Childhood Rhabdomyosarcoma Treatment (PDQ®)
General Information About Childhood Rhabdomyosarcoma
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:
- Embryonal: This type occurs most often in the head and neck area or in the genital or urinary organs. It is the most common type.
- Alveolar: This type occurs most often in the arms or legs, chest, abdomen, genital organs, or anal area. It usually occurs during the teen years.
- Anaplastic: This type rarely occurs in children.
See the following PDQ treatment summaries for more information about soft tissue sarcomas:
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:
- Li-Fraumeni syndrome.
- Pleuropulmonary blastoma.
- Neurofibromatosis type 1 (NF1).
- Beckwith-Wiedemann syndrome.
- Costello syndrome.
- Noonan syndrome.
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:
- A lump or swelling that keeps getting bigger or does not go away. It may be painful.
- Bulging of the eye.
- Trouble urinating or having bowel movements.
- Blood in the urine.
- Bleeding in the nose, throat, vagina, or rectum.
Diagnostic tests and a biopsy are used to detect (find) and diagnose childhood rhabdomyosarcoma.
The following tests and procedures may be used:
- Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient's health habits and past illnesses and treatments will also be taken.
- X-ray: An x-ray of the organs and bones inside the body, such as the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.
- CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, such as the abdomen or pelvis, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.
- MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. This procedure is also called nuclear magnetic resonance imaging (NMRI).
- Bone scan: A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones and is detected by a scanner.
- Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone. Samples are removed from both hipbones. A pathologist views the bone marrow, blood, and bone under a microscope to look for signs of cancer.
- Lumbar puncture: A procedure used to collect cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the spinal column. A sample of CSF is removed and checked under a microscope for signs of cancer cells. This procedure is also called an LP or spinal tap.
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:
- Fine-needle aspiration (FNA) biopsy: The removal of tissue or fluid using a thin needle.
- Core needle biopsy: The removal of tissue using a wide needle. This procedure may be guided using ultrasound, CT scan, or MRI.
- Open biopsy: The removal of tissue through an incision (cut) made in the skin.
The following tests may be done on the sample of tissue that is removed:
- Light microscopy: A laboratory test in which cells in a sample of tissue are viewed under regular and high-powered microscopes to look for certain changes in the cells.
- Immunohistochemistry: A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer.
- Immunocytochemistry: A test that uses antibodies to check for certain antigens in a sample of cells. The antibody is usually linked to a radioactive substance or a dye that causes the cells to light up under a microscope. This type of test may be used to tell the difference between different types of soft tissue sarcoma.
- Reverse transcription-polymerase chain reaction (RT-PCR) test: A laboratory test in which cells in a sample of tissue are studied using chemicals to look for certain changes in the structure or function of genes.
- Cytogenetic analysis: A laboratory test in which cells in a sample of tissue are viewed under a microscope to look for certain changes in the chromosomes.
Certain factors affect prognosis (chance of recovery) and treatment options.
- Where in the body the tumor started.
- The width of the tumor when the cancer was diagnosed.
- Whether the tumor has spread to nearby lymph nodes or distant parts of the body.
- Whether there are certain changes in the genes.
- The type of rhabdomyosarcoma.
- Whether the tumor has been completely removed by surgery.
- Whether the tumor responds to chemotherapy and/or radiation therapy.
- The patient's age and general health.
- Whether the tumor has just been diagnosed or has recurred (come back).
For patients with recurrent cancer, prognosis and treatment depend on the following:
- Where in the body the tumor recurred (came back).
- Whether the tumor was treated with radiation therapy.
- The size of the tumor when the cancer was diagnosed.
- How much time passed between the end of cancer treatment and when the cancer recurred.
Stages of Childhood Rhabdomyosarcoma
After childhood rhabdomyosarcoma has been diagnosed, treatment is based on the stage of the cancer and 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 on the stage and the amount of cancer that remains after surgery to remove the tumor. The pathologist will use a microscope to check the tissues, including lymph nodes, removed during surgery, and the edges of the areas where the cancer was removed. 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.
- Tissue. The cancer spreads from where it began by growing into nearby areas.
- Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body.
- Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body.
Cancer may spread from where it began to other parts of the body.
- Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body.
- Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body.
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:
- Eye or area around the eye.
- Head and neck (but not in the tissue next to the brain and spinal cord).
- Gallbladder and bile ducts.
- In the testes, vagina, or uterus.
