In general, cancer in children and teenagers is uncommon, so it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That’s why more than 60% of children with cancer are treated as part of a clinical trial. Clinical trials are research studies that compare standard treatments (the best proven treatments available) with newer treatments that may be more effective. Investigating new treatments involves careful monitoring using scientific methods, and all participants are followed closely to track their health and progress.

To take advantage of these newer treatments, all children and adolescents with EFT should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating this age group and have access to the latest research. A doctor who specializes in treating children with cancer is called a pediatric oncologist. In many cases, a team of doctors works with a child and the family to provide care; this is called a multidisciplinary team. Pediatric cancer centers often have extra support services for children/teens and their families, such as child life specialists, nutritionists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available.

Children and teenagers with EFT should be treated in clinical trials specifically designed for their disease. A typical treatment plan for EFT consists of systemic therapy (therapy that treats the entire body, such as chemotherapy or stem cell transplantation) combined with localized therapy. Localized therapy is therapy to treat the tumor itself, such as surgery and radiation therapy. When more than one treatment is used, it is called combination therapy. Doctors make treatment decisions based on the stage of the disease and the age of the child, while trying to avoid or reduce long-term side effects of treatment.

The treatment of EFT depends on the size and location of the tumor, whether the tumor has spread, and the child’s overall health.

Descriptions of the most common treatment options for EFT are listed below. Treatment options and recommendations depend on several factors, including the type and stage of cancer, possible side effects, and the patient’s preferences and overall health. 

Chemotherapy

Chemotherapy is the use of drugs to kill cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. Chemotherapy is given by a pediatric or medical oncologist, a doctor who specializes in treating cancer with medication. Some people may receive chemotherapy in their doctor’s office; others may go to the hospital or outpatient clinic. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs at the same time.

When possible, chemotherapy is given before surgery. Sometimes, the doctor may use surgery or radiation therapy (see below) first and then give chemotherapy to eliminate any remaining EFT cells. After chemotherapy, the doctor may use localized surgery or radiation therapy followed by additional chemotherapy to eliminate any remaining EFT cells.

Chemotherapy for EFT is usually injected into a vein or muscle; it is rarely given by mouth. Patients with EFT may receive vincristine (Oncovin, Vincasar PFS), cyclophosphamide (Cytoxan, Neosar), doxorubicin (Adriamycin), ifosfamide (Ifex), and etoposide (Toposar, VePesid). For children with EFT that has not spread to other parts of the body, the standard schedule is to receive chemotherapy every two weeks. Patients with metastatic EFT may also be treated with the above medications and dactinomycin (Cosmegen).

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished.

 The medications used to treat EFT are continually being evaluated. Talking with your child's doctor is often the best way to learn about the medications prescribed for your child, their purpose, and their potential side effects or interactions with other medications. 

Treating neutropenia

People receiving chemotherapy for EFT may be at risk for neutropenia, an abnormally low level of a type of white blood cell called neutrophils. All white blood cells help the body fight infection. Your child’s doctor may give your child medications to increase white blood cell levels. These medications are called white blood cell growth factors, also known as colony-stimulating factors (CSFs). Treating neutropenia is an important part of the treatment plan. 

Surgery

Surgery is the removal of the tumor and surrounding tissue during an operation. When it is possible, surgical removal of the tumor should be performed after chemotherapy (called neoadjuvant chemotherapy). An orthopedic oncologist is a doctor who specializes in treating bone cancer with surgery. Surgery may also be needed to remove any remaining cancer cells after chemotherapy or radiation therapy. 

Often a tumor can be removed without causing disability. However, if the tumor occurs in the arm or leg, surgery to remove much of the bone may affect the limb's ability to function. Bone grafts from other parts of the body may help to reconstruct the limb. A prosthesis (artificial limb) made of metal or plastic bones or joints can replace lost tissue. Physical therapy after surgery can help the child learn to use the limb again. Support services are available to help the child cope with the loss of a limb. 

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other high-energy particles to kill tumor cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. For EFT, radiation therapy is used when surgery is not possible or did not remove all tumor cells, as well as when chemotherapy was not effective.

