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Childhood Rhabdomyosarcoma Treatment (PDQ®)

Patient Version
Last Modified: 05/25/2012

General Information About Childhood Rhabdomyosarcoma

Key Points for This Section


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. 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:

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 possible sign of childhood rhabdomyosarcoma is a lump or swelling that keeps getting bigger.

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. Check with your child's doctor 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.
  • Headache.
  • Trouble urinating or having bowel movements.
  • Blood in the urine.
  • Bleeding in the nose, throat, vagina, or rectum.

Tests that examine the area where symptoms have occurred 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. 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.
  • 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.
  • 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 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.
  • 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 find out what type it is. Because treatment depends on the type of rhabdomyosarcoma, biopsy samples should be checked by a pathologist who has experience in diagnosing rhabdomyosarcoma. The following tests may be done on the sample of tissue that is removed:
    • 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.
    • 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.

The prognosis (chance of recovery) and treatment options depend on the following:

  • Where in the body the tumor started.
  • The width of the tumor when the cancer was diagnosed.
  • Whether the tumor has been completely removed by surgery.
  • 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.
  • 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

Key Points for This Section


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 3 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.

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.

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:

Stage 1

In stage 1, the tumor is any size, has not 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.

Enlarge 4
Tumor size compared to everyday objects; shows various measurements of a tumor compared to a pea, peanut, walnut, and lime 4
Pea, peanut, walnut, and lime show tumor sizes.

Stage 2

In stage 2, cancer is found in an "unfavorable" site (any one area not included in stage 1). The tumor is no larger than 5 centimeters and has not spread to lymph nodes.

Stage 3

In stage 3, cancer is found in an "unfavorable" site (any one area not included in stage 1) and one of the following is true:

  • 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.

Stage 4

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:

Group I

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

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.

Group III

Cancer was partly removed by surgery but there are still some cancer cells (a lump or mass) left that can be seen by x-ray or other imaging test. Cancer has not spread to distant parts of the body.

Group IV

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 chemotherapy agent, 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:

  • An embryonal tumor of any size that is found in a "favorable" site. There may be tumor remaining after surgery that can be seen without a microscope. The cancer may have spread to nearby lymph nodes. The following areas are "favorable" sites:
  • An embryonal tumor of any size that is not found in one of the "favorable" sites listed above. There may be tumor remaining after surgery that can be seen only with a microscope. The cancer may have spread to nearby lymph nodes.

Intermediate-risk childhood rhabdomyosarcoma

Intermediate-risk childhood rhabdomyosarcoma is one of the following:

  • An embryonal tumor of any size that is not found in one of the "favorable" sites listed above. There is tumor remaining after surgery, that can be seen with or without a microscope. The cancer may have spread to nearby lymph nodes.
  • An alveolar tumor of any size in a "favorable" or "unfavorable" site. There may be tumor remaining after surgery that can be seen with or without a microscope. The cancer may have spread to nearby lymph nodes.

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 distant parts of the body.

Recurrent Childhood Rhabdomyosarcoma

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.

Treatment Option Overview

Key Points for This Section


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:

Some cancer treatments cause side effects months or years after treatment has ended.

Side effects from cancer treatment that begin during or after treatment and continue for months or years are called late effects. Late effects of cancer treatment for rhabdomyosarcoma may include:

  • 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 5 for more information.)

Three types of standard treatment are used:

Surgery

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.

Radiation 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. 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, where in the body the tumor started, how much tumor remained after surgery, and whether there is tumor in the nearby lymph nodes.

Chemotherapy

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 chemotherapy agent, 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 6 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 Web site 7.

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

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

Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Targeted therapy with angiogenesis inhibitors is used to treat childhood rhabdomyosarcoma. Angiogenesis inhibitors keep blood vessels from forming in a tumor. This causes the tumor to starve and stop growing or to shrink. Monoclonal antibodies and kinase inhibitors are two types of antiangiogenic agents used to treat childhood rhabdomyosarcoma.

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 used to treat childhood rhabdomyosarcoma attach to and block substances that cause new blood vessels to form in tumors. Monoclonal antibodies may be used with chemotherapy as adjuvant therapy.

Kinase inhibitors stop cells from dividing and may prevent the growth of new blood vessels that tumors need to grow.

