Intravitreal ranibizumab therapy for retinal capillary hemangioblastoma related to von Hippel-Lindau disease.
OBJECTIVE
To evaluate the effect of intravitreal ranibizumab on retinal capillary hemangioblastomas (RCHs) associated with von Hippel-Lindau (VHL) disease that are not amenable or responsive to standard therapy.


METHODS
Prospective, noncomparative, interventional case series.


METHODS
Five patients with VHL-associated RCH with exudative changes and visual loss.


METHODS
Monthly intravitreal injections of ranibizumab (0.5 mg) were given over a course of 6 months for a total of 7 injections, with additional injections considered until week 52. The final study visit was designated as 8 weeks after the final study injection.


METHODS
The primary outcome was the change in best-corrected visual acuity (BCVA) of >/=15 letters at the final visit compared with baseline. Secondary outcomes included change in lesion size, exudation as assessed clinically and by fluorescein angiography, change in retinal thickness as evaluated by optical coherence tomography, and adverse event assessments.


RESULTS
Patients received an average of 10.0+/-3.1 injections over an average period of 47+/-14 weeks, including follow-up. Mean change in BCVA was a decrease of 9+/-20 letters, with 1 patient gaining >/=15 letters, and 2 patients losing >/=15 letters. Changes in both lesion size and exudation were variable.


CONCLUSIONS
Intravitreal ranibizumab, delivered as monotherapy every 4 weeks, had minimal beneficial effects on most VHL-related RCHs. Possible treatment efficacy was demonstrated in the patient with the smallest lesion with less exudation. Future prospective studies are needed to determine the potential role of an antiangiogenic agent, possibly in combination with other therapies for the treatment of such advanced ocular tumors associated with VHL.
Wong
Wai T
WT
Office of Scientific Director, National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.
Liang
Katharine J
KJ
Hammel
Keri
K
Coleman
Hanna R
HR
Chew
Emily Y
EY
D000328:Adult; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D005260:Female; D005451:Fluorescein Angiography; D018325:Hemangioblastoma / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D007267:Injections; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011446:Prospective Studies; D000069579:Ranibizumab; D019572:Retinal Neoplasms / Q000175:diagnosis / Q000188:drug therapy*; D019233:Retreatment; D041623:Tomography, Optical Coherence; D016896:Treatment Outcome; D014792:Visual Acuity; D014822:Vitreous Body; D006623:von Hippel-Lindau Disease / Q000175:diagnosis / Q000188:drug therapy*
D020533:Angiogenesis Inhibitors; D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000069579:Ranibizumab
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