Key Points for This Section

There are different types of treatment for patients with childhood rhabdomyosarcoma. Children with rhabdomyosarcoma should have their treatment planned by a team of doctors with expertise in treating cancer in children. Some cancer treatments cause side effects months or years after treatment has ended. Three types of standard treatment are used: Surgery Radiation therapy Chemotherapy New types of treatment are being tested in clinical trials. High-dose chemotherapy with stem cell transplant Patients may want to think about taking part in a clinical trial. Patients can enter clinical trials before, during, or after starting their cancer treatment. Follow-up tests may be needed.

There are different types of treatment for patients with childhood rhabdomyosarcoma.

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.

Children with rhabdomyosarcoma should have their treatment planned by a team of doctors with expertise in treating cancer in children.

Because rhabdomyosarcoma can form in many different parts of the body, many different kinds of treatments are used. Your child's care will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other health care professionals who are experts in treating children. 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.
• Psychologist.

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

Some cancer treatments cause side effects that continue or appear months or years after cancer treatment has ended. These 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 possible late effects caused by some treatments. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer ¹ for more information.)

Three types of standard treatment are used:

Surgery

Surgery (removing the cancer in an operation) is used to treat childhood rhabdomyosarcoma. Surgery is usually wide local excision, which is removal of the tumor and some of the normal tissue around it, including 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 child's appearance.
• 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.

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 or radiation therapy may be given before surgery to shrink large tumors.

For most children with rhabdomyosarcoma, complete removal of the tumor by surgery is not possible. 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 increase the chances of a cure, 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. 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.

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 spinal column, 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.

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 ².

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.

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 clinical trials database.

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.

Glossary Terms

The area of the body that contains the pancreas, stomach, intestines, liver, gallbladder, and other organs.

Treatment given after the primary treatment to increase the chances of a cure. Adjuvant therapy may include chemotherapy, radiation therapy, hormone therapy, or biological therapy.

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.

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.

The soft, sponge-like tissue in the center of most bones. It produces white blood cells, red blood cells, and platelets.

A term for diseases in which abnormal cells divide without control. Cancer cells can invade nearby tissues and can spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is 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.

A flexible tube used to deliver fluids into or withdraw fluids from the body.

A hollow area or hole. It may describe a body cavity (such as the space within the abdomen) or a hole in a tooth caused by decay.

The individual unit that makes up the tissues of the body. All living things are made up of one or more cells.

Treatment with drugs that kill cancer cells.

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 a clinical study.

Treatment using more than one anticancer drug.

To heal or restore health; a treatment to restore health.

The process of identifying a disease by the signs and symptoms.

The amount of medicine taken, or radiation given, at one time.

Any substance, other than food, that is used to prevent, diagnose, treat or relieve symptoms of a disease or abnormal condition. Also refers to a substance that alters mood or body function, or that can be habit-forming or addictive, especially a narcotic.

A type of radiation therapy that uses a machine to aim high-energy rays at the cancer from outside of the body. Also called external-beam radiation therapy.

Monitoring a person's health over time after treatment. This includes keeping track of the health of people who participate in a clinical study or clinical trial for a period of time, both during the study and after the study ends.

A communication process between a specially trained health professional and a person concerned about the genetic risk of disease. The person's family and personal medical history may be discussed, and counseling may lead to genetic testing.

Refers to the genitalia (external and internal sex organs and glands).

An intensive drug treatment to kill cancer cells, but that also destroys the bone marrow and can cause other severe side effects. High-dose chemotherapy is usually followed by bone marrow or stem cell transplantation to rebuild the bone marrow.

A method of putting fluids, including drugs, into the bloodstream. Also called intravenous infusion.

Use of a syringe and needle to push fluids or drugs into the body; often called a "shot."

A type of radiation therapy in which radioactive material sealed in needles, seeds, wires, or catheters is placed directly into or near a tumor. Also called brachytherapy, radiation brachytherapy, and implant radiation therapy.

Side effects of cancer treatment that appear months or years after treatment has ended. Late effects include physical and mental problems and second cancers.

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.

NCI, 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. It 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 NCI Web site at http://www.cancer.gov. Also called National Cancer Institute.

A doctor who specializes in treating cancer. Some oncologists specialize in a particular type of cancer treatment. For example, a radiation oncologist specializes in treating cancer with radiation.

A part of the body that performs a specific function. For example, the heart is an organ.

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.

Having to do with children.

A doctor who specializes in treating blood disorders in children.

A registered nurse with an advanced degree in nursing who specializes in the care of children.

A surgeon who specializes in the treatment of children. A surgeon removes or repairs a part of the body by operating on the patient.

A specialist who can talk with patients and their families about emotional and personal matters, and can help them make decisions.

Energy released in the form of particles or electromagnetic waves. Common sources of radiation include radon gas, cosmic rays from outer space, and medical x-rays.

A doctor who specializes in using radiation to treat cancer.

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 radiotherapy and irradiation.

Giving off radiation.

A small, radioactive pellet that is placed in or near a tumor. Cancer cells are killed by the energy given off as the radioactive material breaks down and becomes more stable.

To come back or to return.

Treatment with anticancer drugs directed to a specific area of the body.

A healthcare professional who helps people recover from an illness or injury and return to daily life. Examples of rehabilitation specialists are physical therapists and occupational therapists.

Cancer that forms in the soft tissues in a type of muscle called striated muscle. Rhabdomyosarcoma can occur anywhere in the body.

Refers to a new primary cancer in a person with a history of cancer.

A problem that occurs when treatment affects healthy tissues or organs. Some common side effects of cancer treatment are fatigue, pain, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.

A professional trained to talk with people and their families about emotional or physical needs, and to find them support services.

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 spine, backbone, and vertebral column.

The extent of a cancer in the body. Staging is usually based on the size of the tumor, whether lymph nodes contain cancer, and whether the cancer has spread from the original site to other parts of the body.

In medicine, treatment that experts agree is appropriate, accepted, and widely used. Health care providers are obligated to provide patients with the standard of care. Also called standard therapy or best practice.

A cell from which other types of cells develop. For example, blood cells develop from blood-forming stem cells.

A method of replacing immature blood-forming cells that were destroyed by cancer treatment. The stem cells are given to the person after treatment to help the bone marrow recover and continue producing healthy blood cells.

A procedure to remove or repair a part of the body or to find out whether disease is present. An operation.

Treatment with anticancer drugs that travel through the blood to cells all over the body.

A group or layer of cells that work together to perform a specific function.

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 cancerous), or malignant (cancerous). Also called neoplasm.

A blood vessel that carries blood to the heart from tissues and organs in the body.

Surgery to cut out the cancer and some healthy tissue around it.

A type of high-energy radiation. 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.