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K. J. Liang, W. T. Wong, K. Hammel, H. R. Coleman, E. Y. Chew; Intravitreal Ranibizumab for Treatment of Retinal Capillary Hemangioblastoma (RCH) Related to von Hippel-Lindau (VHL) Disease. Invest. Ophthalmol. Vis. Sci. 2008;49(13):5440.
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The formation of retinal capillary hemangioblastomas (RCHs) has been hypothesized to result from increased levels of vascular endothelial growth factor (VEGF). This prospective pilot study was designed to evaluate the safety and efficacy of intravitreal ranibizumab as a treatment for retinal capillary hemangioblastomas (RCHs) related to von Hippel-Lindau (VHL) disease.
5 patients with VHL-related RCHs were enrolled in an open label, prospective, pilot study. Treatment consisted of 7 monthly intravitreal injections over a 6-month period, after which additional monthly injections, up to 1 year, were considered on an individual basis. The designated final study visit was 2 months after the last injection. The primary outcome was a change in best-corrected visual acuity (BCVA) of 15 letters or more at the final visit compared to baseline. Secondary outcomes included a reduction of retinal thickening as documented by optical coherence tomography (OCT), reduction in fluorescein leakage from the lesion, the number of re-treatments and adverse event assessments.
Patients received an average of 10.0±3.1 injections during the study. Three patients were released from the study before the full year (2 patients after 7 injections, and 1 patient after 12 injections) due to disease progression while on treatment. 2 patients experienced a visual acuity improvement of 6 and 15 letters, while the average BCVA decreased by -9±20 letters. Anatomically, during the period of treatment, tumor growth was seen in 2 patients, stability in 2 patients and regression in 1 patient. Only 2 out of 5 patients experienced a decrease in retinal thickness in affected quadrants. 1 patient experienced mild therapy-related adverse events (lid edema and conjunctiva hyperemia). No severe adverse events were recorded.
Although well-tolerated, intravitreal ranibizumab exerted variable effects on VHL-related RCHs, resulting in decreased exudation and tumor stability and/or regression in some cases and increased exudation and tumor growth in others. In at least one patient, changes in tumor activity were clearly related to treatment. Future prospective studies would be useful in determining the optimal route, dosing, duration and application of anti-angiogenic therapy to the treatment of RCHs.
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