Ranibizumab for treatment of type 1 ROP: 5 year outcomes of the RAINBOW Trial

Abstract

Purpose : To report 5 years outcomes of RAnibizumab compared with laser therapy for the treatment of INfants BOrn prematurely With retinopathy of prematurity (RAINBOW) extension study

Methods : RAINBOW was an open label, randomized trial comparing two different intravitreal ranibizumab doses (0.1mg and 0.2mg) with laser therapy for the treatment of Type 1 ROP in 201very low birthweight infants (<1500g). 180 infants entered the RAINBOW extension study which evaluated treatment outcomes to 5 years. Participants had local ophthalmic, development, and health assessments. The primary outcome was "to evaluate the visual function of patients, by assessing the visual acuity at the patient’s fifth birthday". Secondary outcomes included: the safety outcomes; the absence of ocular structural abnormalities; physical development, health status and cognitive skills; Children Visual Function Questionnaire composite and subscale scores; additional ocular data such as the presence of nystagmus, abnormal fixation behaviour, strabismus and abnormal pupillary light reaction and, last but not least the degree of full peripheral retina vascularization.

Results : 156 (87%) children completed the extension study (54 in the ranibizumab 0.2mg group: 55 in the ranibizumab 0.1mg; 47 in the Laser group).
124/156 (79.5%) of children provided ETDRS acuities. The difference in mean ETDRS letter score in the better-seeing eye between ranibizumab 0.2 mg compared to laser was 4.7 letters (95% CI: -1.1, 10.5), and between ranibizumab 0.1 mg and laser was 2.5 letters (95%CI: -3.4, 8.3). The proportion of patients with ETDRS score of ≥71 letters was 32.8% in the ranibizumab 0.2 mg arm, 23.1% the ranibizumab 0.1 mg arm and 20.4%in the laser arm.
Median (IQR) refraction (expressed in spherical equivalent diopters) was 0 (-1.3 to 0-75; n-104) in ranibizumab 0.2mg; -0.25 (-2.00 to 1.0; n. 109) in ranibizumab 0.1mg and -0.50 (-4.0 to 0.69; n 90) in laser group.
Structural retinal abnormalities were present in 11 children (ranibizumab 0.2mg: 1; ranibizumab 0.1mg: 4; Laser: 6).
No differences between the three trial arms were observed for developmental scores, growth attainment or vision-related quality of life.

Conclusions : In the RAINBOW trial outcomes at 5 years confirmed an excess of high myopia following laser compared to ranibizumab, but substantively similar acuity, other ocular and safety outcomes between groups.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.