Home > Patients & Visitors > Health Library > Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment - Patient Information [NCI]
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Childhood rhabdomyosarcoma is a disease in which malignant (cancer) cells form in muscle tissue.
Rhabdomyosarcoma is a type of sarcoma. Sarcoma is cancer of soft tissue (such as muscle), connective tissue (such as tendon or cartilage), or bone. Rhabdomyosarcoma usually begins in muscles that are attached to bones and that help the body move. Rhabdomyosarcoma is the most common type of soft tissue sarcoma in children. It can begin in many places in the body.
There are three main types of rhabdomyosarcoma:
See the following PDQ treatment summaries for information about other types of soft tissue sarcoma:
Certain genetic conditions increase the risk of childhood rhabdomyosarcoma.
Anything that increases the risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your child's doctor if you think your child may be at risk.
Risk factors for rhabdomyosarcoma include having the following inherited diseases:
Children who had a high birth weight or were larger than expected at birth may have an increased risk of embryonal rhabdomyosarcoma.
In most cases, the cause of rhabdomyosarcoma is not known.
A sign of childhood rhabdomyosarcoma is a lump or swelling that keeps getting bigger.
Signs and symptoms may be caused by childhood rhabdomyosarcoma or by other conditions. The signs and symptoms that occur depend on where the cancer forms. Check with your child's doctor if your child has any of the following:
Diagnostic tests and a biopsy are used to detect (find) and diagnose childhood rhabdomyosarcoma.
The diagnostic tests that are done depend in part on where the cancer forms. The following tests and procedures may be used:
If these tests show there may be a rhabdomyosarcoma, a biopsy is done. A biopsy is the removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. Because treatment depends on the type of rhabdomyosarcoma, biopsy samples should be checked by a pathologist who has experience in diagnosing rhabdomyosarcoma.
One of the following types of biopsies may be used:
The following tests may be done on the sample of tissue that is removed:
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis (chance of recovery) and treatment options depend on the following:
For patients with recurrent cancer, prognosis and treatment also depend on the following:
After childhood rhabdomyosarcoma has been diagnosed, treatment is based in part on the stage of the cancer and sometimes it is based on whether all the cancer was removed by surgery.
The process used to find out if cancer has spread within the tissue or to other parts of the body is called staging. It is important to know the stage in order to plan treatment. The doctor will use results of the diagnostic tests to help find out the stage of the disease.
Treatment for childhood rhabdomyosarcoma is based in part on the stage and sometimes on the amount of cancer that remains after surgery to remove the tumor. The pathologist will use a microscope to check the tissues removed during surgery, including tissue samples from the edges of the areas where the cancer was removed and the lymph nodes. This is done to see if all the cancer cells were taken out during the surgery.
There are three ways that cancer spreads in the body.
Cancer can spread through tissue, the lymph system, and the blood:
Cancer may spread from where it began to other parts of the body.
When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood.
The metastatic tumor is the same type of cancer as the primary tumor. For example, if rhabdomyosarcoma spreads to the lung, the cancer cells in the lung are actually rhabdomyosarcoma cells. The disease is metastatic rhabdomyosarcoma, not lung cancer.
Staging of childhood rhabdomyosarcoma is done in three parts.
Childhood rhabdomyosarcoma is staged by using three different ways to describe the cancer:
The staging system is based on the size of the tumor, where it is in the body, and whether it has spread to other parts of the body:
In stage 1, the tumor is any size, may have spread to lymph nodes, and is found in only one of the following "favorable" sites:
Rhabdomyosarcoma that forms in a "favorable" site has a better prognosis. If the site where cancer occurs is not one of the favorable sites listed above, it is said to be an "unfavorable" site.
Tumor sizes. The size of a tumor may be compared to the size of a pea (1 cm), peanut (2 cm), grape (3 cm), walnut (4 cm), lime (5 cm), egg (6 cm), peach (7 cm), or grapefruit (10 cm).
In stage 2, cancer is found in an "unfavorable" site (any one area not described as "favorable" in stage 1). The tumor is no larger than 5 centimeters and has not spread to lymph nodes.
In stage 3, cancer is found in an "unfavorable" site (any one area not described as "favorable" in stage 1) and one of the following is true:
In stage 4, the tumor may be any size and cancer may have spread to nearby lymph nodes. Cancer has spread to distant parts of the body, such as the lung, bone marrow, or bone.
The grouping system is based on whether the cancer has spread and whether all the cancer was removed by surgery:
Cancer was found only in the place where it started and it was completely removed by surgery. Tissue was taken from the edges of where the tumor was removed. The tissue was checked under a microscope by a pathologist and no cancer cells were found.
Group II is divided into groups IIA, IIB, and IIC.
Cancer was partly removed by biopsy or surgery but there is tumor remaining that can be seen with the eye.
Cancer had spread to distant parts of the body when the cancer was diagnosed.
The risk group is based on the staging system and the grouping system.
The risk group describes the chance that rhabdomyosarcoma will recur (come back). Every child treated for rhabdomyosarcoma should receive chemotherapy to decrease the chance cancer will recur. The type of anticancer drug, dose, and the number of treatments given depends on whether the child has low-risk, intermediate-risk, or high-risk rhabdomyosarcoma.
The following risk groups are used:
Low-risk childhood rhabdomyosarcoma
Low-risk childhood rhabdomyosarcoma is one of the following:
Intermediate-risk childhood rhabdomyosarcoma
Intermediate-risk childhood rhabdomyosarcoma is one of the following:
High-risk childhood rhabdomyosarcoma
High-risk childhood rhabdomyosarcoma may be the embryonal type or the alveolar type. It may have spread to nearby lymph nodes and has spread to one or more of the following:
Recurrent childhood rhabdomyosarcoma is cancer that has recurred (come back) after it has been treated. The cancer may come back in the same place or in other parts of the body, such as the lung, bone, or bone marrow. Less often, rhabdomyosarcoma may come back in the breast in adolescent females or in the liver.