- The tumor is no larger than 5 centimeters and cancer has spread to nearby lymph nodes.
- The tumor is larger than 5 centimeters and cancer may have spread to nearby lymph nodes.
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.
- IIA: Cancer was removed by surgery but cancer cells were seen when the tissue, taken from the edges of where the tumor was removed, was viewed under a microscope by a pathologist.
- IIB: Cancer had spread to nearby lymph nodes and the cancer and lymph nodes were removed by surgery.
- IIC: Cancer had spread to nearby lymph nodes and the cancer and lymph nodes were 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 cancer cells were seen.
- Cancer cells are found by an imaging test; and
- there are cancer cells in the fluid around the brain, spinal cord, or lungs, or in fluid in the abdomen; or tumors are found in those areas.
Recurrent Childhood Rhabdomyosarcoma
Treatment Option Overview
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:
- Pediatric surgeon.
- Radiation oncologist.
- Pediatric hematologist.
- Pediatric nurse specialist.
- Geneticist or cancer genetics risk counselor.
- Social worker.
- Rehabilitation specialist.
Some cancer treatments cause side effects months or years after treatment has ended.
- Physical problems.
- Changes in mood, feelings, thinking, learning, or memory.
- Second cancers (new types of cancer).
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:
- Where in the body the tumor started.
- The effect the surgery will have on the way the child will look.
- The effect the surgery will have on the child's important body functions.
- How the tumor responded to chemotherapy or radiation therapy that may have been given first.
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.
Even if 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.
External radiation therapy uses a machine outside the body to send radiation toward the cancer. Certain ways of giving radiation therapy can help keep radiation from damaging healthy tissue. These types of external radiation therapy include the following:
- Conformal radiation uses a computer to create a 3-dimensional (3-D) picture of the tumor. The radiation beams are shaped to fit the tumor.
- Intensity-modulated radiation therapy (IMRT) uses images created by a computer that show the size and shape of the tumor. Thin beams of radiation of different strengths are aimed at the tumor from many angles.
- Fractionated stereotactic radiation therapy uses a rigid head frame attached to the skull to aim radiation directly to a tumor. This causes less damage to nearby healthy tissue. The total dose of radiation is divided into several small doses given over several days. This type of radiation therapy may be used for rhabdomyosarcoma of the head and neck. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy.
- Proton-beam therapy is a type of high-energy, external radiation therapy that uses streams of protons (small, positively-charged particles of matter) to kill tumor cells.
Internal radiation therapy (brachytherapy) uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. Internal radiation therapy is used to treat cancer in areas such as the vagina, vulva, bladder, prostate, head, or neck.
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.
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). Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated.
Every child treated for rhabdomyosarcoma should receive 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.
High-dose chemotherapy with stem cell transplant
High-dose chemotherapy with stem cell transplant is a way of giving high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.
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.
Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. An mTOR inhibitor is a type of targeted therapy that blocks a protein involved in cell division called mTOR. Blocking the action of this protein may keep cancer cells from dividing and prevent new blood vessels from forming in a tumor.
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. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI's listing of clinical trials.
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.
Treatment Options for Childhood Rhabdomyosarcoma
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.
- For tumors of the brain: Treatment may include surgery to remove the tumor, radiation therapy, and chemotherapy.
- For tumors of the head and neck that are in or near the eye: Treatment may include chemotherapy and radiation therapy. If the tumor remains or comes back after treatment with chemotherapy and radiation therapy, surgery to remove the eye and some tissues around the eye may be needed.
- For tumors of the head and neck that are near the brain and spinal cord but not in or near the eye: Treatment may include radiation therapy and chemotherapy.
- For tumors of the head and neck that cannot be removed by surgery: Treatment may include chemotherapy and radiation therapy.
- For tumors of the larynx (voice box): Treatment may include chemotherapy and radiation therapy. Surgery to remove the larynx is usually not done, so that the voice is not harmed.
- Surgery to remove the tumor. If the tumor was not completely removed, a second surgery to remove the tumor may be done.
- For tumors of the hand or foot, radiation therapy and chemotherapy may be given. The tumor may not be removed because the function of the hand or foot would be lessened.
Lymph node dissection (one or more lymph nodes are removed and a sample of tissue is checked under a microscope for signs of cancer).
- For tumors in the arms, lymph nodes near the tumor and in the armpit area are removed.
- For tumors in the legs, lymph nodes near the tumor and in the groin area are removed.
