Purpose: To evaluate the efficacy and safety of monthly intravitreal ranibizumab for the treatment of choroidal neovascularizations (CNV) secondary to angioid streaks (AS) in pseudoxanthoma elasticum (PXE). Design: Twelve-month prospective, open-label, uncontrolled, nonrandomized interventional clinical trial. Methods: In 7 patients, 1 eye with an active CNV was injected with 0.5 mg ranibizumab monthly over 1 year. Distance and reading visual acuity, reading speed, angiographic findings, and central retinal thickness (CRT) on optical coherence tomography were assessed at each visit. Central retinal light increment sensitivity (LIS) was assessed by microperimetry at baseline, at 6 months, and 3 to 4 months after the last injection. Results: Best-corrected visual acuity increased significantly from baseline to month 12 (20/63 or 61 ETDRS letters to 20/32 or 73 ETDRS letters; P =.012). The effect was maintained 3 months later (61 ETDRS letters to 72 ETDRS letters; P =.055). Reading acuity and speed could be maintained throughout the study. Central LIS improved (6.6 dB, SD ± 5.9 at baseline to 7.4 dB, SD ± 6.2 at last follow-up; P <.001). Leakage from active CNVs subsided. Mean change in CRT from baseline to month 12 and 15 was -86 μm (P =.074) and -65 μm (P =.182), respectively. No serious adverse events occurred. Conclusions: Efficacy outcomes indicate a beneficial therapeutic effect of intravitreal ranibizumab on central visual function including retinal LIS. Both the functional and morphologic response based on angiographic and OCT findings to ranibizumab treatment implicate an important pathophysiological role of vascular endothelial growth factor in CNVs secondary to AS in PXE. Intravitreal ranibizumab appears to be a safe and efficacious treatment in these patients.
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