The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. When radiation therapy is given using implants, it is called internal radiation therapy or brachytherapy. A radiation therapy regimen (schedule) usually consists of a specific number of treatments given over a set period of time. Intraoperative radiation therapy (radiation given inside the body during surgery) is being studied in clinical trials.

Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. In the long term, radiation therapy can also interfere with normal bone growth and increase the risk of developing a secondary cancer. 

Stem cell transplantation/bone marrow transplantation

For EFT, stem cell transplantation is an experimental approach that is still under evaluation to determine its effectiveness in the treatment of this disease. It should only be done as part of a clinical trial.

A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the blood stem cells that are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient and family about the risks of this treatment and consider several other factors, such as the type of tumor, results of any previous treatment, and child’s age and general health.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cell: allogeneic (ALLO) and autologous (AUTO). AUTO transplantations are used to treat EFT.

In an AUTO transplant, the patient’s own stem cells are used. The stem cells are obtained from the patient when he or she is in remission from previous treatment. The stem cells are frozen until they are needed, usually after the high-dose treatment (explained below) is completed.

The goal of transplantation is to destroy tumor cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many tumor cells as possible. This also destroys the patient’s bone marrow tissue and suppresses the patient’s immune system. After the high-dose treatment is given, blood stem cells are infused into the patient’s vein to replace the bone marrow and restore normal blood counts from donor cells.

In stem cell transplantation, replacement cells engraft (begin to make new blood cells) and turn into healthy, blood-producing tissue in 10 days to three weeks. Destroying the patient’s own marrow reduces the body’s natural defenses, temporarily leaving the patient at an increased risk of infection. Until the immune system is back to normal, the patient may need antibiotics and blood transfusions.

In an AUTO transplant, there is little risk of tissue rejection because the replacement stem cells are the patient’s own cells. However, there is a risk in an autologous transplant that some of the cells that are put back into the patient could still be cancerous. 

Palliative/supportive care

Cancer and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the cancer, an important part of cancer care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care can help a person at any stage of illness. People often receive treatment for the cancer and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Before treatment begins, talk with your child’s health care team about the possible side effects of the recommended treatment plan and supportive care options. And during and after treatment, be sure to tell your child’s doctor or another health care team member if your child experiences a problem, so it is addressed as quickly as possible. 

Recurrent EFT

A remission is when cancer cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED.

A remission can be temporary or permanent. This uncertainty leads to many children and their parents feeling worried or anxious that the cancer will come back. While many remissions are permanent, it is important to talk with your child’s doctor about the possibility of the cancer returning. Understanding the risk of recurrence and the treatment options may help everyone feel more prepared if the cancer does return. 

If the cancer does return after the original treatment, it is called recurrent cancer. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place (distant recurrence). Recurrence is most common within the first two years after treatment has been completed. However, late recurrences occurring up to five years after treatment are more common with EFT than with other childhood solid tumors.

If there is a recurrence, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your child’s doctor will talk about the treatment options. The next round of treatment depends on where and when the tumor recurred and how it was first treated. The doctor may use chemotherapy, including cyclophosphamide and topotecan (Hycamtin), irinotecan (Camptosar) and temozolomide (Temodar), radiation therapy, and/or may surgically remove new tumors. Stem cell transplantation may also be recommended. Your doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent cancer.

Children with recurrent cancer and their parents often experience emotions such as disbelief or fear. Patients and families are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. 

If treatment fails

Although treatment is successful for the majority of children with cancer, sometimes it is not. If a child’s cancer cannot be cured or controlled, this is called advanced or terminal cancer. This diagnosis is stressful, and it may be difficult to discuss. However, it is important to have open and honest conversations with your child’s doctor and health care team to express your family’s feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families.

Parents or guardians are encouraged to think about where the child would be most comfortable: at home, in a home-like setting elsewhere, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families. Some children may be happier if they can arrange to attend school part-time or keep up other activities and social connections. The child’s health care team can help parents or guardians decide on an appropriate level of activity. Making sure a child is physically comfortable and free from pain is extremely important as part of end-of-life care. 

The death of a child is an enormous tragedy, and families may need support to help them cope with the loss. Pediatric cancer centers often have professional staff and support groups to help with the process of grieving.