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. 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

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 8 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

  • 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.

Rhabdomyosarcoma of the arms or legs

Rhabdomyosarcoma of the chest or abdomen

  • 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 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.

Rhabdomyosarcoma of the kidney

Rhabdomyosarcoma of the bladder and prostate

Rhabdomyosarcoma of the area near the testicles

Rhabdomyosarcoma of the vulva, vagina, uterus or ovary

Metastatic rhabdomyosarcoma

Some of the treatments being studied for rhabdomyosarcoma include the following:

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with previously untreated childhood rhabdomyosarcoma 9. 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 10.

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:

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with recurrent childhood rhabdomyosarcoma 11. 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 10.

Changes to This Summary (05/25/2012)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

Changes were made to this summary to match those made to the health professional version.

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PDQ is a comprehensive cancer database available on NCI's Web site.

PDQ is the National Cancer Institute's (NCI's) comprehensive cancer information database. Most of the information contained in PDQ is available online at NCI's Web site 29. PDQ is provided as a service of the NCI. The NCI is part of the National Institutes of Health, the federal government's focal point for biomedical research.

PDQ contains cancer information summaries.

The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries are available in two versions. The health professional versions provide detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions provide current and accurate cancer information.

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Editorial Boards made up of experts in oncology and related specialties are responsible for writing and maintaining the cancer information summaries. The summaries are reviewed regularly and changes are made as new information becomes available. The date on each summary ("Date Last Modified") indicates the time of the most recent change.

PDQ also contains information on clinical trials.

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." In the United States, about two-thirds of children with cancer are treated in a clinical trial at some point in their illness.

Listings of clinical trials are included in PDQ and are available online at NCI's Web site 10. Descriptions of the trials are available in health professional and patient versions. For additional help in locating a childhood cancer clinical trial, call the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237).

The PDQ database contains listings of groups specializing in clinical trials.

The Children's Oncology Group (COG) is the major group that organizes clinical trials for childhood cancers in the United States. Information about contacting COG is available on the NCI Web site 29 or from the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237).



