Abstract
Purpose :
Despite widespread use for major ocular vasculature diseases, use of anti–vascular endothelial growth factors (VEGF) as primary treatment for nonproliferative diabetic retinopathy (NPDR) is debated. This study synthesized key efficacy results from pivotal clinical trials investigating anti-VEGF for primary treatment of DR.
Methods :
A systematic literature search was conducted using Cochrane, PubMed, and clinical trial registers to identify clinical trials of DR that compared anti-VEGF monotherapy vs laser photocoagulation (any type), sham treatment, or clinical observation. Standard methodologies were used to identify, select, extract data from, and assess quality of the studies. Results of patient (pt) outcomes related to visual acuity (VA) and DR progression on Diabetic Retinopathy Severity Scale are reported.
Results :
Among 975 publications, 13 met inclusion criteria for proliferative DR (PDR; n=5), diabetic macular edema (DME; n=6), and NPDR (n=2). At 2y, anti-VEGF resulted in better VA outcomes vs laser and sham in pts with PDR with or without DME, and pts with DME with variable baseline (BL) DR severity. In pts with NPDR without BL DME, anti-VEGF did not result in significant VA improvements vs sham. At longest follow-up across trials and indications, pts who received anti-VEGF generally showed higher rates of ≥2-step DR improvement on DRSS vs pts who did not (NPDR without BL DME at 2y, 52% vs 13%; PDR with or without DME at 5y, 46% and not evaluable; DME with variable BL DR severity at 3y, 38% vs 22%). Further, pts who received anti-VEGF showed lower rates of ≥2-step DR progression vs pts who did not (NPDR without BL DME at 2y, 4% vs 16%; PDR not reported; DME with variable BL DR severity at 3y, 3% vs 9%). In pts with NPDR without DME at BL, anti-VEGF injections were associated with lower rates of development of PDR and central-involved DME vs sham at 2y (4% vs 11%; 9% vs 21%).
Conclusions :
A growing body of clinical trials shows that anti-VEGF injections can regress DR severity in moderately severe NPDR and PDR and reduce rates of PDR progression and DME development in NPDR without DME at BL. The strongest predictor of anti-VEGF efficacy on DR improvement is BL DR severity, which is independent of BL DME presence/absence; however, anti-VEGF effect on VA depends on BL DME presence/absence. This review supports future research on anti-VEGF for primary treatment of DR with or without DME.
This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.