semaxinib and Hemangioblastoma

Topics: Adult; Angiogenesis Inhibitors; Brain Neoplasms; Female; Hemangioblastoma; Humans; Indoles; Male; Middle Aged; Pyrroles; Retinal Neoplasms; Spinal Cord Neoplasms; Treatment Outcome; Vascular Endothelial Growth Factor A; von Hippel-Lindau Disease

Topics: Angiogenesis Inhibitors; Biopsy, Needle; Cerebellar Neoplasms; Diagnosis, Differential; Follow-Up Studies; Hemangioblastoma; Humans; Immunohistochemistry; Indoles; Magnetic Resonance Imaging; Male; Middle Aged; Neoplasm Recurrence, Local; Pyrroles; Risk Assessment; Treatment Outcome; von Hippel-Lindau Disease

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).. Interventional case report.. 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.. 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.. The VEGF receptor inhibitor SU5416 failed to reduce the size of RHB but was very effective for the associated CME.

Topics: Adult; Angiogenesis Inhibitors; Female; Fluorescein Angiography; Hemangioblastoma; Humans; Indoles; Infusions, Intravenous; Macular Edema; Pyrroles; Receptors, Vascular Endothelial Growth Factor; Retinal Neoplasms; Visual Acuity; von Hippel-Lindau Disease

To present a case of rapid and durable recovery of visual function in a patient with von Hippel-Lindau syndrome and optic nerve head hemangioblastoma after systemic treatment with the vascular endothelial growth factor (VEGF) receptor inhibitor SU5416.. Interventional case report.. Visual function parameters, including visual acuity, automated visual field, and contrast sensitivity, were evaluated using standardized methods repeatedly over a 15-month period. Fundus photographs and fluorescein angiograms were also obtained. Central nervous system lesions were monitored by computed tomography (CT) and magnetic resonance imaging (MRI) scans. Treatment involved the systemic administration of the VEGF receptor inhibitor SU5416.. Clinical presentation, visual acuity using Early Treatment Diabetic Retinopathy Study protocol, Humphrey automated perimetry, (Zeiss Humphrey Systems, Dublin, CA) Vistech contrast sensitivity (Vistech Consultants Inc., Dayton, OH) Farnsworth (Farnsworth-Munsell Color Services, New Windsor, NY) dichotomous panel D-15, retinal photography, fluorescein angiography, and CT and MRI scans.. Within 4 weeks of therapy, visual acuity had improved from 20/32(-2) to 20/16(-1), the visual field had expanded from being circumferentially constricted to within 8 degrees of fixation to normal, and contrast sensitivity improved in all but the lowest spatial frequency (1.5 cycles/degree). No change was observed in lesion size by fundus photography. Improvement has been maintained over 18 months with intermittent SU5416 therapy.. We report rapid, extensive, and durable recovery of visual function after systemic administration of the VEGF receptor inhibitor SU5416 to a patient with VHL syndrome and optic nerve head hemangioblastoma. These findings suggest that continued evaluation of VEGF inhibitors for ocular neovascular disorders is warranted.

Topics: Angiogenesis Inhibitors; Contrast Sensitivity; Female; Fluorescein Angiography; Hemangioblastoma; Humans; Indoles; Magnetic Resonance Imaging; Middle Aged; Optic Disk; Optic Nerve Neoplasms; Pyrroles; Receptor Protein-Tyrosine Kinases; Receptors, Growth Factor; Receptors, Vascular Endothelial Growth Factor; Recovery of Function; Visual Acuity; Visual Fields; von Hippel-Lindau Disease