Original articleIntravitreal Ranibizumab Therapy for Retinal Capillary Hemangioblastoma Related to von Hippel-Lindau Disease
Section snippets
Patients and Methods
This study was an open-label, nonrandomized, prospective, pilot study of intravitreal injections of ranibizumab in patients with RCHs associated with VHL disease with either severe ocular disease or disease not amenable to standard therapy. The diagnosis of VHL disease was made both clinically according to the criteria of Melmon and Rosen21 and genetically by the detection of a disease-causing mutation in the germline sequence of the VHL gene. Inclusion criteria included having best-corrected
Patient 1
A 33-year-old man with systemic VHL disease with renal, pancreatic, cerebellar, and endolymphatic sac involvement, also had severe bilateral ocular disease related to RCHs. Extensive RCH involvement in his left eye had resulted in enucleation 4 years before study enrollment. The right eye had a large juxtapapillary lesion that had been treated with argon laser photocoagulation 12 years prior, resulting in a small centrocecal scotomatous field defect. Visual acuity immediately posttreatment was,
Results
Demographics and ocular characteristics of the 5 patients in this prospective treatment study are given in Table 1.
Active exudative activity related to the RCHs was evident in all study eyes at baseline. Visual acuities at baseline ranged from 20/25 to 20/500 (79 to 18 letters; average, 54±26 letters). After 7 initial injections, patients were assessed for treatment benefit. In 2 patients (patients 1 and 4), treatment benefit was not apparent and injections were stopped. In the remaining
Discussion
In this study, we evaluated the effects of intravitreal ranibizumab (0.5 mg) in 5 patients in treating advanced retinal disease associated with VHL-related RCHs. To our knowledge, this is the first report of the use of ranibizumab and the second prospective study on the use of anti-VEGF therapy for this indication. Aberrantly high levels of VEGF secreted by tumor cells lacking the VHL protein are thought to promote the formation and growth of hemangioblastomas.8 Originally termed vascular
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Manuscript no. 2008-109.
Financial Disclosure(s): No conflicting relationship exists for any author.
Supported by the Intramural Research Program of the National Institutes of Health, Division of Epidemiology and Clinical Research, National Eye Institute.