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K.A. Drenser, M.T. Trese, A. Capone, Jr.; Macugen Therapy for the Treatment of Familial Exudative Vitreoretinopathy . Invest. Ophthalmol. Vis. Sci. 2006;47(13):4496.
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Patients with familial exudative vitreoretinopathy (FEVR) often have mutations affecting the regulation of a cell signaling pathway (Wnt receptor:B–catenin signal transduction pathway) that, when dysregulated, results in abnormal vascular activity with increased vascular endothelial growth factor (VEGF) levels. The purpose of this study is to evaluate the effectiveness of anti–VEGF therapy for the treatment of vitreoretinopathies due to aberrant up–regulation of VEGF.
Patients with vascularly active FEVR recalcitrant to standard interventions, as demonstrated by increasing subretinal exudation in spite of laser, cryo and/or steroid treatment, were treated with Macugen (anti–VEGF antibody) injection. Retrospective chart review was performed to analyze the outcomes of these patients. Clinical assessment of subretinal exudation and vascular activity was evaluated with fundus photography, fluorescein angiography and OCT. Visual acuity was evaluated as a functional test of treatment.
Four patients treated with Macugen were found to have decreased subretinal exudation and decreased vascular activity at 6 weeks post–injection. Visual acuity was also improved at this visit. Two patients developed an increased fibrotic response requiring surgery. Both patients underwent vitrectomy and had improved clinical appearance and visual acuity 2 months after surgery compared to pre–Macugen treatment. All patients maintained improved vision 6 months after initial Macugen treatment.
Macugen therapy is an effective treatment option for patients with FEVR who continue to have subretinal exudation and vascular activity despite standard interventions. Patients have improved clinical appearance and increased visual activity following treatment. Even patients requiring vitrectomy to address the fibrotic response enjoy improved clinical outcomes.
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