Use of the tyrosine kinase inhibitor sunitinib in a patient with von Hippel-lindau disease: Targeting angiogenic factors in pheochromocytoma and other von Hippel-Lindau disease-related tumors

Camilo Jimenez, Maria E. Cabanillas, Libero Santarpia, Eric Jonasch, Karen L. Kyle, Elizabeth A. Lano, Surena F. Matin, Rodolfo F. Nunez, Nancy D. Perrier, Alexandria Phan, Thereasa A. Rich, Beejal Shah, Michelle D. Williams, Steven G. Waguespack

Research output: Contribution to journalArticlepeer-review

119 Scopus citations

Abstract

Context: von Hippel-Lindau disease is characterized by highly vascularized tumors of multiple organs. Evidence Acquisition:Wepresent a patient with von Hippel-Lindau disease with multiple renal and pancreatic tumors and a malignant pheochromocytoma infiltrative of the sacrum and associated with lymph nodule metastases. The pheochromocytoma expressed high protein level of vascular endothelial growth factor and platelet-derived growth factor-β receptor. The patient presented with a poor performance status, severe pelvic pain, weight loss, and manifestations of catecholamine excess. Evidence Synthesis: Treatment against malignant pheochromocytoma with surgery, chemotherapy, or participation in clinical trials was not feasible because of the patient's poor performance status, the presence of multiple tumors, and the extension of the pheochromocytoma into the bones. Patient was treated with sunitinib, a potent tyrosine kinase inhibitor of vascular endothelial growth factor, platelet-derived growth factor, RET, c-KIT, and FLT-3 receptors. Six months of treatment with sunitinib was associated with normalization of the patient's performance status and blood pressure, absence of symptoms of catecholamine excess, weight gain, disappearance of pain, shrinkage of each of the tumors (50% in the largest renal tumor,38%in the largest islet cell tumor, 21%in the pelvic malignant pheochromocytoma), and reduction of plasma normetanephrines and chromogranin A. Conclusion: This study provides evidence that targeting tyrosine kinase receptors such as the vascular endothelial growth factor pathway and the platelet-derived growth factor-β receptor may have value in the treatment of VHL-related tumors including pheochromocytoma.

Original languageEnglish (US)
Pages (from-to)386-391
Number of pages6
JournalJournal of Clinical Endocrinology and Metabolism
Volume94
Issue number2
DOIs
StatePublished - Feb 2009

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Endocrinology
  • Clinical Biochemistry
  • Biochemistry, medical

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