How Von Hippel-Lindau Disease Is Treated

If you are diagnosed with Von Hippel-Lindau (VHL) disease, tumors or cysts may grow in up to 10 areas of the body. Your treatment plan will depend on the problems these growths cause. Surgery is the main treatment for most VHL-associated tumors.

Treatment options will be determined by your symptoms, test results, imaging studies, and overall health. In this article, the treatments for VHL disease will be explored in detail.

Hands of operating room staff performing surgery

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Active Surveillance

The VHL mutation that causes tumors to form cannot be repaired or prevented. However, the complications of VHL syndrome can be managed with active surveillance, closely monitoring progress through imaging scans and lab tests. When tumors are caught early, treatment is often more successful.

If tumors are small and/or slow growing, they may not need treatment. Your doctor will observe the tumor with scans and determine if and when treatment is necessary. 

Surgeries and Specialist-Driven Procedures

People who have VHL disease may experience tumors and/or cysts in up to ten parts of the body, including the brain, spine, eyes, kidneys, pancreas, adrenal glands, inner ears, reproductive tract, liver, and lung.

The type of surgery you'll need will depend on where the VHL-related tumors or cysts are in your body.

Hemangioblastoma

Hemangioblastomas are benign (noncancerous) tumors that are found in the brain, spinal cord, and eye.

If hemangioblastomas are small and grow slowly, they may not need treatment. Tumors that are large, grow fast, or cause symptoms can be surgically removed or treated with focused radiation therapy.

Pheochromocytoma

A pheochromocytoma is a type of tumor found in the adrenal glands or certain nerve cells. These are usually noncancerous. Removal of a pheochromocytoma may require taking out the whole adrenal gland. This is called an adrenalectomy.

Kidney Cancer

VHL increases the risk of developing renal cell carcinoma, (RCC), a type of kidney cancer. Kidney tumors are usually surgically removed when they grow larger than 3 centimeters in diameter or if they grow quickly.

Treatment options include:

  • Partial or complete nephrectomy, surgical removal of part or all of the kidney
  • Radiofrequency ablation, which uses radiofrequency waves to destroy the tumor
  • Cryotherapy, which uses extreme cold to remove tissue

Eye Tumors

It is important that eye problems caused by VHL receive early diagnosis and treatment. This helps reduce the chance of vision loss.

Tumors found on the outer regions of the retina can be treated with laser photocoagulation (treatment with laser beams that heat and destroy tumors), or cryotherapy (treatment with extremely cold probes that destroy the tumor).

Pancreatic Cysts and Tumors

Cysts in the pancreas do not usually require treatment. Rarely, if a cyst becomes very large, your doctor may shrink it by using a needle to draw fluid from the cyst.

Pancreatic neuroendocrine tumors require close follow-up because they may spread to other parts of the body, which can be very dangerous. Generally, if a suspicious pancreatic neuroendocrine tumor becomes larger than 3 centimeters, it is surgically removed.

Recap

Surgery is the main treatment for VHL-related tumors. Every surgical treatment comes with some risk. Your doctor will talk to you about the potential risks and side effects of surgical and specialist procedures.

Prescriptions

A new drug approved by the Food and Drug Administration (FDA) in August 2021 may help people with VHL avoid or delay surgery by shrinking their tumors.

The FDA approved Welireg (belzutifan) to treat VHL-associated renal cell carcinoma, central nervous system hemangioblastomas, and pancreatic neuroendocrine tumors that don’t require immediate surgery.

The approval is based on results from a small clinical trial that tested Welireg in people with VHL-associated renal cell carcinoma. All participants also had other VHL-associated tumors.

After 18 months, nearly half of the participants had kidney tumor shrinkage of at least 30%, and most of those people’s tumors were still responding after one year. Welireg also shrank VHL-associated brain, pancreatic, and eye tumors.  

Recap

A new drug called Welireg (belzutifan) was approved by the FDA in August 2021 to help treat certain VHL tumors in the kidney, central nervous system, and pancreas. It showed promising results in clinical trials.

Over-the-Counter (OTC) Therapies

Currently, there are no over-the-counter therapies designed to treat VHL. Any OTC medications (including herbal remedies) will need to be discussed with your physician to check that they won't interfere with your treatment plan.

Lifestyle

Family history is the main risk factor of VHL. Research into the role that lifestyle factors play in the growth of these tumors is ongoing.

Because VHL is also associated with some cancerous tumors, those who are diagnosed with this genetic syndrome are encouraged to quit smoking, maintain a healthy weight, exercise routinely, and limit alcohol to decrease their cancer risk. 

Complementary and Alternative Medicine (CAM)

No alternative therapies have been shown to improve the outcome of VHL treatment. However, certain mind-body interventions such as meditation and journaling may be useful in helping patients navigate the mental and emotional challenges that can arise when undergoing treatment for VHL-related tumors.

Summary

Surgery is the main treatment for most VHL-associated tumors. The type of surgery you have will depend on the location of the tumors. A new medication, Welireg, was recently approved by the FDA for the treatment of some VHL tumors. This medication may work to shrink tumors.

A Word From Verywell

Finding out you have tumors or cysts growing as a result of VHL can be frightening but there are treatment options available.

Surgery and radiation therapy are popular treatments, depending on the site of the tumor. Sometimes, if the tumor is slow growing and not causing any symptoms, your doctor may recommend regular screening tests before undertaking any treatment.

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4 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
  1. National Cancer Institute. Von Hippel-Lindau disease (PDQ)–health professional version. Updated November 12, 2021.

  2. Goyal N, Agrawal D, Singla R, Kale SS, Singh M, Sharma BS. Stereotactic radiosurgery in hemangioblastoma: Experience over 14 yearsJ Neurosci Rural Pract. 2016;7(1):23-27. doi:10.4103/0976-3147.172165

  3. Escudier B, Porta C, Schmidinger M, et al. Renal cell carcinoma: ESMO clinical practice guidelines for diagnosis, treatment and follow-upAnnals of Oncology. 2019;30(5):706-720. doi:10.1093/annonc/mdz056

  4. American Society of Clinical Oncology. Kidney cancer: types of treatment. Updated October 2020.