Monotherapy with thalidomide for treatment of spinal cord hemangioblastomas in a patient with von Hippel-Lindau disease.

Von Hippel-Lindau (VHL) disease is a cancer-prone syndrome characterized by abnormalities in vascular proliferation and the development of both the visceral and CNS tumors. Complications from hemangioblastoma are among the principal causes of death from this syndrome. Antiangiogenic therapy has been used with different modalities in patients suffering from such complications. Here, we describe an adolescent with VHL complicated by progressive, multifocal spinal hemangioblastomas. Treatment with single-agent thalidomide over the course of 3 years was associated with an unexpected stabilization of the disease. The antiangiogenic effect of thalidomide may be associated with the control of progressive hemangioblastoma. (c) 2009 Wiley-Liss, Inc.