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300 Longwood Avenue
Boston, MA 02115
(617) 355-6000
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My Child Has:
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Rhabdomyosarcoma
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Rhabdomyosarcoma is a cancerous tumor that originates in the soft tissues of the body, particularly in the skeletal muscles that attach to bone and help the body to move. The most common sites for this tumor to be found include the head, neck, bladder, vagina, arms, legs, and trunk.
Rhabdomyosarcoma can also be found in places where skeletal muscles are absent or very small, such as in the prostate, middle ear, and bile duct system. The cancer cells associated with this disease can spread (metastasize) to other areas of the body.
As you read further below, you will find general information about rhabdomyosarcoma. If you would like to view summary information about cancer first, see the cancer overview.
Embryonal rhabdomyosarcoma, the most common type of rhabdomyosarcoma, usually occurs in children under 6 years of age. Alveolar rhabdomyosarcoma occurs in older children and accounts for about 20 percent of all cases.
Rhabdomyosarcoma is the most common soft tissue sarcoma in childhood. In the United States, about 250 children are diagnosed with rhabdomyosarcoma each year. This disease affects children between the ages of 2 to 20 years of age, but can occur at any age. For unknown reasons, males are affected slightly more often than females.
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It is believed that some rhabdomyosarcoma tumors begin developing in the fetus. Rhabdomyoblasts are the cells at the initial stages of development of an unborn baby. These cells will mature and develop into muscles. There has been much research into the gene structure of these rhabdomyoblasts and possible detection of a gene error that can produce the disease later in development.
Rhabdomyosarcomas usually have some type of chromosome abnormality in the cells of the tumor, which are responsible for the tumor formation. In children with an embryonal rhabdomyosarcoma, there is usually an abnormality of chromosome #11. In alveolar rhabdomyosarcoma, a rearrangement in the chromosome material between chromosomes #2 and #13 is usually present. This rearrangement changes the position and function of genes, causing a fusion of genes referred to as a "fusion transcript." Patients have an abnormal fusion transcript involving two genes known as PAX3 and FKHR. This important discovery has led to improvements in diagnosing rhabdomyosarcoma. Rhabdomyosarcomas are also more common in children with neurofibromatosis or Li-Frameni syndrome, which are genetic disorders. Li-Frameni syndrome is a clustering of soft tissue cancers in a family, caused by mutations in a tumor suppressor gene called p53, which results in uncontrolled cell growth. There has been no association between rhabdomyosarcoma and environmental exposures.
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The following are the most common symptoms of rhabdomyosarcoma. However, each child may experience symptoms differently. Many symptoms depend on the size and the location of the tumor. Symptoms may not be present until the tumor is very large, especially if it is located deep in the muscle or in the stomach. Symptoms may include:
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a tumor or mass that can be seen or felt (may or may not be painful)
- bleeding from the nose, vagina, rectum, or throat (may occur if the location of the tumor is in these areas)
- tingling, numbness, pain, and movement (may be affected if the tumor compresses nerves in the area)
- protrusion of the eye or a drooping eyelid (may indicate a tumor behind the area)
The symptoms of rhabdomyosarcoma may resemble other conditions or medical problems. Always consult your child's physician for a diagnosis.
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In addition to a complete medical history and physical examination, diagnostic procedures for rhabdomyosarcoma may include:
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- biopsy of the tumor - a sample of tissue removed from the tumor and examined under a microscope
- blood and urine tests
- multiple imaging studies, including:
- computerized tomography scan (also called a CT or CAT scan) - a diagnostic imaging procedure that uses a combination of x-rays and computer technology to produce cross-sectional images (often called slices), both horizontally and vertically, of the body. A CT scan shows detailed images of any part of the body, including the bones, muscles, fat, and organs. CT scans are more detailed than general x-rays.
- magnetic resonance imaging (MRI) - a diagnostic procedure that uses a combination of large magnets, radiofrequencies, and a computer to produce detailed images of organs and structures within the body.
- x-ray - a diagnostic test which uses invisible electromagnetic energy beams to produce images of internal tissues, bones, and organs onto film
- ultrasound (also called sonography) - a diagnostic imaging technique which uses high-frequency sound waves and a computer to create images of blood vessels, tissues, and organs. Ultrasounds are used to view internal organs as they function, and to assess blood flow through various vessels.
- bone scans - pictures or x-rays taken of the bone after a dye has been injected that is absorbed by bone tissue. These are used to detect tumors and bone abnormalities.
- bone marrow aspiration/biopsy - a procedure that involves a small amount of bone marrow fluid and tissue to be taken, usually from part of the hip bones, to further examine the number, size, and maturity of blood cells and/or abnormal cells.
