Treatment of vascularly active familial exudative vitreoretinopathy with pegaptanib sodium (Macugen).

PURPOSE: To report results of treatment of vascularly active familial exudative vitreoretinopathy (FEVR) with pegaptanib sodium (Macugen; Eyetech Pharmaceuticals, New York, NY) injection.

METHODS

In a retrospective case series, four patients with vascularly active FEVR, as demonstrated by increasing subretinal exudation despite photocoagulation, cryotherapy, and/or intravitreal steroid injection, received a single intravitreal injection of pegaptanib sodium. Preinjection and postinjection fundus photography, fluorescein angiography, and optical coherence tomography were performed to evaluate the changes in visual acuity, vascular activity, and amount of exudation.

RESULTS

The mean follow-up period was 11.2 months (range, 8.1-15.5 months) after the first intravitreal injection. All four patients had a decrease in exudation after treatment with pegaptanib sodium documented by a decrease in subretinal exudate by fundus photography and decreased leakage by fluorescein angiography. After reduction of exudation, two patients required vitrectomy to relieve vitreoretinal traction. Visual acuity improved in two patients, stabilized in one patient, and worsened in one patient secondary to tractional retinal detachment. No injection-associated systemic or ocular complications were observed in any of the treated patients.

CONCLUSIONS

Intravitreal injection of pegaptanib sodium is a potential treatment option for patients with FEVR and worsening exudation despite treatment with standard therapy. Vitreoretinal traction may develop with rapid resolution of subretinal exudates, requiring surgical intervention. However, visual acuity can improve after retinal traction is released. Further studies using anti-vascular endothelial growth factor agents are needed to better understand treatment of FEVR.