Rhabdomyosarcoma - Childhood: Treatment Options

Approved by the Cancer.Net Editorial Board, 04/2016

ON THIS PAGE: You will learn about the different ways doctors use to treat children with this type of cancer. To see other pages, use the menu.

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’s why more than 60% of children with cancer are treated as part of a clinical trial. Clinical trials are research studies that compare the standard of care with newer treatments that may be more effective. The “standard of care” is the best treatment known. 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 devoted exclusively to 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.  

Treatment overview

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.

Descriptions of the most common treatment options for rhabdomyosarcoma are listed below. 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. Also, talk about the goals of each treatment with the doctor and what your child can expect while receiving the treatment. Learn more about making treatment decisions.


Surgery is the removal of the tumor and some surrounding healthy 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 specializes in treating cancer using surgery.

Even children who have a rhabdomyosarcoma tumor that can be completely removed by surgery still require chemotherapy (see below). If the tumor cannot be completely removed or is unable to be removed by surgery, it is called inoperable or unresectable. If a tumor is inoperable, the cancer is treated with chemotherapy and radiation therapy to kill the cancer cells. If a tumor is unresectable, a biopsy is needed to determine the type of tumor (see Diagnosis).

Learn more about the basics of cancer surgery.

Chemotherapy and Risk Groups

Chemotherapy is the use of drugs to destroy cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Chemotherapy is given by a pediatric or medical oncologist, a doctor who specializes in treating cancer with medication.

Systemic chemotherapy gets into the bloodstream to reach cancer cells throughout the body. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle or a pill or capsule that is swallowed (orally).

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 drug is often called VAC.

Different studies are underway to determine 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).

  • low risk

  • intermediate risk

  • and high risk.

The idea is that the intensity of the therapy can be made less if the chance of the cancer coming back is lower. Nearly all children with rhabdomyosarcoma are treated on clinical trials. In this way, a very good standard of care can be delivered and compared to new combinations of treatment already showing promise, in order to find out what the next best standard treatment should become. In the United States, most research on childhood rhabdomyosarcoma takes place through the COG. Other individual hospitals may also offer studies that are independent of the COG. Learn more about current COG studies for rhabdomyosarcoma.

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea. The side effects most common for the specific drugs used to treat rhabdomyosarcoma are listed below.

  • VAC therapy: Nausea, vomiting, and bone marrow suppression, meaning the 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, 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 and preparing for treatment. The medications used to treat cancer are constantly being evaluated. Talking with your 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. Learn more about your child’s prescriptions by using searchable drug databases.

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 (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.

Getting care for symptoms and side effects

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 is any treatment that focuses on reducing symptoms, improving quality of life, and supporting patients and their families. Any person, regardless of age or type and stage of cancer, may receive palliative care. It works best when palliative care is started as early as needed in the cancer treatment process. 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.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. Your child may also receive palliative treatments similar to those meant to eliminate the cancer, such as chemotherapy, surgery, or radiation therapy. Talk with the doctor about the goals of each treatment in your treatment plan.

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

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’s important to talk with your child’s doctor about the possibility of the cancer returning. Understanding your 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 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).

When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After 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 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 often experience emotions such as disbelief or fear. Patients and their families are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

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. 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 in children with higher risk disease 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 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. Hospice care is a type of palliative care for people who are expected to live less than 6 months. It is designed to provide the best possible quality of life for people who are near the end of life. 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. 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.

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. Or, use the menu to choose another section to continue reading this guide.