Phase I/II Randomized Study of Proton Beam with Anti-Vascular Endothelial Growth Factor for Exudative Age-Related Macular Degeneration: One-Year Results.

Objective: To assess the safety and efficacy of proton beam therapy (PBT) as an adjunct to intravitreal anti-vascular endothelial growth factor (VEGF) for the treatment of exudative age-related macular degeneration.

Design: Phase I/II, interventional, prospective, randomized, sham-controlled double-blinded study.

Participants: Eyes with newly diagnosed exudative age-related macular degeneration with vision between 20/40 and 20/400 were included. Exclusion criteria included diabetes or other ocular comorbidities affecting vision.

Methods: Eyes were randomized to receive either 16 GyE, 24 GyE, or sham PBT. All eyes had 3 monthly intravitreal anti-VEGF treatments, followed by monthly visits with treatments as needed.

Main Outcome Measures: Mean change in best-corrected visual acuity (BCVA), mean number of anti-VEGF injections, proportion of eyes with >15 letters BCVA decrease, proportion of eyes developing radiation retinopathy or papillopathy, proportion of eyes with cataract progression, and mean changes central retinal thickness on OCT and lesion size on angiography at 1 year.

Results: Of 30 enrolled eyes, 22 completed follow-up monthly for 12 months for analysis. The BCVA improved by a mean of 8 letters (0.48±0.36 logarithm of the minimum angle of resolution) overall from baseline. Overall, central retinal thickness decreased from 340±155 to 246±48 (P = 0.008) at 12 months. The mean change in BCVA and central retinal thickness was not different among the 3 study groups. The mean number of anti-VEGF injections at 12 months was 6.13 for sham irradiation arm, 5.52 in the 16 GyE arm, and 3.83 for the 24 GyE arm (P = 0.004 between sham and 24 GyE). No eye had severe visual loss, radiation retinopathy, or papillopathy.

Conclusions: No safety issue was noted associated with combining 16 GyE or 24 GyE PBT with intravitreal anti-VEGF therapy in eyes with exudative age-related macular degeneration. Overall improvements in BCVA and imaging parameters were not affected by the addition of PBT, but the number of anti-VEGF treatments needed was significantly lower with the addition of 24 GyE PBT.