N/A
N=340
Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau Disease
Bottom Line
View on ClinicalTrials.gov: NCT00062166 ↗Enrolled (actual)
340
Serious AEs
6.5%
Results posted
Nov 2018
Primary outcome: Primary: Number of Patients With Pancreatic Lesions Defined by Simple Cysts, Microcystic Adenomas, Neuroendocrine Tumors & Other Solid Lesions of the Pancreas Who Had Significant Growth in Lesions or Symptoms Related to the Lesions Requiring Surgical Intervention — 47 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- —
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- National Cancer Institute (NCI)
- Primary completion
- Nov 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Pancreatic Lesions Defined by Simple Cysts, Microcystic Adenomas, Neuroendocrine Tumors & Other Solid Lesions of the Pancreas Who Had Significant Growth in Lesions or Symptoms Related to the Lesions Requiring Surgical Intervention |
47 | — |
| SECONDARY Percentage of Participants With Exon 3 Mutation Compared to Participants With Exon 1 or 2 Von Hippel Lindau (VHL) Mutations Who Required an Intervention |
53.97; 46.03 | 0.02 sig |
| SECONDARY Number of Participants From Which We Obtained Tissue From Pancreatic Lesions and Normal Tissue for Genetic Analysis |
46 | — |
| SECONDARY Count of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0) |
22 | — |
| SECONDARY Number of Participants With Missense or Non-missense Mutations |
76; 80 | — |
Summary
Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. These tumors are more aggressive in some people than in others. To learn more about this disease, its genetic cause and how best to treat it, this study will 1) identify patients with VHL who have pancreatic lesions; 2) examine the characteristics of the lesions and how fast they grow; 3) study how well imaging tests can reveal lesion characteristics that will help in diagnosis; and 4) perform genetic studies using blood and, when possible, tissue samples.
Patients 12 years of age and older with VHL involving the pancreas may be eligible for this study. Participants will undergo some or all of the following tests and procedures:
* Interviews with a cancer doctor, cancer nurses, and a surgeon (if surgery is recommended).
* Computed tomography (CT) scan of the abdomen, chest, or pelvis. This test uses x-rays to produce images of body tissues and organs in small sections.
* Magnetic resonance imaging (MRI) of the abdomen. This test uses radio waves and a strong magnetic field to produce images of body tissues and organs.
* Ultrasound of the abdomen. This test uses sound waves to create images body tissues and organs.
* Blood tests for routine laboratory chemistries, for tests specific to the pancreas, and for genetic studies
* 24-hour urine studies
After the tests are completed, the doctor will discuss the results with the patient. Patients with a pancreatic tumor that requires surgery will be offered the option of an operation to remove as much tumor as possible. Patients with lesions that are not appropriate for surgery will be asked to return to National Institutes of Health (NIH) for scans and x-rays every year to monitor growth of the lesions. If surgery should become advisable in the future, the option will be discussed at that time. Patients with pancreatic cysts will be asked to return to NIH every 2 years for scans and x-rays to monitor their condition.
Eligibility Criteria
- INCLUSION CRITERIA:
Patients who have been diagnosed with Von Hippel Lindau (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:
- Pancreatic cyst(s).
- Solid lesions suspicious for microcystic adenoma(s).
- Solid enhancing lesions suspicious for primitive neuroectodermal tumor (PNET)(s).
- 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 National Institutes of Health (NIH) for follow-up.
Patients/parent must be able to sign an informed consent.
EXCLUSION CRITERIA
Patients unwilling to undergo serial non-invasive imaging.
Data sourced from ClinicalTrials.gov (NCT00062166). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.