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A Prospective Natural History Study of VHL Patients With CNS Hemangioblastomas

8 Years
75 Years
Open (Enrolling)
Hemangioblastoma, Hippel Lindau Disease

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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.

Inclusion Criteria


Patients will be recruited from the von Hippel-Lindau (VHL) clinic at the National
Institutes of Health (NIH). All patients will have the diagnosis of von Hippel-Lindau
disease confirmed using existing established criteria and meet the following requirements:

Consenting men and women between the ages of 18 and 75, inclusive.

Males and females between the ages of 8-18 whose parent/guardian can provide permission.

Presence of one or more cerebral, cerebellar or spinal cord hemangioblastomas on screening

Provide written informed consent prior to participation in the trial.

Karnofsky Performance Scale Score greater than 60.

Capacity to undergo MRI scanning.


Clinically unstable condition.

Karnofsky Performance Scale Score less than 60.

Being treated by any chemotherapy, immunotherapy, or steroids.

Allergy to CT or MRI contrast agents.

Contraindication to MRI scanning such as surgery that involves metal clips or wires which
might be expected to cause tissue damage or produce image artifacts.

Type of Study:


Study Design:


Principal Investigator

John D Heiss, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Institute of Neurological Disorders and Stroke (NINDS)


United States: Federal Government

Study ID:




Start Date:

June 2000

Completion Date:

Related Keywords:

  • Hemangioblastoma
  • Hippel Lindau Disease
  • Brain
  • Brainstem
  • Cyst
  • Magnetic Resonance Imaging
  • Spinal Cord
  • von Hippel-Lindau
  • Von Hippel-Lindau Disease
  • Spinal Cord Neoplasms
  • Hemangioblastoma



National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland  20892