COT-02 TREATMENT EXPERIENCE OF PAZOPANIB FOR A CASE OF VON-HIPPEL LINDAU DISEASE

Abstract

INTRODUCTIONvon Hippel-Lindau (VHL) disease is an autosomal dominant genetic disorder associated with neoplastic lesions in multiple organs. Here, we report our experience with a patient with VHL disease presenting with complications of renal tumors, wherein pancreatic cystic lesions and renal neoplastic lesions were reduced in size with the administration of pazopanib at our department.PATIENTThe patient was a 26-year-old man who presented with hiccups and was diagnosed with medullary hemangioblastoma with a cyst that was resected. The other central nervous system lesions were located in the right optic nerve sheath, cervical and thoracic spinal cord, with multiple cystic type tumors in the pancreas, renal tumors, and epididymal tumors. Although the family history was unclear, the clinical diagnostic criteria for VHL disease were met, and mutations were found in the VHL gene analysis. Six months after the initiation of pazopanib therapy at a dose of 800 mg/day, there was no remarkable change in the hemangioblastoma on imaging; however, the pancreatic and renal lesions had shrunk. In addition, new lesions did not appear. Adverse events included diarrhea, graying of hair.DISCUSSIONPazopanib is a multi-tyrosine kinase inhibitor that inhibits angiogenesis and inhibits tumor growth. In VHL disease, pancreatic and renal tumors influence the survival prognosis, and for hemangioblastoma, the lesions increase in number and size and the corresponding surgery affects the functional prognosis. Although there was a poor tumor-reduction effect on the hemangioblastoma, there was a supposed inhibitory effect on the appearance of new lesions and the enlargement of the existing lesions.CONCLUSIONPazopanib administration resulted in the shrinkage or regression of pancreatic and renal lesions. In addition, it inhibited the increase in number and size of hemangioblastomas. Further, prolonging the surgical treatment interval may help maintain the patient’s quality of life.