Epithelioid Hemangioendothelioma (EHE) | Diagnosis & Treatment

How do doctors diagnose epithelioid hemangioendothelioma?

Diagnosing epithelioid hemangioendothelioma (EHE) is a process that happens in a few stages, in part because the skin lesions associated with EHE are often confused with more common skin conditions. Most often, the first step will be a complete medical history and thorough physical exam. Additional tests may include:

Certain characteristics of your child’s EHE may determine whether it requires more aggressive treatment, including:

  • its location
  • the appearance of a biopsy under a microscope
  • whether the tumor has changed in size or spread to other tissues

What are the treatment options for epithelioid hemangioendothelioma?

Your child’s treatment depends on the size and location of the tumor and whether there are multiple tumor sites.

In rare cases, the tumor may shrink or go away without treatment. If not, there are multiple treatment options available. Your child’s care team will discuss the benefits of each one. They include:

  • Watchful waiting: If your child has no symptoms and there are no signs the tumor is progressing, the doctor may want to observe the tumor for a period of time to determine whether treatment is necessary
  • Surgery: Small or easily accessible tumors may be fully removed. Tumors completely removed with surgery most likely do not recur. Sometimes, complete removal is not possible due to the size, location or presence of multiple tumor sites. In these cases, remaining tumors may require further therapy and can come back after treatment
  • Medication management: A customized drug therapy plan can help relieve symptoms and prevent further growth of tumors that can’t be completely removed with surgery

Medications

Currently, no drug specifically targets EHE cells. However, some tumors shrink and even become invisible with therapy. Some of these medications are categorized as “chemotherapy,” meaning they may also be used for some types of cancer.

Medications used to treat EHE include:

  • Sirolimus: Also known as rapamycin, this oral medication suppresses the immune system and slows the growth of abnormal lymphatic vessels that form the tumor. This can help shrink EHE tumors and improve symptoms, including pain
  • Tyrosine kinase inhibitors: These drugs, designed as targeted therapies for cancers, have shown short-term success with EHE. Examples include sorafenib, sunitinib and pazopanib.
  • Vincristine: This chemotherapy drug targets all dividing cells within the body and is therefore used to treat many cancers. It is also used for aggressive benign vascular tumors.
  • Interferon: The body produces interferon to combat infections or control inflammation. It has been formulated into a medication that targets blood vessel growth.
  • Multi-agent chemotherapy: EHE tumors that grow rapidly, spread to other tissues or do not respond to other medications may require more aggressive drug therapy. However, this combination of medications is rarely needed in children and young adults with EHE.

How are aggressive epithelioid hemangioendothelioma tumors treated?

In very aggressive cases, or when tumors do not respond to these therapies, additional treatment options may include:

  • Embolization: This minimally invasive procedure blocks the main artery that feeds the tumor. With the blood supply cut off, the tumor will often shrink. We do not attempt embolization in cases where several arteries feed into the tumor, which is often the case with EHE.
  • Radiation: If we can’t remove your child’s tumor surgically and it doesn’t respond to chemotherapy or other medications, we may consider using radiation therapy in rare cases for solitary tumors or individual symptomatic tumors.

What is the follow-up care for epithelioid hemangioendothelioma?

Your child should have regular follow-up appointments during and after treatment to check for:

  • recurrence of the tumor
  • any drops in platelet count
  • growth of the lesion

Your child’s care team will give you a schedule of follow-up care to see how your child is responding to therapy and to monitor for any late effects of therapy.

What is the long-term outlook for children with epithelioid hemangioendothelioma?

EHE is such a rare condition and behaves so differently from person to person that it’s difficult to determine the long-term outlook for your child.

The prognosis for your child greatly depends on:

  • the extent of the disease (how large the tumor is and whether it has spread)
  • the size and location of the tumor, which determine whether it can be surgically removed (tumors arising in the liver and those that are in multiple locations may be more aggressive)
  • the results of the biopsied tissue (areas of cell death and markers of cell replication may indicate more aggressive tumors)
  • response to therapy
  • the overall health of your child
  • your child's tolerance of specific medications, procedures, or therapies
  • new developments in treatment