title: Treatment of von Hippel-Lindau retinal hemangioblastoma by the vascular endothelial growth factor receptor inhibitor SU5416 is more effective for associated macular edema than for hemangioblastomas.
abstract: OBJECTIVE
  To test the efficacy of the novel vascular endothelial growth factor (VEGF) receptor inhibitor SU5416, in a case of refractory von Hippel-Lindau (VHL) retinal hemangioblastoma (RHB).
  
  
  METHODS
  Interventional case report.
  
  
  METHODS
  Patient included in a multicenter phase II trial. A 30-year-old woman presenting with VHL disease and multiple RHB on her only eye, refractory to conventional treatments, had decreased visual acuity due to cystoid macular edema (CME). SU5416 was administered intravenously for 7 months. Best-corrected visual acuity (BCVA) and macular thickness were measured by optical coherence tomography.
  
  
  RESULTS
  Under treatment, the size of the RHB did not change, but CME improved significantly. Best-corrected visual acuity rose from 20/40 to 20/25. However, CME recurred after the end of the treatment.
  
  
  CONCLUSIONS
  The VEGF receptor inhibitor SU5416 failed to reduce the size of RHB but was very effective for the associated CME.
authors.affiliation: Service d'Ophtalmologie, Hôpital Lariboisière, Assistance Publique-Hôpitaux de Paris and Université Paris VII, Paris, France.
authors.forename: Jean François
authors.initials: JF
authors.lastname: Girmens
authors.forename: Ali
authors.initials: A
authors.lastname: Erginay
authors.forename: Pascale
authors.initials: P
authors.lastname: Massin
authors.forename: Paul
authors.initials: P
authors.lastname: Scigalla
authors.forename: Alain
authors.initials: A
authors.lastname: Gaudric
authors.forename: Stéphane
authors.initials: S
authors.lastname: Richard
mesh_terms: D000328:Adult; D020533:Angiogenesis Inhibitors / Q000008:administration & dosage / Q000627:therapeutic use*; D005260:Female; D005451:Fluorescein Angiography; D018325:Hemangioblastoma / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D007211:Indoles / Q000008:administration & dosage / Q000627:therapeutic use*; D007262:Infusions, Intravenous; D008269:Macular Edema / Q000175:diagnosis / Q000188:drug therapy*; D011758:Pyrroles / Q000008:administration & dosage / Q000627:therapeutic use*; D040262:Receptors, Vascular Endothelial Growth Factor / Q000037:antagonists & inhibitors*; D019572:Retinal Neoplasms / Q000175:diagnosis / Q000188:drug therapy*; D014792:Visual Acuity; D006623:von Hippel-Lindau Disease / Q000175:diagnosis / Q000188:drug therapy*
chemical_list: D020533:Angiogenesis Inhibitors; D007211:Indoles; D011758:Pyrroles; C116890:Semaxinib; D040262:Receptors, Vascular Endothelial Growth Factor