Treatment of von Hippel-Lindau retinal hemangioblatoma by the vacular endothelial growth factor receptor inhibitor U5416 i more effective for aociated macular edema than for hemangioblatoma

Purpoe To tet the efficacy of the novel vacular endothelial growth factor (VEGF) receptor inhibitor U5416, in a cae of refractory von Hippel-Lindau (VHL) retinal hemangioblatoma (RHB). Deign Interventional cae report. Method Patient included in a multicenter phae II trial. A 30-year-old woman preenting with VHL dieae and multiple RHB on her only eye, refractory to conventional treatment, had decreaed viual acuity due to cytoid macular edema (CME). U5416 wa adminitered intravenouly for 7 month. Bet-corrected viual acuity (BCVA) and macular thickne were meaured by optical coherence tomography. Reult Under treatment, the ize of the RHB did not change, but CME improved ignificantly. Bet-corrected viual acuity roe from 20/40 to 20/25. However, CME recurred after the end of the treatment. Concluion The VEGF receptor inhibitor U5416 failed to reduce the ize of RHB but wa very effective for the aociated CME.