Rhabdomyosarcoma - Childhood: Types of Treatment

Approved by the Cancer.Net Editorial Board, 07/2022

ON THIS PAGE: You will learn about the different types of treatments doctors use for children with rhabdomyosarcoma. Use the menu to see other pages.

In general, cancer in children is uncommon. This means it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That is why more than 60% of children with cancer are treated as part of a clinical trial. A clinical trial is a research study that tests a new approach to treatment. The “standard of care” is the best treatment known based on previous clinical trials. Clinical trials may test such approaches as a new drug, a new combination of existing treatments, or new doses of current therapies. The health and safety of all children participating in clinical trials are closely monitored.

The Children’s Oncology Group (COG) is a National Cancer Institute-supported clinical trials cooperative group that does childhood and adolescent cancer research. The COG conducts national clinical trials that are available for children with rhabdomyosarcoma.

To take advantage of the latest treatments, children with cancer should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating children with cancer and have access to the latest research. A doctor who specializes in treating children with cancer is called a pediatric oncologist. If a pediatric cancer center is not nearby, general cancer centers sometimes have pediatric specialists who are able to be part of your child’s care.

How rhabdomyosarcoma is treated

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 also often have extra support services for children and their families, such as child life specialists, dietitians, physical and occupational therapists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available.

Children and adolescents with rhabdomyosarcoma require multidisciplinary therapy planning, which means using combinations of chemotherapy, surgery, and/or radiation therapy. Treatment options and recommendations depend on several factors, including the subtype, stage, and group of rhabdomyosarcoma, possible side effects, and the patient’s preferences and overall health.

Take time to learn about your child’s treatment options and be sure to ask questions about things that are unclear. Talk with your child’s doctor about the goals of each treatment and what your child can expect while receiving the treatment. These types of talks are called "shared decision-making." Shared decision-making is when you and your doctors work together to choose treatments that fit the goals of your child's care. Shared decision-making is particularly important for rhabdomyosarcoma because there are different treatment options. Learn more about making treatment decisions.

The common types of treatments used for rhabdomyosarcoma are described below. Your child’s care plan also includes treatment for symptoms and side effects, an important part of cancer care.


Surgery is the removal of the tumor and some surrounding tissue during an operation, leaving a negative margin or no trace of cancer in the healthy tissue. Surgical oncologists and orthopedic oncologists are doctors who specialize in treating this type of cancer using surgery.

Surgery is usually the first treatment for childhood rhabdomyosarcoma, unless there are distant metastases. However, even when the rhabdomyosarcoma tumor can be completely removed by surgery, chemotherapy is still required afterwards (see below).

If the tumor cannot be completely removed or is unable to be removed by surgery, it is called inoperable or unresectable. In these cases, the cancer is treated with chemotherapy and radiation therapy to destroy the cancer cells. If a tumor is unresectable, a biopsy is needed to determine the type of tumor (see Diagnosis).

Before surgery, talk with your child's health care team about the possible side effects from the specific surgery your child will have. Learn more about the basics of cancer surgery.

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Chemotherapy and risk groups

Chemotherapy is the use of drugs to destroy cancer cells, usually by keeping the cancer cells from growing, dividing, and making more cells.

Chemotherapy for rhabdomyosarcoma is given by injection into a vein. The drugs that are used most often in North America for rhabdomyosarcoma are vincristine (Oncovin, Vincasar), dactinomycin (Cosmegen, Lyovac Cosmegen), and cyclophosphamide (Cytoxan, Clafen, Neosar). This combination of drugs is often called VAC.

Different studies are underway to continue determining the best medications and doses, and often these studies are classified based on the likelihood (or the risk) that the disease will recur (come back).

There are 3 main risk groups for rhabdomyosarcoma:

  • Low risk

  • Intermediate risk

  • High risk

The idea is that the intensity of the therapy can be reduced if the chance of the cancer coming back is lower. Nearly all children with rhabdomyosarcoma are treated on clinical trials. In this way, the standard of care can be delivered and compared to new treatment combinations that may show potential promise in order to find out if the standard of care can be improved. In the United States, most research on childhood rhabdomyosarcoma takes place through the Children's Oncology Group (COG). Other individual hospitals may also offer studies that are independent of the COG. Learn more about current COG studies for rhabdomyosarcoma. Please note this link takes you to a separate website.

