Anti-VEGF therapy is on the horizon for aggressive posterior ROP

Anti-angiogenic therapy for aggressive posterior retinopathy of prematurity (ROP) may be a feasibletherapy for these children with this form of ROP, which develops in profoundly immature neonates. TheBLOCK-ROP study, which is slated to begin soon, will add to the limited knowledge of the safety andefficacy of an anti-vascular endothelial growth factor (VEGF) drug in treating posterior ROP, AnthonyCapone Jr., MD, reported during Retina Subspecialty Day at the annual meeting of the American Academyof Ophthalmology.

"With the advent of FDA-approved drugs for anti-VEGF treatment, the possibility of treating eyesoff-label with an anti-VEGF drug has become possible," he said. The rationale for this treatmentapproach is that VEGF promotes retinal vascularization. The current standard of care is lasertreatment to the peripheral retina, which is not universally effective in fostering regression ofROP.

The largest experience to date with anti-VEGF therapy for ROP was carried out in Mexico, Portugal,and New York, in which 53 eyes were treated with one injection of bevacizumab (Avastin, Genentech)and followed for a mean of 6 months. The study included patients who received the standard of carefor ROP, eyes that were untreated because of poor visualization, and eyes with high-riskpre-threshold ROP in an institution where laser was unavailable, Dr. Capone recounted. He is clinicalassociate professor of ophthalmology, Oakland University, Royal Oak, MI.

"All eyes responded favorably to treatment regarding neovascularization; five eyes worsened. Therewere no serious systemic adverse events. The authors concluded that further studies were needed todetermine the safety and long-term efficacy of the treatment," Dr. Capone said.

The BLOCK-ROP study, a phase I trial will be carried out in the United States and Canada beginning in the fourthquarter of 2007, picks up the challenge and will evaluate the safety of one injection of bevacizumab(0.75 mg) into the vitreous cavity in 22 patients.

"The scientific rationale for using anti-VEGF therapy for ROP is compelling. This is an exciting timein ROP therapy. Approval of standard phase I and II data is appropriate considering the vulnerabilityof the target population. In the interim, caution is warranted with use of anti-VEGF drugs outside ofa clinical trial in premature neonates," Dr. Capone concluded.