- Radiation therapy.
- For tumors in the chest or abdomen (including the chest wall or abdominal wall): Surgery (wide local excision) may be done. If the tumor is large, chemotherapy, and sometimes radiation therapy, is given to shrink the tumor before surgery.
- For tumors of the pelvis: Surgery (wide local excision) may be done. If the tumor is large, chemotherapy, and sometimes radiation therapy, is given to shrink the tumor before surgery. Some pelvic tumors may be treated with biopsy, rather than wide local excision, followed by radiation therapy.
- For tumors of the diaphragm: A biopsy of the tumor is followed by chemotherapy and radiation therapy to shrink the tumor. Surgery may be done later to remove any remaining cancer cells.
- For tumors of the gallbladder or bile ducts: Surgery is done to remove as much of the tumor as possible, followed by chemotherapy and radiation therapy.
- For tumors of the muscles or tissues around the anus or between the vulva and the anus or the scrotum and the anus: Surgery is done to remove as much of the tumor as possible and some nearby lymph nodes, followed by chemotherapy and radiation therapy.
- For tumors that are only at the top of the bladder: Surgery (wide local excision) is done.
- For tumors of the prostate or bladder (other than the top of the bladder):
- Chemotherapy and radiation therapy are given first to shrink the tumor. If cancer cells remain after chemotherapy and radiation therapy, the tumor is removed by surgery. Surgery may include removal of the prostate, part of the bladder, or pelvic exenteration without removal of the rectum. (This may include removal of the lower colon and bladder. In girls, the cervix, vagina, ovaries, and nearby lymph nodes may be removed).
- Chemotherapy is given first to shrink the tumor. Surgery to remove the tumor, but not the bladder or prostate, is done. Internal radiation therapy may be given after surgery.
- Rhabdomyosarcoma of the testicular area is usually treated with surgery to remove the testicle and spermatic cord.
- The lymph nodes in the back of the abdomen may be checked for cancer, especially if the lymph nodes are enlarged or the child is older than 9 years. Radiation therapy may be given if the tumor cannot be completely removed by surgery. CT scans may be done every 3 months after surgery to see if the cancer is growing in nearby lymph nodes.
- For tumors of the vulva and vagina: Treatment may include chemotherapy followed by surgery to remove the tumor. Internal or external radiation therapy may be given after surgery.
- For tumors of the uterus: Treatment may include chemotherapy with or without radiation therapy. Sometimes surgery may be needed to remove any remaining cancer cells.
- For tumors of the cervix: Treatment may include chemotherapy followed by surgery to remove any remaining tumor.
- For tumors of the ovary: Treatment may include combination chemotherapy followed by surgery to remove any remaining tumor.
Radiation therapy may be given for tumors that have spread to the brain, spinal cord, or lungs.
Treatment is also given to the site where the tumor first formed.
The following treatment is being studied for rhabdomyosarcoma:
- A clinical trial of immunotherapy.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with previously untreated childhood rhabdomyosarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child's doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI Web site.
Recurrent Childhood Rhabdomyosarcoma
Treatment options for recurrent childhood rhabdomyosarcoma are based on many factors, including where in the body the cancer has come back, what type of treatment the patient had before, and the needs of the individual child. Treatment may include one or more of the following:
- Chemotherapy with one or more anticancer drugs.
- Radiation therapy.
- A clinical trial of new anticancer drugs.
- A clinical trial of high-dose chemotherapy followed by stem cell transplant using the patient's own stem cells.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with recurrent childhood rhabdomyosarcoma. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child's doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI Web site.
To Learn More About Childhood Rhabdomyosarcoma
For more information from the National Cancer Institute about childhood rhabdomyosarcoma, see the following:
- Soft Tissue Sarcoma Home Page
- Computed Tomography (CT) Scans and Cancer
- Drugs Approved for Rhabdomyosarcoma
- Targeted Cancer Therapies
For more childhood cancer information and other general cancer resources, see the following:
- Childhood Cancers
- CureSearch for Children's Cancer
- Late Effects of Treatment for Childhood Cancer
- Adolescents and Young Adults with Cancer
- Young People with Cancer: A Handbook for Parents
- Cancer in Children and Adolescents
- Cancer Staging
- Coping with Cancer: Supportive and Palliative Care
- Questions to Ask Your Doctor About Cancer
- Cancer Library
- For Survivors and Caregivers