A serum metabolomics study of patients with nAMD in response to anti-VEGF therapy

Intravitreal injection of anti-vascular endothelial growth factor (anti-VEGF) is the current standard of treatment for choroidal neovascularization (CNV) secondary to neovascular age-related macular degeneration (nAMD), but there are no diagnostic tools to predict response of these therapies. We hypothesize that differences in baseline metabolic profiles of patients with nAMD may influence responsiveness to anti-VEGF therapy, and thus provide prognosticating information for these patients. A prospective study was performed on 100 patients with nAMD treated with anti-VEGF therapy. We classified patients into two groups: responders (n = 54) and non-responders (n = 46). The expression levels of glycerophosphocholine,LysoPC (18:2) and PS (18:0/20:4) were higher in non-responders and these findings were verified in the validation cohort, implicating that reductions in these three metabolites can be used as predictors for responsiveness to anti-VEGF therapy during the initial loading phase for patients with nAMD. Our study also provided new insights into the pathophysiological changes and molecular mechanism of anti- VEGF therapy for nAMD patients.