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.
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.
Girmens
Jean François
JF
Service d'Ophtalmologie, Hôpital Lariboisière, Assistance Publique-Hôpitaux de Paris and Université Paris VII, Paris, France.
Erginay
Ali
A
Massin
Pascale
P
Scigalla
Paul
P
Gaudric
Alain
A
Richard
Stéphane
S
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*
D020533:Angiogenesis Inhibitors; D007211:Indoles; D011758:Pyrroles; C116890:Semaxinib; D040262:Receptors, Vascular Endothelial Growth Factor