Trial Information

A Prospective Natural History Study of VHL Patients With CNS Hemangioblastomas

Hemangioblastomas of the cerebellum, brainstem and spinal cord are frequent tumors in
patients with von Hippel-Lindau (VHL) disease. Rarely, these lesions also affect the
cerebrum. Patients often have multiple lesions, many of which are associated with cysts or
syrinx. The current treatment for symptomatic lesions is surgical resection. Focused
radiation is also being used in selected cases in an attempt to provide tumor control. The
natural history of central nervous system (CNS) lesions in patients with VHL has not been
addressed in a prospective study. It is not clear at which point these lesions will begin to
grow, or develop cysts in the cerebellum or syrinx in the spinal cord, and systemic factors
that influence tumor growth have not been identified. By identifying factors that predict or
influence tumor progression or cyst development, we can more accurately recommend surgical
or medical intervention at appropriate times and avoid unnecessary treatment for stable
lesions. This study will collect prospective radiological and clinical data on growth of the
central nervous system (CNS) hemangioblastomas and associated cysts. We will also
prospectively collect information on systemic processes that may influence tumor
progression, such as puberty, menopause, pregnancy, effects of hormone therapy, tumor load,
serum VEGF levels, hemoglobin/hematocrit levels, and erythropoietin.