Kaposiform hemangioendothelioma

Having identified your child’s condition, we’re able to begin the process of treating her, so that we may ultimately return your child to good health. At Children’s Hospital Boston, we consider you and your child integral parts of the care team and not simply recipients of care. You and your care team will work together to customize a plan of care for your child.

What are the treatment options for my child?

Kaposiform hemangioendothelioma (KHE) is a complex condition, so your child will likely be treated by some or all of the following specialists:

• hematologist-oncologist
• dermatologist
• plastic surgeon
• general surgeon
• interventional radiologist

Your care team (your child’s doctor and an experienced vascular anomalies specialist) will outline the benefits of all the treatment options listed below with you.

Observation — If your child has a mild lesion with no pain and a normal platelet count, your doctor may want to simply observe the tumor for a period of time.

Steroids — Your doctor may recommend treating the tumor with steroids first. While they’re not effective in most cases (around 12 percent of the tumors respond to steroids), the treatment is easier (taken by mouth) than other options and is well tolerated.

Chemotherapy — If steroids don’t work, your doctor may recommend chemotherapy to slow the tumor by interfering with the growth of blood vessel cells. While the word “chemotherapy” can be scary, rest assured that the treatment for KHE has few of the side effects of traditional chemotherapy (no hair loss and minimal or no nausea or effects on the immune system).

The first chemotherapy drug we recommend is vincristine. Vincristine slows down the growth of the tumor and can even stop growth or shrink tumors in some cases.

• We give children chemotherapy through an IV central line (also called a central venous catheter).
  • A surgeon will implant a small port-a-cath (you won’t see it, but will be able to feel a bump) in your child’s chest, which allows the medicine to go directly into a vein.
  • It’s a minor surgery in which the surgeon makes a small incision and then inserts the port, linking it to a large vein in your child’s chest.
  • Once the course of treatment is completed, a surgeon will remove the port-a-cath.

• Every one to two weeks for about six months, your child will receive an IV dose of vincristine.
  • This is administered by experienced nurses and physicians either at Children’s or through our partnership with the Jimmy Fund Clinic next door at the Dana-Farber Cancer Institute. This therapy can also be coordinated with a clinic or hospital convenient for you.

Most children tolerate vincristine very well. Your care team will explain the few, minor side effects your child may experience. Being able to anticipate these side effects can help you prepare for, and, in some cases, prevent these symptoms from occurring.

Interferon therapy — In cases where vincristine is not effective and your child is at least 12 months old, your doctor may recommend injections of interferon. Interferon is a drug that is used for a variety of disorders to limit new blood vessel formation and reduce blood supply to the tumor.

The regimen and length of therapy is variable and is customized for your child by your doctor and care team. Injections can be given daily, three times a week or weekly and are usually continued until your child’s platelet count rises and the pain around the lesion subsides.

Other treatments

In very aggressive cases (or situations where your child’s tumor doesn’t respond to any of these therapies), your doctor has other options:

• Clinical trials — As the home of the largest vascular anomalies center in the world, we often have access to new drugs involved in clinical trials.You can search current and upcoming clinical trials here at Children’s.
• Embolization — This is a process in which a doctor inserts a block into 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; and if that’s the case, embolization is less successful and we usually don’t attempt it.
• 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. Because of the location and size of these tumors, they are quite difficult to remove and the surgery can often cause more harm than good.
• Radiation — This was historically used to try to control the growth of these tumors, but is associated with significant short- and long-term side effects. We may consider using it in conjunction with one of the other treatments in very rare cases.

My child’s doctor recommended a platelet transfusion. Is that a good idea?

Very rarely. What happens in a platelet transfusion is that the new platelets will be quickly attracted to the tumor and trapped.

• Your child’s lesion will quickly grow and may become extremely painful.
  • Over time, the lesion will shrink back to its normal size over time, but your child’s platelet count will not improve because of the transfusion.

We only recommend platelet transfusion if your child is bleeding badly.

• It’s important to treat the tumor, not the platelet count.
  • Your child’s platelet count will go up as the tumor shrinks (because it’s not trapping as many platelets).
  • We focus on shrinking the tumor, understanding that the platelet count will normalize as the tumor becomes less aggressive.

Supportive care

We want to maximize the safety and effectiveness of whatever therapy you and your child’s doctor choose. Supportive care involves preventing and treating infections and side effects of treatment to keep your child as comfortable as possible while we’re working to shrink the tumor.

Follow-up care

Your child should have regular follow-up during and after treatment to check for recurrence of the tumor, a drop in platelet count or a worsening 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.

We’ll also want to monitor for any problems because of the tissue involved as the tumor grows. Sometimes, there can be scarring that could limit your child’s movement or cause discomfort. Clinic visits after therapy are also important to monitor for any late effects of therapy.

After we have succeeded in shrinking the tumor, there are often some residual marks on your child’s skin. Depending on where they are and how large they are, you may want to consult with one of our plastic surgeons to discuss corrective options.

Rehabilitation

Children with KHE often need multidisciplinary care. Each of these disciplines is available at Children’s with experts dedicated to the care of children with complex medical conditions. Some specialists your doctor may recommend for your child include the following:

• 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.

• 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, he or 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.

Coping and support

Hearing that your child has KHE may be scary. We hope that this site reassures you with reliable information and begins to prepare you for your next steps. There are also a number of other resources to help you and your family through this difficult time.

Patient education: From the first office visit, our nurses will be on hand to walk you through your child’s treatment and help answer any questions you may have — What kinds of treatment are available? What symptoms might my child have? They will also reach out to you by phone, continuing the care and support you received while at Children’s.

Parent to parent: Want to talk with someone whose child has been treated for KHE? We may be able to put you in touch with other families who can share their experience.

Faith-based support: If you are in need of spiritual support, we will help connect you with the Children’s chaplaincy. Our program includes nearly a dozen clergy representing Episcopal, Jewish, Lutheran, Muslim, Roman Catholic, Unitarian and United Church of Christ traditions who will listen to you, pray with you and help you observe your own faith practices during your hospital experience.

Social work and mental health professionals: Our social workers and mental health clinicians have helped many other families in your situation. We can offer counseling and assistance with issues such as coping with your child’s diagnosis, stresses relating to coping with illness and dealing with financial difficulties.

On our For Patients and Families site, you can read all you need to know about:

• getting to Children’s
• accommodations
• navigating the hospital experience
• resources that are available for your family

Our Vascular Anomalies Center offers links to organizations that offer support and education for parents who have a child with a vascular anomaly.