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Evaluation of the Natural History and Management of Pancreatic Lesions Associated With Von Hippel-Lindau

12 Years
Open (Enrolling)
Pancreatic Lesions

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Trial Information

Evaluation of the Natural History and Management of Pancreatic Lesions Associated With Von Hippel-Lindau


Patients with the familial cancer syndrome von Hippel-Lindau (VHL) demonstrate
manifestations in a variety of organs among them the pancreas. Pancreatic manifestations can
range from benign cysts and micro cystic adenomas to neuroendocrine tumors of the pancreas
which are capable of regional and distant spread. These neuroendocrine tumors can result in
life-threatening complications.

This protocol is designed to identify VHL patients with pancreatic manifestations and to
follow these patients with serial imaging studies and germ line and tissue genetic analysis.


Primary Objectives

To identify patients with VHL having pancreatic lesions defined by simple cysts, microcystic
adenomas, neuroendocrine tumors and other solid lesions of the pancreas.

To follow patients with VHL and pancreatic manifestations by serial examination with
non-invasive imaging studies.

For patients with solid lesions of the pancreas, to determine the rate of growth and to
correlate the growth rate with clinical measures of disease progression.

To validate non-invasive imaging methods for differentiating benign solid lesions from
lesions with malignant potential.

To characterize the time from initial presentation with pancreatic tumors to the time that
surgery is recommended.

Secondary Objectives:

To obtain blood samples from patients to determine VHL mutation status and subtype the
mutations for potential correlation with disease severity.

When possible, to obtain tissue from pancreatic lesions for genetic analysis including CGH,
tissue proteomics, and cDNA microarray analysis.


Patients greater than or equal to 12 years of age who have been diagnosed with VHL.

Patients/parent must be able to sign an informed consent and be willing to return to NIH for


Demographic data will be collected from the medical record and patient interview for each
patient participant. Data will be securely stored in a computerized database.

Patients will be evaluated by the Urologic Oncology Branch personnel as indicated to rule
out or manage other manifestations of VHL. Imaging studies of regions other than the chest
and abdomen will be dictated by best clinical practice for the workup and management of VHL
manifestations as has been previously published.

All patients enrolled on this study will be offered genetic counseling by a trained genetic

After their initial on-study evaluation, patients who are not found to have solid lesions of
the pancreas but rather have only cystic disease of the pancreas, will be re-screened every
two years with non-invasive imaging studies.

Surgical resection of solid lesions of the pancreas will be recommended based on previously
published criteria.

Based on our analysis of likelihood of tumor growth or risk of metastasis, data will be
analyzed every two years and appropriate revisions will be made to the surgical management
guidelines, if indicated by data analysis.

Projected accrual will be 20 patients per year for a total of 15 years. Thus, we anticipate
accruing 300 patients on this protocol.

Inclusion Criteria


Patients who have been diagnosed with VHL using the following criteria: either germ line
analysis (12) or clinical criteria and a family history (8, 12) and who have at least 1
pancreatic manifestation of VHL as documented on any non-invasive imaging study. These
manifestations include:

1. Pancreatic cyst(s).

2. Solid lesions suspicious for microcystic adenoma(s).

3. Solid enhancing lesions suspicious for PNET(s).

4. Any other solid lesion(s) of the pancreas.

Age greater than or equal to 12 years of age.

Patients must be willing to return to NIH for follow-up.

Patients/parent must be able to sign an informed consent.


Patients unwilling to return to the NIH for follow-up.

Patients unwilling to undergo serial non-invasive imaging.

Patients without evidence of pancreatic lesions.

Type of Study:


Study Design:


Principal Investigator

Electron Kebebew, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Cancer Institute (NCI)


United States: Federal Government

Study ID:




Start Date:

April 2003

Completion Date:

Related Keywords:

  • Pancreatic Lesions
  • Imaging Studies
  • Rate of Tumor Growth
  • Familial Cancer Syndrome
  • Surgical Resection
  • Von Hippel-Lindau Disease



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