Treatments for Kaposiform Hemangioendothelioma in Children

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Treatments for kaposiform hemangioendothelioma in children

What are the treatment options for KHE?

The treatment goals with kaposiform hemangioendothelioma (KHE) are to relieve symptoms, reverse KMP if it is present, and shrink the tumor. Ideally, thorough treatment will prevent problems later in life, including tumor recurrence. Some tumors shrink and even become invisible with therapy.
KHE treatment takes an interdisciplinary, team-based approach. Often the team is led by a hematologist/oncologist. Some or all of the following specialists may be involved as well:

  • dermatologist
  • plastic surgeon
  • general surgeon
  • interventional radiologist

Your care team will outline the benefits of all the KHE treatment options listed below:

Observation — For superficial tumors without low platelets, your child’s doctor may want to simply observe the tumor for a period of time. This allows your child’s care team to determine how the tumor is behaving.

Some mild KHE tumors go away without treatment, so if we observe the tumor for a few months and see that it’s shrinking, we probably won’t recommend any treatment.

However, if a KHE tumor grows, we can immediately step in with aggressive treatment to stabilize, shrink or remove it.

Steroids — In mild cases, your doctor may recommend treating the tumor with oral steroids first. While only about 12 percent of KHE tumors respond to steroids, oral steroids are safe and may help other treatments work better.

Some patients with superficial lesions may be treated with medicated creams or lotions, including creams containing steroids or timolol. It is unclear whether these ointments offer benefit, however, as such lesions often improve in time without therapy.

Other medications — Some of the commonly recommended medications for KHE are categorized as “chemotherapy,” meaning they may also be used for cancer treatment. But individual medications on this list do not lead to all the side effects many people associate with chemotherapy. Your doctor will explain the common and uncommon side effects of the particular medications relevant to your child’s case. Two drugs that may be considered are:

  • Vincristine — This chemotherapy drug targets all dividing cells within the body and is therefore used to treat many cancers. While KHE is not a cancer, there are dividing cells in the tumor. Vincristine has been used successfully for decades to treat KHE.
  • Sirolimus — This drug appears to target blood vessel growth. The VAC is rapidly gaining experience using sirolimus to treat patients with vascular anomalies, and has recommended sirolimus for patients with KHE both here and at other centers around the world with promising early results.
It is not yet known whether one of these two drugs is a better option than the other for treating KHE. Researchers are currently exploring that question in a clinical trial.

During therapy, it's best for an experienced hematology/oncology team to monitor your child's blood counts. To do so, we work closely with the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

Other treatments for KHE

In very aggressive cases, or in situations where a tumor doesn’t respond to any of these therapies, your doctor may consider:

  • Surgery — Your doctor will only recommend surgery if your child’s tumor is very small (and we’re able to completely remove it) or extremely aggressive. The location and size of some KHE tumors make them quite difficult to remove; in such cases, surgery is avoided because it might do more harm than good.
  • Embolization — This is a process in which a doctor blocks the main artery that’s feeding your child’s tumor. With the blood supply cut off, the tumor will often shrink. However, KHE is most often fed by a number of arteries instead of one main artery; if that’s the case, we usually don’t attempt embolization.
  • Radiation — This was historically used to try to control the growth of these tumors, but it is associated with significant short- and long-term side effects. In very rare cases we may consider using it in conjunction with one of the other treatments.

Is platelet transfusion helpful?

Very rarely. KHE will quickly trap new platelets, causing the tumor to quickly swell and possibly become painful. However, platelet transfusions can be given to stop significant bleeding or prevent bleeding during necessary procedures.

It’s important to treat the tumor, not the platelet count. We focus on shrinking the tumor, understanding that a child’s platelet count will go up as the tumor shrinks, because it’s not trapping as many platelets.

Will my child need follow-up care for KHE?

Your child should have regular follow-up during and after treatment to check for recurrence of the tumor, any drops in platelet count, or growth of the lesion. Your child’s health 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.
If we succeed in shrinking the tumor, there may be some residual marks on your child’s skin. Depending on where they are and how large they are, scarring can sometimes limit your child’s movement or cause discomfort. You may want to consult with a plastic surgeon to discuss corrective options.

Other specialists that care for patients with KHE

  • Physical therapists — Sometimes the tumor can affect surrounding tissue and muscles, making it hard or painful for your child to move an arm or a leg. A physical therapist can help ease the pain and increase your child’s range of movement, strength and flexibility. Physical therapists can also provide care with compression wrapping, which is helpful in some patients.
  • Occupational therapists — Children with KHE sometimes get behind on developing motor skills because of physical problems related to their tumors. An occupational therapist can get your child back on track.
  • Counselors — As your child gets older, she may be uncomfortable with the skin lesion, especially if it’s in a prominent location such as on the face or neck. Our counselors can help your child deal with the psychological and social issues related to having a birthmark. Counselors often have important roles for siblings of patients as well.

What is the long-term outlook for my child?

Many patients have an excellent long-term outlook through successful KHE treatment. KHE is a very rare tumor and we are actively researching how to predict long-term outcomes from this lesion. In general, you child's prognosis depends on:

  • how quickly the diagnosis is made and appropriate medical therapy is started
  • the size, location and extent of disease
  • the tumor's initial response to therapy

It’s important to see a specialist quickly when KHE is suspected to allow early discussions about treatment. Specialists can then attempt to shrink the tumor before it
affects muscles or other tissues nearby. Early treatment could also help reduce long-term complications by preventing further growth.

Will my child’s tumor come back after treatment?

There is some evidence that these tumors can recur, especially if initial treatment is incomplete. However, if your child’s tumor does recur, we have good success treating it again, reducing its size and stabilizing your child’s platelet count.

Kaposiform hemangioendothelioma: Reviewed by Cameron C. Trenor III, MD, © Boston Children's Hospital, 2016

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