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Knickelbein JE, Jacobs-El N, Wong WT, et al. Systemic Sunitinib Malate Treatment for Advanced Juxtapapillary Retinal Hemangioblastomas Associated with von Hippel-Lindau Disease. Ophthalmol Retina. 2017;1(3):181-187doi: 10.1016/j.oret.2016.10.007.
Knickelbein, J. E., Jacobs-El, N., Wong, W. T., Wiley, H. E., Cukras, C. A., Meyerle, C. B., & Chew, E. Y. (2017). Systemic Sunitinib Malate Treatment for Advanced Juxtapapillary Retinal Hemangioblastomas Associated with von Hippel-Lindau Disease. Ophthalmology. Retina, 1(3), 181-187. https://doi.org/10.1016/j.oret.2016.10.007
Knickelbein, Jared E, et al. "Systemic Sunitinib Malate Treatment for Advanced Juxtapapillary Retinal Hemangioblastomas Associated with von Hippel-Lindau Disease." Ophthalmology. Retina vol. 1,3 (2017): 181-187. doi: https://doi.org/10.1016/j.oret.2016.10.007
Knickelbein JE, Jacobs-El N, Wong WT, Wiley HE, Cukras CA, Meyerle CB, Chew EY. Systemic Sunitinib Malate Treatment for Advanced Juxtapapillary Retinal Hemangioblastomas Associated with von Hippel-Lindau Disease. Ophthalmol Retina. 2017 May-Jun;1(3):181-187. doi: 10.1016/j.oret.2016.10.007. PMID: 28670632; PMCID: PMC5490440.
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Ophthalmol Retina. 2017 May-Jun;1(3):181-187. doi: 10.1016/j.oret.2016.10.007.

Systemic Sunitinib Malate Treatment for Advanced Juxtapapillary Retinal Hemangioblastomas Associated with von Hippel-Lindau Disease.

Ophthalmology. Retina

Jared E Knickelbein, Naima Jacobs-El, Wai T Wong, Henry E Wiley, Catherine A Cukras, Catherine B Meyerle, Emily Y Chew

Affiliations

  1. Laboratory of Immunology, National Eye Institute, National Institutes of Health, Bethesda, MD.
  2. Division of Epidemiology and Clinical Applications, National Eye Institute, National Institutes of Health, Bethesda, MD.
  3. Unit on Neuron-Glia Interactions in Retinal Disease, National Eye Institute, National Institutes of Health, Bethesda, MD.
  4. Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD.

PMID: 28670632 PMCID: PMC5490440 DOI: 10.1016/j.oret.2016.10.007

Abstract

PURPOSE: To describe the clinical course of advanced juxtapapillary retinal capillary hemangioblastomas (RCH) 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.

DESIGN: Observational case review.

PARTICIPANTS: Three patients with advanced VHL-related juxtapapillary RCH treated with systemic sunitinib malate.

METHODS: 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 RCH, and degree of exudation were recorded at each visit. Optical coherence tomography (OCT) and fluorescein angiography were also obtained at some visits.

MAIN OUTCOME MEASURES: Visual acuity, size of RCH, and degree of exudation.

RESULTS: Three patients with advanced VHL-associated juxtapapillary RCH were treated with systemic sunitinib malate. While none of the patients lost vision during therapy, treatment with sunitinib malate did not improve visual acuity or reduce the size of RCH. Improvements in RCH-associated retinal edema were observed in two patients. All patients experienced multiple adverse effects, including thyroid toxicity, thrombocytopenia, nausea, fatigue, jaundice, and muscle aches. Two of the three patients had to discontinue treatment prematurely and the third required dose reduction.

CONCLUSIONS: Systemic sunitinib malate may be useful in slowing progression of ocular disease from VHL-associated RCH. However, significant systemic adverse effects limited its use in this small series, and systemic sunitinib malate may not be safe for treatment of RCH 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 RCH.

Keywords: platelet-derived growth factor; retinal capillary hemangioblastoma; sunitinib; vascular endothelial growth factor; von Hippel-Lindau disease

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