Childhood Rhabdomyosarcoma Treatment (PDQ®)
General Information About Childhood Rhabdomyosarcoma
Rhabdomyosarcoma is a type of sarcoma. Sarcoma is cancer of soft tissue (such as muscle), connective tissue (such as tendon or cartilage), and bone. Rhabdomyosarcoma usually begins in muscles that are attached to bones and that help the body move. Rhabdomyosarcoma is the most common type of sarcoma found in the soft tissues of children. It can occur 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, or genital or anal areas. It usually occurs during the teen years.
- Anaplastic: This type rarely occurs in children.
See the following PDQ treatment summaries for more information about sarcomas:
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. Parents who think their child may be at risk should discuss this with the child's doctor. Risk factors for rhabdomyosarcoma include having the following inherited diseases:
- Li-Fraumeni syndrome.
- Neurofibromatosis type 1 (NF1).
- Beckwith-Wiedemann syndrome.
- Costello syndrome.
- Noonan syndrome.
- MEN2A syndrome.
High birth weight and larger than expected size at birth are linked with an increased risk of embryonal rhabdomyosarcoma.
In most cases, the cause of rhabdomyosarcoma is not known.
Lumps and other symptoms may be caused by childhood rhabdomyosarcoma. The symptoms that occur depend on where the cancer forms. Other conditions may cause the same symptoms. A doctor should be consulted if any of the following problems occur:
- 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.
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. 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.
- Lumbar puncture: A procedure used to collect cerebrospinal fluid (CSF) from the spinal column to check for cancer cells. This is done by placing a needle between two bones in the spine and into the spinal column to remove a sample of CSF. This procedure is also called an LP or spinal tap.
- Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram.
- Biopsy: The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. The biopsy is done after imaging tests are done. If rhabdomyosarcoma is found, the pathologist will determine the type. Because treatment depends on the type of rhabdomyosarcoma, patients should ask to have biopsy samples checked by a pathologist who has experience in diagnosing rhabdomyosarcoma.
- 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.
- Light and electron 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 study: A laboratory test in which a substance such as an antibody, dye, or radioisotope is added to a sample of cancer tissue to test for certain antigens. This type of study is used to tell the difference between different types of cancer.
The prognosis (chance of recovery) and treatment options depend on the following:
- Where in the body the tumor started.
- The size of the tumor at the time of diagnosis.
- Whether the tumor has been completely removed by surgery.
- Whether the tumor has spread to nearby lymph nodes or distant parts of the body.
- The type of rhabdomyosarcoma.
- 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).
- How much time passed between the end of cancer treatment and when the cancer recurred.
Stages of Childhood Rhabdomyosarcoma
The process used to find out if cancer has spread within the muscle 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 determine 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.
The three ways that cancer spreads in the body are:
- Through tissue. Cancer invades the surrounding normal tissue.
- Through the lymph system. Cancer invades the lymph system and travels through the lymph vessels to other places in the body.
- Through the blood. Cancer invades the veins and capillaries and travels through the blood to other places in the body.
When cancer cells break away from the primary (original) tumor and travel through the lymph or blood to other places in the body, another (secondary) tumor may form. This process is called metastasis. The secondary (metastatic) tumor is the same type of cancer as the primary tumor. For example, if breast cancer spreads to the bones, the cancer cells in the bones are actually breast cancer cells. The disease is metastatic breast cancer, not bone cancer.
Childhood rhabdomyosarcoma is staged by using three different ways to describe the cancer:
- A staging system.
- A grouping system.
- A risk group.
- 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 or vagina (but not in the kidney, bladder, or prostate).
- The tumor is 5 centimeters or smaller and cancer has spread to nearby lymph nodes.
- The tumor is larger than 5 centimeters and cancer may have spread to nearby lymph nodes.
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 seen.
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 no cancer cells were seen.
Recurrent Childhood Rhabdomyosarcoma
Treatment Option Overview
Different types of treatments are available for children with 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.
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.
- 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.)
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 normal tissue around it, including the lymph nodes. When an extra amount of normal tissue is removed from around the tumor, it is called an en bloc removal of a cuff of normal tissue. 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.
For most children with rhabdomyosarcoma, complete removal of the tumor by surgery is not possible.
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, with or without radiation therapy, after surgery to kill any cancer cells that are left. 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. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated. The type and amount of radiation therapy and when it is given depend on where in the body the tumor started, how much tumor remained after surgery, and the age of the child.
Types of external radiation therapy include the following:
- Conformal radiation uses a computer to create a 3-dimensional 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 intensities 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, causing less damage to nearby healthy tissue. The total dose of radiation is divided into several small doses given over several days. This procedure is also called stereotactic external-beam radiation therapy and stereotaxic radiation therapy. It may be used for rhabdomyosarcomas of the head and neck.
- 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.
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.
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 Web site.
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.
