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How Von Hippel-Lindau Is Treated

Von Hippel-Lindau is a genetic disease that forms tumors. These can be treated with medication, chemotherapy, or surgery. Von Hippel-Lindau (VHL) disease is a genetic disorder in which a mutation in the VHL gene causes cells to grow abnormally. VHL is a tumor-suppressive gene. When it functions normally, the gene prevents tumors from growing.When this gene is mutated,

  • Posted on 27th Apr, 2022 11:30 AM
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Von Hippel-Lindau (VHL) disease is a genetic disorder in which a mutation in the VHL gene causes cells to grow abnormally. VHL is a tumor-suppressive gene. When it functions normally, the gene prevents tumors from growing.

When this gene is mutated, however, cells can grow abnormally. This abnormal cell growth leads to the formation of tumors in various areas of the body, some of which can be cancerous.

The most common tumors that develop in VHL are:

  • Hemangioblastomas: Noncancerous (benign) tumors that usually are found in the brain, spine, and retina (light-sensing part of the eye)
  • Pheochromocytoma: A noncancerous tumor on the adrenal gland that secretes hormones
  • Pancreatic cysts or pancreatic neuroendocrine cancer
  • Endolymphatic sac tumor: A noncancerous tumor of the inner ear
  • Kidney cysts or a type of cancer called clear cell renal cell carcinoma (ccRCC)

Although there is no cure for VHL, the symptoms it causes can be treated. Often, the most common treatment is surgery to remove the cyst or tumor causing the problem. If the tumor is cancerous, medications such as chemotherapy, immunotherapy, or targeted therapy may also need to be given.

This article will review the treatment options for someone with von Hippel-Lindau disease. 

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Home Remedies and Lifestyle 

There aren’t any home remedies that will treat VHL or the tumors that it causes. However, maintaining a healthy lifestyle can be helpful. Lifestyle factors that can be important for overall health in someone with VHL include:

  • Not smoking: Smoking can increase the risk of developing cancer.
  • Physical activity: Adults should get about 30 minutes of activity every day and should limit the amount of time spent sitting around doing things such as watching TV.
  • Healthy diet: Eating a healthy diet can be important in cancer prevention. Sugary drinks should be avoided. Fresh fruits and vegetables and whole grains should be eaten daily. Limiting alcohol intake can be helpful as well.

Over-the-Counter (OTC) Therapies 


There are no over-the-counter treatments for VHL. However, discuss any OTC medications or supplements with your healthcare team. Some may interact with medications given for VHL.

Prescriptions 

In 2021, the first prescription medication called Welireg (belzutifan) was approved for adults with VHL who have hemangioblastomas in the brain, pancreatic neuroendocrine tumors, or renal cell carcinoma. It can be used when treatment is needed for these tumors, but they don’t require immediate surgery.

Welireg works by inhibiting hypoxia-inducible factor-2 alpha, which is a pathway cells use to grow abnormally. By blocking this pathway, the tumors aren’t able to have an adequate blood supply to continue to grow.

In people with neuroendocrine tumors of the pancreas, treatment may require medications, including:

  • Somatuline Depot (lanreotide)
  • Sandostatin (octreotide)
  • Zortress (everolimus)
  • Sutent (sunitinib)
  • Chemotherapy 


For people with renal cell carcinoma, treatment can include:

  • Chemotherapy
  • Immunotherapy (uses the immune system to fight the tumor)
  • Inlyta (axitinib)
  • Sutent (sunitinib)
  • Votrient (pazopanib)
  • Cabometyx (cabozantinib)
  • Lenvima (lenvatinib)
  • Zortress (everolimus)
  • Torisel (temsirolimus)
  • Avastin (bevacizumab)

Surgeries and Specialist-Driven Procedures 

Routine imaging is a standard practice for those with VHL. Routine imaging with computed tomography (CT) scans or magnetic resonance imaging (MRI) can be helpful to find tumors when they are small and may be treated before they cause significant problems.

For brain or spinal cord hemangioblastomas or cysts, surgical removal of the entire lesion may be required. For cysts, which are filled with fluid, it is important for the surgeon to remove the entire cyst, as it can fill with fluid again if any of the cyst is left behind.

Another potential treatment for these tumors is called stereotactic radiation, in which precise beams of radiation are delivered to the tumors to damage the DNA in the cells instead of removing them surgically.

For renal cell carcinoma, either part of the kidney or the entire kidney may need to be removed surgically. Localized treatment, such as cryoablation or radiofrequency ablation may be done when kidney tumors are small.

During cryoablation, special equipment is used to freeze the tumor to kill the cells. During radiofrequency ablation, a similar procedure is used to burn the cancer cells.

Surgery is often required for the hormone-producing pheochromocytomas on the adrenal glands, even if they aren’t causing any symptoms.

Those with endolymphatic sac tumors in the inner ear must undergo surgical removal of the tumors to prevent hearing loss. These tumors can also cause balance problems and surgical removal of the tumor can prevent balance from getting worse.

Complementary and Alternative Medicine (CAM) 

No complementary or alternative medicine practices have been proven to treat VHL. Before using any CAM, discuss it with your healthcare team. Some CAM treatments may interact with medications and procedures you are undergoing.

Summary

VHL is a genetic disorder that doesn’t have a cure. It requires routine imaging of the body to evaluate for the presence of tumors.

Maintaining a healthy lifestyle through a nutritious diet and exercise and refraining from smoking are important for overall health for those with VHL.

A medication called Welireg was approved in 2021 to treat some tumors caused by VHL. Other medications that can treat the cancers caused by VHL are forms of chemotherapy, immunotherapy, and targeted therapy.

Surgery is a common intervention for VHL. It is used to treat many of the tumors and cysts caused by this disease.

A Word From Verywell 

It can be stressful living with VHL, and wondering about the possibility of developing cancer someday can be scary. Keeping up with the imaging tests ordered by your healthcare provider is important in catching tumors early, before they become large and start causing symptoms or other problems.

It is also important to notify your healthcare team of any new symptoms that develop, and let them know of any concerns you have. 

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