Anti-VEGF drug ranibizumab: A new treatment option against retinopathy of prematurity?

Retinopathy of prematurity (ROP) is an eye disease that only occurs in very premature babies (usually preterm babies born before the 31st week or born with a birthweight below 1250 g). With ROP, unwanted blood vessels grow on the retina in the back of the baby’s eye that can lead to serious eye problems and even blindness. There are two main treatment approaches, laser surgery and anti-VEGF injections. The latter aims at inhibiting VEGF, the growth factor mainly responsible for the excessive vessel growth on the retina.

The randomized, double-blind, multicenter clinical trial CARE-ROP (Comparing Alternative Ranibizumab dosages for safety and Efficacy in Retinopathy Of Prematurity) [1], published in JAMA Pediatrics, has investigated two different doses of the anti-VEGF drug ranibizumab for their safety and efficacy in the treatment of ROP. Ranibizumab is a drug that is licensed against eye diseases in adults with age-related macular degeneration (AMD), diabetic macular edema and other eye diseases.

The researchers around Stahl et al. found that both low ranibizumab dosages (24 vs. 40 % of the adult dose) were equally effective in treating acute ROP. Side effects, both locally in the eye and systemically, were limited and comparable between the two groups. Infants treated with the lower dose showed a tendency for superior development of the retina (retinal vascularization) at the end of the study, compared to the higher dose. This difference between the two doses, however, was not statistically significant.

Image © A. Stahl \ CARE-ROP

Although the CARE-ROP study was small with only 19 patients, it was the first study worldwide to investigate two different doses of ranibizumab in ROP in a randomised multicenter setting. The CARE-ROP study found that ranibizumab is effective in treating severe ROP. Larger studies are currently underway to validate these findings. Long-term effects of the treatment still need to be investigated, and results of the 1- and 2-year data are to be expected at the beginning of 2020.

For more information regarding the CARE-ROP study please contact

[1] Comparing alternative ranibizumab dosages for safety and efficacy in retinopathy of prematurity: A randomized clinical trial.Stahl A, Krohne TU, Eter N, et al. (2018)JAMA Pediatr. doi: 10.1001/jamapediatrics.2017.4838