Anti-VEGF treatment in Pseudoxanthoma elasticum: preliminary results of a long-term ophthalmological follow-up

The pseudoxanthoma elasticum (PXE) retinopathy is characterized by peau d'orange and angioid streaks, with choroidal neovascularization, subretinal bleeding and (central) blindness as most severe complications. Among all features in the PXE phenotypic spectrum, the retinopathy is considered to have the highest impact on quality of life of PXE patients. The introduction of anti-angiogenic treatment using antibodies against Vascular Endothelial Growth Factor (VEGF), such as ranibizumab or bevacizumab over a decade ago and more recently aflibercept, led to a tremendous improvement of the patients' ophthalmological prognoses by preventing blindness in numerous PXE patients. As such it has become the standard-of-care in the treatment of the complications resulting from the PXE retinopathy. Moreover, in some cases, intra-ocular anti-VEGF injections are being used in a preventive manner. In this study, we have compared fundus imaging of patients prior to the anti-VEGF era with patients who underwent multiple anti-VEGF injections. Further, the PXE patient cohort treated with anti-VEGF drugs was thoroughly assessed with longitudinal follow up comparing the ophthalmological phenotype prior to and after treatment. Visual acuity, anatomical and functional characteristics of the retinopathy were characterized using BCVA measurement (1/20: 2 letters; starting from 1/10: 5 letters per vision level), slit-lamp, Goldman visual field examination and optical coherence tomography (OCT), fundus imaging with white light, infrared and blue reflectance, infrared and blue autofluorescence and a fluorescein angiography if needed. We will describe the evolution of the ophthalmological phenotype with and without anti-VEGF treatment. Importantly, we observed retinal fibrosis in some of our patients who underwent multiple anti-VEGF injections in order to preserve their eyesight. Fibrosis, a complication, which has already been described in proliferative diabetic retinopathy, can indeed on its own deteriorate the patients' vision. Very recently, in vitro experiments showed an upregulation of pro-fibrotic agents after application of clinical doses of bevacizumab, thereby identifying a causal link between anti-VEGF treatment and retinal fibrosis. As a consequence, concerns must arise about the long-term effects and safety of anti-VEGF treatment. In conclusion, it might not be beneficial to use anti-VEGF treatment unlimitedly, e.g. in a preventive setting, in PXE. In absence of good clinical guidelines, the time has come to draft the criteria for anti-VEGF treatment in PXE patients.

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