Glossary Terms

abdomen (AB-doh-men)
The area of the body that contains the pancreas, stomach, intestines, liver, gallbladder, and other organs.
alveolar rhabdomyosarcoma (al-VEE-oh-ler RAB-doh-MY-oh-sar-KOH-muh)
A soft tissue tumor that is most common in older children and teenagers. It begins in embryonic muscle cells (cells that develop into muscles in the body). It can occur at many places in the body, but usually occurs in the trunk, arms, or legs. Also called ARMS.
anal (AY-nul)
Having to do with the anus. The anus is the opening of the rectum (last part of the large intestine) to the outside of the body.
analysis (uh-NA-lih-sis)
A process in which anything complex is separated into simple or less complex parts.
anaplastic (A-nuh-PLAS-tik)
A term used to describe cancer cells that divide rapidly and have little or no resemblance to normal cells.
antibody (AN-tee-BAH-dee)
A protein made by plasma cells (a type of white blood cell) in response to an antigen (a substance that causes the body to make a specific immune response). Each antibody can bind to only one specific antigen. The purpose of this binding is to help destroy the antigen. Some antibodies destroy antigens directly. Others make it easier for white blood cells to destroy the antigen.
antigen (AN-tih-jen)
Any substance that causes the body to make a specific immune response.
Beckwith-Wiedemann syndrome (BEK-with-VEE-deh-mahn SIN-drome)
A rare, overgrowth disorder in which babies are large at birth and may develop low blood sugar. Other common symptoms are a large tongue, large internal organs, and defects of the abdominal wall near the navel. Beckwith-Wiedemann syndrome increases the risk of developing certain cancers, especially Wilms tumor.
biopsy (BY-op-see)
The removal of cells or tissues for examination by a pathologist. The pathologist may study the tissue under a microscope or perform other tests on the cells or tissue. There are many different types of biopsy procedures. The most common types include: (1) incisional biopsy, in which only a sample of tissue is removed; (2) excisional biopsy, in which an entire lump or suspicious area is removed; and (3) needle biopsy, in which a sample of tissue or fluid is removed with a needle. When a wide needle is used, the procedure is called a core biopsy. When a thin needle is used, the procedure is called a fine-needle aspiration biopsy.
biopsy specimen (BY-op-see SPEH-sih-men)
Tissue removed from the body and examined under a microscope to determine whether disease is present.
blood (blud)
A tissue with red blood cells, white blood cells, platelets, and other substances suspended in fluid called plasma. Blood takes oxygen and nutrients to the tissues, and carries away wastes.
bone marrow (bone MAYR-oh)
The soft, sponge-like tissue in the center of most bones. It produces white blood cells, red blood cells, and platelets.
bone marrow aspiration (bone MAYR-oh AS-pih-RAY-shun)
A procedure in which a small sample of bone marrow is removed, usually from the hip bone, breastbone, or thigh bone. A small area of skin and the surface of the bone underneath are numbed with an anesthetic. Then, a special wide needle is pushed into the bone. A sample of liquid bone marrow is removed with a syringe attached to the needle. The bone marrow is sent to a laboratory to be looked at under a microscope. This procedure may be done at the same time as a bone marrow biopsy.
bone marrow biopsy (bone MAYR-oh BY-op-see)
A procedure in which a small sample of bone with bone marrow inside it is removed, usually from the hip bone. A small area of skin and the surface of the bone underneath are numbed with an anesthetic. Then, a special, wide needle is pushed into the bone and rotated to remove a sample of bone with the bone marrow inside it. The sample is sent to a laboratory to be looked at under a microscope. This procedure may be done at the same time as a bone marrow aspiration.
bone scan (bone skan)
A technique to create images of bones on a computer screen or on film. A small amount of radioactive material is injected into a blood vessel and travels through the bloodstream; it collects in the bones and is detected by a scanner.
bowel (BOW-ul)
The long, tube-shaped organ in the abdomen that completes the process of digestion. The bowel has two parts, the small bowel and the large bowel. Also called intestine.
cancer (KAN-ser)
A term for diseases in which abnormal cells divide without control and can invade nearby tissues. Cancer cells can also spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is a cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is a cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is a cancer that starts in blood-forming tissue such as the bone marrow, and causes large numbers of abnormal blood cells to be produced and enter the blood. Lymphoma and multiple myeloma are cancers that begin in the cells of the immune system. Central nervous system cancers are cancers that begin in the tissues of the brain and spinal cord. Also called malignancy.
cartilage (KAR-tih-lij)
A tough, flexible tissue that lines joints and gives structure to the nose, ears, larynx, and other parts of the body.
cell (sel)
The individual unit that makes up the tissues of the body. All living things are made up of one or more cells.
cerebrospinal fluid (seh-REE-broh-SPY-nul FLOO-id)
The fluid that flows in and around the hollow spaces of the brain and spinal cord, and between two of the meninges (the thin layers of tissue that cover and protect the brain and spinal cord). Cerebrospinal fluid is made by tissue called the choroid plexus in the ventricles (hollow spaces) in the brain. Also called CSF.
chromosome (KROH-muh-some)
Part of a cell that contains genetic information. Except for sperm and eggs, all human cells contain 46 chromosomes.
clinical trial (KLIH-nih-kul TRY-ul)
A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical study.
condition (kun-DIH-shun)
In medicine, a health problem with certain characteristics or symptoms.
connective tissue (kuh-NEK-tiv TIH-shoo)
Supporting tissue that surrounds other tissues and organs. Specialized connective tissue includes bone, cartilage, blood, and fat.
