Abstract
Purpose :
To evaluate the efficacy and safety of intravitreal aflibercept injection (IAI) versus laser photocoagulation in infants with retinopathy of prematurity (ROP).
Methods :
BUTTERFLEYE (NCT04101721) was a randomized, open-label, non-inferiority, Phase 3 trial in which treatment-naïve infants (gestational age ≤32 weeks or birthweight ≤1500 g) with ROP were randomized 3:1 to receive IAI 0.4 mg or laser at baseline. The primary endpoint was the proportion of infants without active ROP and unfavorable structural outcomes at Week 52 of chronological age (non-inferiority margin: 5%). Secondary endpoints included safety.
Results :
Overall, 120 infants (IAI 0.4 mg: n=93; laser: n=27) were treated. Mean (SD) gestational age at birth was 27.3 (2.7) weeks. Treatment success rate (IAI versus laser) was 79.6% versus 77.8% (adjusted difference [95.1% CI]: 1.8% [-15.7%, 19.3%]). Mean (SD) time required to administer IAI versus laser was 10.7 (17.2) versus 129.2 (95.6) minutes/infant (nominal P<0.0001). Ocular treatment-emergent adverse events (TEAEs) were reported in 18.3% and 25.9% of infants in the IAI and laser groups, respectively. Serious ocular TEAEs were reported in 6.5% and 11.1% of IAI- and laser-treated infants, respectively. One death was reported in the IAI group and was unrelated to treatment.
Conclusions :
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. Other efficacy and safety endpoints for IAI 0.4 mg provide strong evidence for its utility in the management of treatment-requiring ROP.
This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.