Pegaptanib sodium for occult choroidal neovascularization in neovascular age-related macular degeneration: a...

To assess the effects of pegaptanib in the treatment of subfoveal occult choroidal neovascularisation (CNV) associated with neovascular age-related macular degeneration (NV-AMD) in a compassionate use program in France.

Pegaptanib was authorized for patients with CNV-associated visual impairment and in whom usual care (thermal laser photocoagulation or photodynamic therapy with verteporfin) was not appropriate. Patients with occult CNV lesions received intravitreous pegaptanib (0.3 mg every 6 weeks) and were followed with repeated fluorescein angiography, scanning laser ophthalmoscopy-infracyanine green angiography, and ocular coherence tomography through 52 weeks.

Of 56 patients (predominantly occult, N=22; purely occult, N=8; occult with chorioretinal anastomosis, N=12; occult with pigment epithelial detachment, N=14), 30% had earlier treatment. All received eight pegaptanib injections. At week 52, 79% were responders (lost <15 letters of visual acuity), 43% gained >or=0 letters, and 9% gained >or=15 letters. The best functional results were obtained in the predominantly and pure occult subgroups (responders, 86 and 75%; gained >or=0 letters, 50 and 50%). Maximum visual outcomes that correlated with morphologic improvements on each diagnostic imaging tool were seen after at least three injections. No significant ocular or systemic adverse events occurred.

Treatment with pegaptanib was associated with objective functional improvements that can be correlated with objective clinical improvements on routine diagnostic imaging tools in patients with occult NV-AMD. Optimum treatment results appear after at least 4 months of therapy in the majority of cases.