Intravitreal aflibercept injection appears effective, safe in ROP
A slightly greater proportion of infants with retinopathy of prematurity (ROP) treated with intravitreal aflibercept injection (IAI) had no active disease and unfavorable structural outcomes compared to those treated with laser photocoagulation, although the difference was not statistically significant enough to meet the pre-determined non-inferiority margin of 5% in the randomized, open-label, non-inferiority, Phase 3 BUTTERFLEYE trial, according to data presented at the 2023 AAPOS Annual Meeting. However, the study showed that IAI was effective and safe for the treatment of ROP, with strong evidence supporting its use as a management option.
The study included 120 infants who had never received any previous treatment, with a mean gestational age of 27.3 weeks or a birthweight of 1500 g. These infants were randomly assigned in a 3:1 ratio to receive either IAI or laser therapy at the start of the trial. The main goal of the study was to determine if IAI was not inferior to laser therapy in terms of reducing active ROP and unfavorable structural outcomes in infants at 52 weeks of age (with a non-inferiority margin of 5%). The study also evaluated the safety of the treatments as a secondary endpoint.
A total of 93 patients were treated with either IAI and 27 patients with laser therapy. The success rate of treatment was 79.6% for IAI and 77.8% for laser therapy, and the difference was not statistically significant. The time required to administer IAI was significantly shorter than laser therapy.
Ocular treatment-emergent adverse events (TEAEs) were reported in 18.3% of IAI-treated infants and 25.9% of laser-treated infants, while serious ocular TEAEs were reported in 6.5% of IAI-treated infants and 11.1% of laser-treated infants. One death was reported in the IAI group, but it was not related to the treatment.
“Active ROP and unfavorable structural outcomes were absent in a numerically greater proportion of IAI- than laser-treated infants, although the 5% non-inferiority margin was not met,” the authors concluded. “Other efficacy and safety endpoints for IAI 0.4 mg provide strong evidence for its utility in the management of treatment-requiring ROP.”
Orge FH, et al. Effect of Intravitreal Aflibercept Versus Laser Photocoagulation for Retinopathy of Prematurity: Results from the Phase 3 BUTTERFLEYE Trial. Presented at: 2023 AAPOS Annual Meeting.