contrast material (KON-trast muh-TEER-ee-ul)
A dye or other substance that helps show abnormal areas inside the body. It is given by injection into a vein, by enema, or by mouth. Contrast material may be used with x-rays, CT scans, MRI, or other imaging tests.
Costello syndrome (KOS-teh-loh SIN-drome)
A rare, genetic disorder marked by developmental problems, being shorter than normal, mental retardation, heart problems, unusual facial features, and extra folds of skin around the neck, hands, and feet. People with Costello syndrome have an increased risk of certain types of cancer, such as rhabdomyosarcoma (a soft tissue tumor) and neuroblastoma (cancer of immature nerve cells).
CT scan (… skan)
A series of detailed pictures of areas inside the body taken from different angles. The pictures are created by a computer linked to an x-ray machine. Also called CAT scan, computed tomography scan, computerized axial tomography scan, and computerized tomography.
cytogenetics (SY-toh-jeh-NEH-tix)
The study of chromosomes and chromosomal abnormalities.
diagnosis (DY-ug-NOH-sis)
The process of identifying a disease, such as cancer, from its signs and symptoms.
electron microscope (ee-LEK-tron MY-kroh-SKOPE)
A microscope (device used to magnify small objects) that uses electrons (instead of light) to produce an enlarged image. An electron microscope shows tiny details better than any other type of microscope.
embryonal rhabdomyosarcoma (em-BRY-uh-nul RAB-doh-MY-oh-sar-KOH-muh)
A soft tissue tumor that is most common in infants and young children. It begins in muscles, usually in the head, neck, or genitourinary tract. Also called ERMS.
gene (jeen)
The functional and physical unit of heredity passed from parent to offspring. Genes are pieces of DNA, and most genes contain the information for making a specific protein.
genital (JEH-nih-tul)
Refers to the genitalia (external and internal sex organs and glands).
imaging test (IH-muh-jing …)
A type of test that makes pictures of areas inside the body. Some examples of imaging tests are CT scans and MRIs. Also called imaging procedure.
immunohistochemistry (IH-myoo-noh-HIS-toh-KEH-mih-stree)
A technique used to identify specific molecules in different kinds of tissue. The tissue is treated with antibodies that bind the specific molecule. These are made visible under a microscope by using a color reaction, a radioisotope, colloidal gold, or a fluorescent dye. Immunohistochemistry is used to help diagnose diseases, such as cancer, and to detect the presence of microorganisms. It is also used in basic research to understand how cells grow and differentiate (become more specialized).
inherited (in-HAYR-ih-ted)
Transmitted through genes that have been passed from parents to their offspring (children).
injection (in-JEK-shun)
Use of a syringe and needle to push fluids or drugs into the body; often called a "shot."
laboratory test (LA-bruh-tor-ee...)
A medical procedure that involves testing a sample of blood, urine, or other substance from the body. Tests can help determine a diagnosis, plan treatment, check to see if treatment is working, or monitor the disease over time.
Li-Fraumeni syndrome (lee-FRAH-meh-nee SIN-drome)
A rare, inherited predisposition to multiple cancers, caused by an alteration in the p53 tumor suppressor gene.
light microscope (lite MY-kroh-SKOPE)
A microscope (device to magnify small objects) in which objects are lit directly by white light.
lumbar puncture (LUM-bar PUNK-cher)
A procedure in which a thin needle called a spinal needle is put into the lower part of the spinal column to collect cerebrospinal fluid or to give drugs. Also called spinal tap.
lymph node (limf node)
A rounded mass of lymphatic tissue that is surrounded by a capsule of connective tissue. Lymph nodes filter lymph (lymphatic fluid), and they store lymphocytes (white blood cells). They are located along lymphatic vessels. Also called lymph gland.
medical history (MEH-dih-kul HIH-stuh-ree)
A record of information about a person’s health. A personal medical history may include information about allergies, illnesses, surgeries, immunizations, and results of physical exams and tests. It may also include information about medicines taken and health habits, such as diet and exercise. A family medical history includes health information about a person’s close family members (parents, grandparents, children, brothers, and sisters). This includes their current and past illnesses. A family medical history may show a pattern of certain diseases in a family.
microscope (MY-kroh-SKOPE)
An instrument that is used to look at cells and other small objects that cannot be seen with the eye alone.
MRI
A procedure in which radio waves and a powerful magnet linked to a computer are used to create detailed pictures of areas inside the body. These pictures can show the difference between normal and diseased tissue. MRI makes better images of organs and soft tissue than other scanning techniques, such as computed tomography (CT) or x-ray. MRI is especially useful for imaging the brain, the spine, the soft tissue of joints, and the inside of bones. Also called magnetic resonance imaging, NMRI, and nuclear magnetic resonance imaging.
National Cancer Institute (NA-shuh-nul KAN-ser IN-stih-TOOT)
The National Cancer Institute, part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government's principal agency for cancer research. The National Cancer Institute conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the National Cancer Institute Web site at http://www.cancer.gov. Also called NCI.
neurofibromatosis type 1 (NOOR-oh-FY-broh-muh-TOH-sis ...)
A rare genetic condition that causes brown spots and tumors on the skin, freckling in skin areas not exposed to the sun, tumors on the nerves, and developmental changes in the nervous system, muscles, bone, and skin. Also called NF1.
Noonan syndrome (NOO-nun SIN-drome)
A genetic disorder marked by unusual facial features, being shorter than normal, learning problems, heart defects, problems with blood clotting, defects in the skeleton, and fertility problems in males.
organ (OR-gun)
A part of the body that performs a specific function. For example, the heart is an organ.
pathologist (puh-THAH-loh-jist)