Side Effects

Fear of treatment side effects is common after a diagnosis of cancer, but it may help to know that preventing and controlling side effects is a major focus of your child’s health care team. This is called palliative or supportive care, and it is an important part of the overall treatment plan, regardless of the stage of disease.

Common side effects from each treatment option for EFT are described in detail within the Treatment section. Side effects depend on a variety of factors, including the cancer’s stage, the length and dosage of treatment(s), and your child’s overall health.

Before treatment begins, talk with your child’s doctor about possible side effects of each type of treatment your child will be receiving. Ask which side effects are most likely to happen, when they are likely to occur, and what can be done to prevent or relieve them. And, ask about the level of caregiving your child may need during treatment and recovery, as family members and friends often play an important role in the care of a person with EFT. 

In addition to physical side effects, there may be psychosocial (emotional and social) effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team who can help with coping strategies. These strategies can be different based on the age of your child. 

During and after treatment, be sure to tell the health care team about the side effects you experience/your child experiences, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care.

After Treatment

After treatment for EFT ends, talk with your child’s doctor about developing a follow-up care plan. This plan may include regular physical examinations and/or medical tests to monitor your child’s recovery for the coming months and years. All children and teens treated for cancer, including EFT, should have life-long follow-up care by a doctor or health care team familiar with the late effects of chemotherapy and radiation therapy.

Follow-up care includes bone scans, CT scans, MRI scans, and x-rays.

Children treated with chemotherapy will need to be monitored for potential problems with their sexual development and fertility (ability to have children). Your child may need to be referred to an endocrinologist (a doctor who specializes in problems with glands and the endocrine system) for evaluation in these areas. Other late effects may include delayed or stunted growth and delayed or missed developmental milestones. 

Based on the type of treatment the child received, the doctor will determine what examinations and tests are needed to check for long-term side effects and the possibility of secondary cancers. Your child’s doctor can recommend the necessary screening tests. Follow-up care should also address the child’s quality of life, including any developmental or emotional concerns. 

The child’s family is encouraged to organize and keep a record of the child’s medical information. That way, as the child enters adulthood, he or she has a clear, written history of the diagnosis, the treatment given, and the doctor’s recommendations about the schedule for follow-up care. The doctor’s office can help you create this, and it should include recommendations from the doctor about the schedule for follow-up care. This information will be valuable to doctors who care for your child during his or her lifetime. 

Children and teens who have had cancer can also enhance the quality of their future by following established guidelines for good health into and through adulthood, including not smoking, maintaining a healthy weight, eating a balanced diet, and participating in regular physical activity. Talk with the doctor about developing a plan that is best for your child’s needs.

Latest Research

Doctors are working to learn more about EFT, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the diagnostic and treatment options best for your child.

New chemotherapy. Researchers are evaluating the use of vincristine (Vincasar PFS), cyclophosphamide (Cytoxan, Neosar), and topotecan (Hycamtin) in addition to standard chemotherapy for patients with newly diagnosed, localized EFT.

Allogeneic stem cell transplantation. In an allogeneic (ALLO) stem cell transplantation , the person is treated with high doses of chemotherapy and/or radiation therapy to kill as many tumor cells as possible and to prevent the person's immune system from rejecting the donated stem cells. After the high-dose therapy is given, stem cells obtained from a healthy donor (usually a sibling) are infused into the patient's bloodstream. Allogeneic stem cell transplants combined with sirolimus (Rapamune) and other drugs have been shown to inhibit growth of Ewing cells in the laboratory.

Bilateral lung radiation. The effectiveness of bilateral lung radiation is being examined in patients with metastatic Ewing sarcoma. In this treatment, radiation therapy is given to both lungs after completion of chemotherapy, and, for some patients, autologous (AUTO) stem cell transplant is recommended.

Insulin-like growth factor receptor-1 (IGF-1R) antibodies. The IGFR is an important growth protein for sarcomas. Inhibiting its activity may be an important new way to improve sarcoma treatment. This new class of treatment is being studied in people with recurrent EFT alone or in combination with other drugs, such as temsirolimus (Torisel).

Supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current EFT treatments, in order to improve patients’ comfort and quality of life.