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• W2564654074 abstract "To describe the clinical course of advanced juxtapapillary retinal capillary hemangioblastomas (RCHs) associated with von Hippel-Lindau (VHL) disease treated with systemic sunitinib malate, an agent that inhibits both anti–vascular endothelial growth factor and anti–platelet-derived growth factor signaling. Observational case review. Three patients with advanced VHL-related juxtapapillary RCHs treated with systemic sunitinib malate. Patient 1 was followed routinely every 4 months while on systemic sunitinib prescribed by her oncologist for metastatic pancreatic neuroendocrine and kidney tumors. Patients 2 and 3 were part of a prospective clinical trial evaluating the use of systemic sunitinib for ocular VHL lesions during a period of 9 months. Visual acuity, size of RCHs, and degree of exudation were recorded at each visit. Optical coherence tomography and fluorescein angiography were also obtained at some visits. Visual acuity, size of RCHs, and degree of exudation. Three patients with advanced VHL-associated juxtapapillary RCHs were treated with systemic sunitinib malate. Although none of the patients lost vision during therapy, treatment with sunitinib malate did not improve visual acuity or reduce the size of RCHs. Improvements in RCH-associated retinal edema were observed in 2 patients. All patients experienced multiple adverse effects, including thyroid toxicity, thrombocytopenia, nausea, fatigue, jaundice, and muscle aches. Two of the 3 patients had to discontinue treatment prematurely and the third required dose reduction. Systemic sunitinib malate may be useful in slowing progression of ocular disease from VHL-associated RCHs. However, significant systemic adverse effects limited its use in this small series, and systemic sunitinib malate may not be safe for treatment of RCHs when used at the doses described in this report. Further studies are required to determine if this medication, used at lower doses with different treatment strategies; other medications in the same class; or drugs directed at multiple targets in the tumor may be safer and more effective for the treatment of advanced VHL-associated RCHs." @default.
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• W2564654074 date "2017-05-01" @default.
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• W2564654074 title "Systemic Sunitinib Malate Treatment for Advanced Juxtapapillary Retinal Hemangioblastomas Associated with von Hippel-Lindau Disease" @default.
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• W2564654074 doi "https://doi.org/10.1016/j.oret.2016.10.007" @default.
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