Treatment of retinopathy of prematurity with vascular endothelial growth factor inhibitors.

ROP remains a major cause of childhood blindness worldwide. The smallest, sickest infants develop the most severe forms of zone 1 ROP. Such eyes may not be successfully treated by near confluent laser to the avascular retina (current standard of care). With an understanding of ROP pathogenesis, vascular endothelial growth factor inhibitors (anti-VEGF) are being given only when VEGF is elevated in retina and vitreous. Careful screening allows proper timing of administration. Ideal dose (perhaps different for mild and severe cases) and drug (interrupting only pathologic neovascularization and not normal angiogenesis) remain unproven. The author discusses controversial use of anti-VEGF with documented efficacy, observed local complications, and potential systemic toxicities (none observed in six years) to allow retention of vision for severe zone 1 ROP. The benefits have been demonstrated, however, local and systemic risks in these developing premature infants must be carefully studied (both short and long term).