Monoclonal antibody therapy is a type of targeted therapy being studied in the treatment of childhood rhabdomyosarcoma. Monoclonal antibody therapy uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Monoclonal antibodies may be used in combination with chemotherapy as adjuvant therapy.
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.
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.
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. This is sometimes called re-staging.
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 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
A link to a list of current clinical trials is included for each treatment section. For some types or stages of cancer, there may not be any trials listed. Check with your doctor for clinical trials that are not listed here but may be right for you.
Previously Untreated Childhood Rhabdomyosarcoma
- For tumors of the head and neck that are not near the brain and spinal cord and not in or near the eye: Treatment may include surgery (wide local excision). Some lymph nodes may be removed from the same side of the neck as the tumor. Surgery will be followed by chemotherapy with or without radiation therapy.
- For tumors of the head and neck that are in or near the eye: A biopsy of the tumor is done, followed by 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 cannot be removed by surgery: Chemotherapy and radiation therapy are given.
- For tumors of the arms or legs: Surgery (wide local excision and en bloc removal of a cuff of normal tissue) may be done. 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. A second surgery may be done to remove any remaining tumor cells.
- For tumors in the chest wall or abdominal wall: Surgery (wide local excision) may be done. A second surgery may be done to remove any remaining tumor cells.
- For tumors of the chest or abdomen, including the retroperitoneum and pelvis: Chemotherapy, and sometimes radiation therapy, is given to shrink the tumor first, followed by surgery (wide local excision). The surgery is done to remove as much of the remaining tumor as is safely possible.
- For tumors of the gallbladder or bile ducts: Surgery is done to remove as much of the tumor as is safely possible, followed by chemotherapy.
- 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 is safely possible and some nearby lymph nodes, followed by chemotherapy.
- Rhabdomyosarcoma of the testicular area is usually treated with surgery to remove the testicle and spermatic cord. Sometimes a biopsy of the lymph nodes in the back of the abdomen is done, especially if the lymph nodes are enlarged or the child is older than 9 years. CT scans may be done every 3 months after surgery to see if the cancer is growing in nearby lymph nodes.
- For tumors that are only at the top of the bladder: Surgery (wide local excision) is done.
- For tumors of the prostate or other parts of the bladder: Chemotherapy and radiation therapy are given first to shrink the tumor. A biopsy is done and if cancer cells remain, surgery to remove the tumor is done. 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).
- Rhabdomyosarcoma of the vagina and vulva is usually treated with chemotherapy and radiation therapy.
- Rhabdomyosarcoma of the uterus is usually treated with chemotherapy and radiation therapy. Sometimes surgery may be needed to remove any remaining cancer cells.
- For tumors of the brain: Surgery to remove the tumor followed by radiation therapy and chemotherapy.
- For tumors of the larynx (voice box): A biopsy of the tumor is followed by chemotherapy and radiation therapy. Surgery is usually not done, so that the voice is not harmed.
- For tumors of the diaphragm: A biopsy of the tumor is followed by chemotherapy to shrink the tumor. Surgery may be done later to remove any remaining cancer cells.
- For tumors of the kidney: Surgery is done to remove as much of the tumor as is safely possible.
- For tumors of the ovary: Combination chemotherapy may be followed by surgery to remove the remaining tumor.
- Metastatic rhabdomyosarcoma is not usually treated with surgery other than a biopsy for diagnosis. However, if the cancer has spread to the lungs, chemotherapy and radiation therapy are given to shrink the tumor, followed by surgery to remove any remaining cancer cells in the lung.
- Combination chemotherapy, with or without radiation therapy.
- A clinical trial of different doses and schedules of combination chemotherapy, with or without radiation therapy.
- Combination chemotherapy.
- A clinical trial of new combinations of anticancer drugs, with and without targeted therapy (monoclonal antibody).
- A clinical trial of immunotherapy.
Radiation therapy options
- Conformal radiation therapy.
- Intensity-modulated radiation therapy.
- Fractionated stereotactic radiation therapy.
- Proton-beam therapy.
- A clinical trial of brachytherapy (internal radiation therapy) for cancer in areas such as the vagina, vulva, bladder, prostate, head, or neck.
- A clinical trial of second-look surgery after chemotherapy is given to decrease the amount of radiation therapy that will be needed.
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. 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 agents.
- Radiation therapy.
- A clinical trial of different schedules and combinations of chemotherapy.
- A clinical trial of new chemotherapy regimens.
- A clinical trial of new anticancer drugs.
- A clinical trial of targeted therapy.
- 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. 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 Sarcomas: Questions and Answers
- Advances in Targeted Therapies
- Targeted Cancer Therapies
For more childhood cancer information and other general cancer resources from the National Cancer Institute, see the following:
- What You Need to Know About™ Cancer
- 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
- Care for Children and Adolescents with Cancer
- Understanding Cancer Series: Cancer
- Cancer Staging
- Coping with Cancer: Supportive and Palliative Care
- Cancer Library
- Information for Survivors/Caregivers/Advocates