Anti-vascular endothelial growth factor therapy for subfoveal choroidal neovascularization secondary to age-related macular degeneration: phase II study results.

PURPOSE: There is evidence to suggest that anti-vascular endothelial growth factor (anti-VEGF) therapy may be useful in treating ocular neovascularization. A phase IA single intravitreal injection study of anti-VEGF therapy for patients with subfoveal choroidal neovascularization (CNV) secondary to age-related macular degeneration (AMD) revealed a good safety profile. We performed a phase II multiple injection study of anti-VEGF therapy with and without photodynamic therapy for patients with subfoveal CNV secondary to AMD to determine the safety profile of multiple injection therapy.

DESIGN: A phase II multiple-dose safety study.

PARTICIPANTS/METHODS: Twenty-one patients were treated with intravitreal injection with and without photodynamic therapy.

MAIN OUTCOME MEASURES: Clinical evidence of toxicity and complications.

RESULTS: No drug-related serious adverse events were revealed. Ophthalmic evaluation revealed that 87.5% of patients who received the anti-VEGF aptamer alone showed stabilized or improved vision 3 months after treatment and that 25% of eyes demonstrated a 3 line or greater improvement in vision on the Early Treatment of Diabetic Retinopathy Study chart during this period. A 60% 3 line gain at 3 months was noted in patients who received both the anti-VEGF aptamer and photodynamic therapy.

CONCLUSIONS: Anti-VEGF therapy is a promising treatment for various forms of ocular neovascularization, including AMD. Multiple intravitreal injections of the anti-VEGF aptamer were well tolerated in this phase II study. Further clinical trials are necessary to demonstrate the efficacy and long-term safety of anti-VEGF therapy for AMD.