Growth Factor Therapy For Stage 2 Early-Diagnosed Familial Exudative Vitreoretinopathy

Retina. 2026 Mar 1;46(3):539-547. doi: 10.1097/IAE.0000000000004714.

Summary

FEVR: Anti-VEGF is effective for regressing high vascular activity in Stage 2 disease. May promote continued vascular growth towards the periphery. Retrospective, 24 eyes.

Abstract

Purpose: To report the efficacy of anti-vascular endothelial growth factor (vascular endothelial growth factor) therapy in stage 2 familial exudative vitreoretinopathy 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.

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% confidence intervals, 0.0005-0.0014; P < 0.001). The segmented mixed model demonstrated that the relative disc-vascularized border/disc-fovea ratio increased at a rate of 0.004 per week (95% confidence intervals, 0.0023-0.0056) until 35.4 weeks posttreatment.

Conclusion: Anti-VEGF therapy may effectively regress the high vascular activity of stage 2 early-diagnosed familial exudative vitreoretinopathy and may promote continued vascular growth toward the periphery.