The side effects of chemotherapy depend on the particular drug and dose used, but they typically include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea. Common side effects from the specific drugs used to treat rhabdomyosarcoma are listed below.

  • VAC therapy: Nausea, vomiting, and bone marrow suppression, which means your child’s immune system functioning may be reduced, raising the risk of infection and other problems. Although very rare, a few patients may have significant liver damage.

  • Doxorubicin: Nausea, vomiting, bone marrow suppression, and sores in the mouth. This drug may have long-term effects on the heart.

  • Irinotecan: Loose bowel movements.

  • Ifosfamide and etoposide: Nausea, vomiting, bone marrow suppression, and decreased kidney function.

  • Temozolomide: Nausea, vomiting, and bone marrow suppression.

Learn more about managing common side effects of cancer treatment. Most side effects usually go away once treatment is finished. However, long term effects are possible, including that intensive therapy used for intermediate- and high-risk rhabdomyosarcoma may cause permanent infertility (inability to have a child). Talk with your doctor about this possibility and whether your family should talk with a fertility specialist before cancer treatment begins.

Learn more about the basics of chemotherapy.

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Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist.

The most common type of radiation treatment is called external-beam radiation therapy, which is radiation therapy given from a machine outside the body. When radiation treatment is given using implants, it is called internal radiation therapy or brachytherapy. A radiation therapy regimen, or schedule, usually consists of a specific number of treatments given over a set period of time.

Side effects from radiation therapy include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. Learn more about the basics of radiation therapy.

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Physical, emotional, and social effects of cancer

Cancer and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative care or supportive care. It is an important part of your child’s care that is included along with treatments intended to slow, stop, or eliminate the cancer.

Palliative care focuses on improving how your child feels during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type and stage of cancer, may receive this type of care. And it often works best when it is started right after a cancer diagnosis. People who receive palliative care along with treatment for the cancer often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. Your child may also receive palliative treatments similar to those meant to get rid of the cancer, such as chemotherapy, surgery, or radiation therapy.

Before treatment begins, talk with your child’s doctor about the goals of each treatment in the recommended treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.

During treatment, your child’s health care team may ask you to answer questions about your child’s symptoms and side effects and to describe each problem. Be sure to tell the health care team if your child is experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.

Learn more about the importance of tracking side effects in another part of this guide. Learn more about palliative care in a separate section of this website.

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Recurrent rhabdomyosarcoma

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

A remission may be temporary or permanent. This uncertainty causes many people to worry 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 your child’s risk of recurrence and the treatment options may help you feel more prepared if the cancer does return. Learn more about coping with the fear of recurrence.

If the cancer returns 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).

If a recurrence happens, a new cycle of testing will begin to learn as much as possible about it. After this testing is done, your child’s doctor will talk with you about treatment options. The treatment plan may involve chemotherapy, but it depends on how much of the tumor can be surgically removed, where the cancer recurred, and the treatment the child received previously. Often, new experimental treatments in clinical trials for recurrent rhabdomyosarcoma are offered at specialized centers. Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.

Children with recurrent cancer sometimes experience emotions such as disbelief or fear. Parents and their families are encouraged to talk with their child's health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

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If treatment does not work

Although treatment is successful for many children with cancer, sometimes it is not. If a child’s cancer cannot be cured or controlled, this is called advanced or terminal cancer. In rare cases, this may occur during the original diagnosis and treatment or after initial therapy has been completed and there is a recurrence. The likelihood of treatment failure tends to be higher when there is higher risk disease (see "Chemotherapy and risk groups," above) at the time of the original diagnosis.

This diagnosis is stressful, and advanced cancer may be difficult to discuss. However, it is important to have open and honest conversations with your child’s health care team to express your family’s feelings, preferences, and concerns. The health care team has special skills, experience, and knowledge to support patients and their families and is there to help.

Hospice care is designed to provide the best possible quality of life for people who are expected to live less than 6 months. Parents and guardians are encouraged to talk with the health care team about hospice options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Some children may be happier and more comfortable if they can 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. Learn more about caring for a terminally ill child and advanced cancer care planning.

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. Learn more on grieving the loss of a child.

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The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for people with cancer. Use the menu to choose a different section to read in this guide.