A doctor who identifies diseases by studying cells and tissues under a microscope.
PDQ
PDQ is an online database developed and maintained by the National Cancer Institute. Designed to make the most current, credible, and accurate cancer information available to health professionals and the public, PDQ contains peer-reviewed summaries on cancer treatment, screening, prevention, genetics, complementary and alternative medicine, and supportive care; a registry of cancer clinical trials from around the world; and directories of physicians, professionals who provide genetics services, and organizations that provide cancer care. Most of this information, and more specific information about PDQ, can be found on the NCI's Web site at http://www.cancer.gov/cancertopics/pdq. Also called Physician Data Query.
pelvis (PEL-vus)
The lower part of the abdomen, located between the hip bones.
physical examination (FIH-zih-kul eg-ZA-mih-NAY-shun)
An exam of the body to check for general signs of disease.
prognosis (prog-NO-sis)
The likely outcome or course of a disease; the chance of recovery or recurrence.
radiation therapy (RAY-dee-AY-shun THAYR-uh-pee)
The use of high-energy radiation from x-rays, gamma rays, neutrons, protons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy), or it may come from radioactive material placed in the body near cancer cells (internal radiation therapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Also called irradiation and radiotherapy.
radioactive (RAY-dee-oh-AK-tiv)
Giving off radiation.
radioisotope (RAY-dee-oh-I-suh-tope)
An unstable form of a chemical element that releases radiation as it breaks down and becomes more stable. Radioisotopes may occur in nature or be made in a laboratory. In medicine, they are used in imaging tests and in treatment. Also called radionuclide.
recover (ree-KUH-ver)
To become well and healthy again.
rectum (REK-tum)
The last several inches of the large intestine closest to the anus.
recur (ree-KER)
To come back or to return.
recurrent cancer (ree-KER-ent KAN-ser)
Cancer that has recurred (come back), usually after a period of time during which the cancer could not be detected. The cancer may come back to the same place as the original (primary) tumor or to another place in the body. Also called recurrence.
research study (reh-SERCH STUH-dee)
A scientific study of nature that sometimes includes processes involved in health and disease. For example, clinical trials are research studies that involve people. These studies may be related to new ways to screen, prevent, diagnose, and treat disease. They may also study certain outcomes and certain groups of people by looking at data collected in the past or future.
rhabdomyosarcoma (RAB-doh-MY-oh-sar-KOH-muh)
Cancer that forms in the soft tissues in a type of muscle called striated muscle. Rhabdomyosarcoma can occur anywhere in the body.
risk factor (... FAK-ter)
Something that increases the chance of developing a disease. Some examples of risk factors for cancer are age, a family history of certain cancers, use of tobacco products, being exposed to radiation or certain chemicals, infection with certain viruses or bacteria, and certain genetic changes.
sarcoma (sar-KOH-muh)
A cancer of the bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue.
scanner (SKA-ner)
In medicine, an instrument that takes pictures of the inside of the body.
soft tissue (... TIH-shoo)
Refers to muscle, fat, fibrous tissue, blood vessels, or other supporting tissue of the body.
sonogram (SAH-noh-gram)
A computer picture of areas inside the body created by bouncing high-energy sound waves (ultrasound) off internal tissues or organs. Also called ultrasonogram.
spinal column (SPY-nul KAH-lum)
The bones, muscles, tendons, and other tissues that reach from the base of the skull to the tailbone. The spinal column encloses the spinal cord and the fluid surrounding the spinal cord. Also called backbone, spine, and vertebral column.
standard therapy (... THAYR-uh-pee)
Treatment that experts agree is appropriate, accepted, and widely used. Also called best practice, standard medical care, and standard of care.
surgery (SER-juh-ree)
A procedure to remove or repair a part of the body or to find out whether disease is present. An operation.
symptom (SIMP-tum)
An indication that a person has a condition or disease. Some examples of symptoms are headache, fever, fatigue, nausea, vomiting, and pain.
tendon (TEN-dun)
Tough, fibrous, cord-like tissue that connects muscle to bone or another structure, such as an eyeball. Tendons help the bone or structure to move.
throat (throte)
The hollow tube inside the neck that starts behind the nose and ends at the top of the trachea (windpipe) and esophagus (the tube that goes to the stomach). The throat is about 5 inches long, depending on body size. Also called pharynx.
tissue (TIH-shoo)
A group or layer of cells that work together to perform a specific function.
tumor (TOO-mer)
An abnormal mass of tissue that results when cells divide more than they should or do not die when they should. Tumors may be benign (not cancer), or malignant (cancer). Also called neoplasm.
ultrasound (UL-truh-SOWND)
A procedure in which high-energy sound waves are bounced off internal tissues or organs and make echoes. The echo patterns are shown on the screen of an ultrasound machine, forming a picture of body tissues called a sonogram. Also called ultrasonography.
urinary (YOOR-ih-NAYR-ee)
Having to do with urine or the organs of the body that produce and get rid of urine.
urine (YOOR-in)
Fluid containing water and waste products. Urine is made by the kidneys, stored in the bladder, and leaves the body through the urethra.
vagina (vuh-JY-nuh)
The muscular canal that goes from the uterus to the outside of the body. During birth, the baby passes through the vagina. Also called birth canal.
vein (vayn)
A blood vessel that carries blood to the heart from tissues and organs in the body.
x-ray (EX-ray)
A type of radiation used in the diagnosis and treatment of cancer and other diseases. In low doses, x-rays are used to diagnose diseases by making pictures of the inside of the body. In high doses, x-rays are used to treat cancer.

