Intravitreal anti-vascular endothelial growth factor therapy with pegaptanib for advanced von Hippel-Lindau disease of the retina.
OBJECTIVE
This pilot study was designed to provide preliminary data concerning the safety and efficacy of pegylated anti-vascular endothelial growth factor (VEGF) therapy, pegaptanib, for patients with juxtapapillary or large peripheral angiomas secondary to von Hippel-Lindau (VHL) disease.


METHODS
This study was an open label, nonrandomized, prospective, pilot study of intravitreal injections of pegaptanib (3 mg/100 microL), given every 6 weeks for minimum of 6 injections. Five patients with severe ocular VHL lesions were enrolled in the study. The primary outcome of this study was a change of > or =15 letters (3 lines) in best-corrected visual acuity by 1 year. Secondary outcomes included changes in macular thickness, as determined by optical coherence tomography, and changes in fluorescein leakage.


RESULTS
Two of five patients completed the course of treatment and 1 year of follow-up. These two patients had progressive decrease in retinal hard exudate and reduction in central retinal thickness measured by optical coherence tomography. One of these two patients had improvement in visual acuity of 3 lines. No significant change in fluorescein leakage or tumor size was detected in either patient. Lesions in the other three patients continued to progress despite treatment, and these patients did not complete the entire treatment course. One patient developed a tractional retinal detachment. Additional serious adverse events included transient postinjection hypotony in two eyes.


CONCLUSIONS
Intravitreal injections of anti-VEGF therapy (pegaptanib) may decrease retinal thickening minimally and reduce retinal hard exudates in some patients with advanced VHL angiomas. This finding may be related to a reduction in vasopermeability, because there was no apparent effect of treatment on the size of the primary retinal angiomas in this small pilot study.
Dahr
Sam S
SS
Division of Epidemiology and Clinical Research, National Eye Institute, National Institutes of Health, Bethesda, Maryland 20892, USA.
Cusick
Michael
M
Rodriguez-Coleman
Hanna
H
Srivastava
Sunil K
SK
Thompson
Darby J
DJ
Linehan
W Marston
WM
Ferris
Frederick L
FL
Chew
Emily Y
EY
D000328:Adult; D052157:Aptamers, Nucleotide / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D005451:Fluorescein Angiography; D018324:Hemangioma, Capillary / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D007267:Injections; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011446:Prospective Studies; D019572:Retinal Neoplasms / Q000175:diagnosis / Q000188:drug therapy*; D041623:Tomography, Optical Coherence; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors*; D014792:Visual Acuity; D014822:Vitreous Body; D006623:von Hippel-Lindau Disease / Q000175:diagnosis / Q000188:drug therapy*
D052157:Aptamers, Nucleotide; C467484:VEGFA protein, human; D042461:Vascular Endothelial Growth Factor A; C495058:pegaptanib