Single-Arm Nonrandomized Phase II study of Pazopanib in Patients with von Hippel-Lindau Disease

摘要

Background:No approved systemic therapy exists for von Hippel-Lindau disease, an autosomal dominant disorder with pleiotropic organ manifestations, including clear cell renal cell carcinomas, retinal, cerebellar and spinal hemangioblastomas, pheochromocytomas, pancreatic serous cystadenomas, and pancreatic neuroendocrine tumors. This prospective phase II study was designed to assess the efficacy and safety of pazopanib in patients with von Hippel-Lindau disease.