Table of Links

1http://www.cancer.gov/cancertopics/pdq/treatment/child-soft-tissue-sarcoma/Pati
ent
2http://www.cancer.gov/cancertopics/pdq/treatment/adult-soft-tissue-sarcoma/Pati
ent
3http://www.cancer.gov/cancertopics/pdq/treatment/childrhabdomyosarcoma/Patient/
#Section_19
4http://www.cancer.gov/PublishedContent/MediaLinks/309851.html
5http://www.cancer.gov/cancertopics/pdq/treatment/lateeffects/Patient
6http://www.cancer.gov/cancertopics/druginfo/soft-tissue-sarcoma#dal2
7http://cancer.gov/clinicaltrials
8http://www.cancer.gov/cancertopics/pdq/treatment/childrhabdomyosarcoma/Patient/
Page4#Section_50
9http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?Diagnosis=41431&tt=1&a
mp;format=1&cn=1
10http://www.cancer.gov/clinicaltrials
11http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?Diagnosis=43196&tt=1&a
mp;format=1&cn=1
12http://www.cancer.gov/cancertopics/types/childrhabdomyosarcoma
13http://www.cancer.gov/cancertopics/factsheet/sites-types/soft-tissue-sarcoma
14http://www.cancer.gov/cancertopics/understandingcancer/targetedtherapies
15http://www.cancer.gov/cancertopics/factsheet/Therapy/targeted
16http://www.cancer.gov/cancertopics/wyntk/overview
17http://www.cancer.gov/cancertopics/types/childhoodcancers
18http://www.curesearch.org
19http://www.cancer.gov/cancertopics/aya
20http://www.cancer.gov/cancertopics/youngpeople
21http://www.cancer.gov/cancertopics/factsheet/NCI/children-adolescents
22http://www.cancer.gov/cancertopics/understandingcancer/cancer
23http://www.cancer.gov/cancertopics/factsheet/Detection/staging
24http://www.cancer.gov/cancertopics/coping
25http://www.cancer.gov/cancertopics/cancerlibrary/questions
26http://www.cancer.gov/cancertopics/literature
27http://dccps.nci.nih.gov/ocs/resources.html
28https://livehelp.cancer.gov
29http://cancer.gov
30https://pubs.cancer.gov/ncipl