There are different types of treatment for patients with childhood rhabdomyosarcoma.
Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.
Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.
Children with rhabdomyosarcoma should have their treatment planned by a team of health care providers who are experts in treating cancer in children.
Because rhabdomyosarcoma can form in many different parts of the body, many different kinds of treatments are used. Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care providers who are experts in treating children with rhabdomyosarcoma and who specialize in certain areas of medicine. These may include the following specialists:
Treatment for childhood rhabdomyosarcoma may cause side effects.
For information about side effects that begin during treatment for cancer, see our Side Effects page.
Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment for rhabdomyosarcoma may include:
Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.)
Three types of standard treatment are used:
Surgery (removing the cancer in an operation) is used to treat childhood rhabdomyosarcoma. A type of surgery called wide local excision is often done. A wide local excision is the removal of tumor and some of the tissue around it, including the lymph nodes. A second surgery may be needed to remove all the cancer. Whether surgery is done and the type of surgery done depends on the following:
In most children with rhabdomyosarcoma, it is not possible to remove all of the tumor by surgery.
Rhabdomyosarcoma can form in many different places in the body and the surgery will be different for each site. Surgery to treat rhabdomyosarcoma of the eye or genital areas is usually a biopsy. Chemotherapy, and sometimes radiation therapy, may be given before surgery to shrink large tumors.
After the doctor removes all the cancer that can be seen at the time of the surgery, patients will be given chemotherapy after surgery to kill any cancer cells that are left. Radiation therapy may also be given. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy.
Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or stop them from growing. There are two types of radiation therapy:
The type and amount of radiation therapy and when it is given depends on the age of the child, the type of rhabdomyosarcoma, where in the body the tumor started, how much tumor remained after surgery, and whether there is tumor in the nearby lymph nodes.
External radiation therapy is usually used to treat childhood rhabdomyosarcoma but in certain cases internal radiation therapy is used.
Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy).
Chemotherapy may also be given to shrink the tumor before surgery in order to save as much healthy tissue as possible. This is called neoadjuvant chemotherapy.
Every child treated for rhabdomyosarcoma should receive systemic chemotherapy to decrease the chance the cancer will recur. The type of anticancer drug, dose, and the number of treatments given depends on whether the child has low-risk, intermediate-risk, or high-risk rhabdomyosarcoma.
See Drugs Approved for Rhabdomyosarcoma for more information.
New types of treatment are being tested in clinical trials.
This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.
Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This type of cancer treatment is also called biologic therapy or biotherapy.
There are different types of immunotherapy:
Immune checkpoint inhibitor. Checkpoint proteins, such as PD-L1 on tumor cells and PD-1 on T cells, help keep immune responses in check. The binding of PD-L1 to PD-1 keeps T cells from killing tumor cells in the body (left panel). Blocking the binding of PD-L1 to PD-1 with an immune checkpoint inhibitor (anti-PD-L1 or anti-PD-1) allows the T cells to kill tumor cells (right panel).
Targeted therapy is a type of treatment that uses drugs or other substances to attack cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation do. There are different types of targeted therapy:
Patients may want to think about taking part in a clinical trial.
For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.
Patients can enter clinical trials before, during, or after starting their cancer treatment.
Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.
Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI's clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.
Follow-up tests may be needed.
Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.
For information about the treatments listed below, see the Treatment Option Overview section.
Previously Untreated Childhood Rhabdomyosarcoma
The treatment of childhood rhabdomyosarcoma often includes surgery, radiation therapy, and chemotherapy. The order that these treatments are given depends on where in the body the tumor started, the size of the tumor, the type of tumor, and whether the tumor has spread to lymph nodes or other parts of the body. See the Treatment Option Overview section of this summary for more information about surgery, radiation therapy, and chemotherapy used to treat children with rhabdomyosarcoma.
Rhabdomyosarcoma of the brain and head and neck
Rhabdomyosarcoma of the arms or legs
Rhabdomyosarcoma of the chest, abdomen, or pelvis
Rhabdomyosarcoma of the kidney
Rhabdomyosarcoma of the bladder or prostate
Rhabdomyosarcoma of the area near the testicles
Rhabdomyosarcoma of the vulva, vagina, uterus, cervix, or ovary
Treatment, such as chemotherapy, radiation therapy, or surgery to remove the tumor, is given to the site where the tumor first formed. If the cancer has spread to the brain, spinal cord, or lungs, radiation therapy may also be given to the sites where the cancer has spread.
The following treatment is being studied for metastatic rhabdomyosarcoma:
Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available.
Refractory or Recurrent Childhood Rhabdomyosarcoma
Treatment options for refractory or recurrent childhood rhabdomyosarcoma are based on many factors, including where in the body the cancer has come back, what type of treatment the child had before, and the needs of the child.
Treatment of refractory or recurrent rhabdomyosarcoma may include one or more of the following:
For more information from the National Cancer Institute about childhood rhabdomyosarcoma, see the following:
For more childhood cancer information and other general cancer resources, see the following:
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Purpose of This Summary
This PDQ cancer information summary has current information about the treatment of childhood rhabdomyosarcoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.
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Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change.
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Clinical Trial Information
A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.
Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237).
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PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Rhabdomyosarcoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/rhabdomyosarcoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389279]
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Last Revised: 2019-05-14
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