Abstract
von Hippel-Lindau (VHL) disease, caused by germline mutations in the VHL gene, is a hereditary autosomal-dominant disorder which predisposes the individual to various malignant and benign tumors. VHL acts as a tumor suppressor, mainly through the negative regulation of hypoxia-inducible factors. Molecular-targeted drugs against vascular endothelial growth factor-signaling pathways, a target of hypoxia-inducible factors, have recently been introduced into clinical practice for the treatment of patients with sporadic renal cell carcinoma and pancreatic neuroendocrine tumors. However, whether such treatments are effective in patients with VHL disease remains to be elucidated. We herein report a Japanese patient with VHL disease who was successfully treated with sunitinib for approximately 5 years.
MeSH terms
-
Adenoma, Islet Cell / complications
-
Adenoma, Islet Cell / diagnosis
-
Adenoma, Islet Cell / drug therapy*
-
Adult
-
Antineoplastic Agents / administration & dosage*
-
Antineoplastic Agents / pharmacology
-
Carcinoma, Renal Cell / complications
-
Carcinoma, Renal Cell / diagnosis
-
Carcinoma, Renal Cell / drug therapy*
-
Drug Administration Schedule
-
Fatal Outcome
-
Genes, Tumor Suppressor / drug effects
-
Germ-Line Mutation / drug effects
-
Humans
-
Indoles / administration & dosage*
-
Indoles / pharmacology
-
Kidney Neoplasms / complications
-
Kidney Neoplasms / diagnosis
-
Kidney Neoplasms / drug therapy*
-
Male
-
Pancreatic Neoplasms / complications
-
Pancreatic Neoplasms / diagnosis
-
Pancreatic Neoplasms / drug therapy*
-
Pyrroles / administration & dosage*
-
Pyrroles / pharmacology
-
Signal Transduction / drug effects
-
Sunitinib
-
Time Factors
-
Vascular Endothelial Growth Factor A / antagonists & inhibitors*
-
von Hippel-Lindau Disease / complications*
Substances
-
Antineoplastic Agents
-
Indoles
-
Pyrroles
-
VEGFA protein, human
-
Vascular Endothelial Growth Factor A
-
Sunitinib