- lumbar puncture/spinal tap - a special needle is placed into the lower back, into the spinal canal. This is the area around the spinal cord. The pressure in the spinal canal and brain can then be measured. A small amount of cerebral spinal fluid (CSF) can be removed and sent for testing to determine if there is an infection or other problems. CSF is the fluid that bathes your child's brain and spinal cord.
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Diagnosis involves staging and classifying the tumor, which is important in determining treatment options. Staging is the process of finding out whether cancer has spread, and, if so, how far. There are various staging systems that are used for rhabdomyosarcoma. Always consult your child's physician for more information on staging. One method of staging is the TNM staging system. This system uses tumor (T), nodes (N), and metastases (M) to differentiate the disease by stages. This system evaluates the disease before surgery and classifies it by one of the four following stages:
- stage I - tumors involving the area near the eye, the head, neck, and genitourinary tract (except the prostate and bladder). The tumor is localized, meaning the tumor has not spread to other areas of the body.
- stage II - small, localized tumors less than 5 cm in any site not in stage I. There are no tumor cells in the surrounding lymph nodes.
- stage III - localized tumor at any site not included in stage I that is larger than 5 cm and/or has spread to surrounding lymph nodes.
- stage IV - disease that has spread to other areas of the body at the time of diagnosis.
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Specific treatment for rhabdomyosarcoma will be determined by your child's physician based on:
- your child's age, overall health, and medical history
- extent of the disease
- your child's tolerance for specific medications, procedures, or therapies
- how your child's physician expects the disease may progress
- your opinion or preference
Treatment for rhabdomyosarcoma requires close coordination between surgeons, pediatric oncologists, and radiotherapists. The response of tumors is very much dependent on their site of origin. A series of studies have been performed by the Intergroup Rhabdomyosarcoma Study (IRS) which has defined the basis for treatment of rhabdomyosarcoma. Children's Hospital participates in the treatment protocols of this organization.
Surgery is generally required to both provide a diagnosis of the tumor type as well as provide information regarding the stage (extent of spread) of the tumor. Treatment with chemotherapy, with or without radiation therapy, is then determined by the extent of removal of the tumor as well as by its stage and location.
Tumors arising particularly in the bladder and prostate receive initial chemotherapy prior to attempts at surgical removal or treatment with radiation. Tumors arising in the muscles of the extremity are often treated initially with surgical removal, and the completeness of removal determines the need for additional chemotherapy, with or without radiotherapy. Tumors around the eye are very responsive to chemotherapy and radiation, so they rarely require surgical removal.
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A summary of these treatments follows:
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- surgery - biopsy and surgical removal of tumors, performed by surgeons. Tumors in the head and neck area are often treated with surgical removal, if total removal is possible. Depending on the size and location of the tumor, it is sometimes necessary to remove all or part of a limb. In most cases, however, limb-sparing surgery is used to avoid amputation. Through limb-sparing surgery, as much of the tumor as possible is removed. Radiation therapy and/or chemotherapy are given either before surgery to shrink the tumor, or after surgery to kill remaining cancer cells.
- chemotherapy - a drug treatment that works by interfering with the cancer cell's ability to grow or reproduce. Different groups of drugs work in different ways to fight cancer cells and shrink tumors. Chemotherapy may be used alone for some types of cancer or in conjunction with other therapy such as radiation or surgery. Often, a combination of chemotherapy drugs is used to fight a specific cancer. Certain chemotherapy drugs may be given in a specific order depending on the type of cancer it is being used to treat. While chemotherapy can be quite effective in treating certain cancers, the agents do not differentiate normal healthy cells from cancer cells. Because of this, there can be many adverse side effects during treatment. Being able to anticipate these side effects can help the care team, parents, and child prepare, and, in some cases, prevent these symptoms from occurring, if possible.
Chemotherapy is systemic treatment, meaning it is introduced to the bloodstream and travels throughout the body to kill cancer cells. Chemotherapy can be given:
- as an injection into the muscle or fat tissue
- intravenously (directly to the bloodstream; also called IV)
- intrathecally - chemotherapy given directly into the spinal column with a needle
- radiation therapy - using high-energy rays (radiation) from a specialized machine to damage or kill cancer cells and shrink tumors
- lumbar puncture - to administer medication and treat cancer cells if found in this site
- stem cell transplant - a treatment involving stem cells, a specific type of cell from which all blood cells develop. Stem cells develop into red blood cells to carry oxygen, white blood cells to fight disease and infection, and platelets to aid in blood clotting. Transplantation of normal stem cells from another person is used to help restore normal blood production in patients whose own ability to make any or all of these blood cells has been compromised by cancer, intensive cancer treatment, or other types of damage or abnormality. The use of cells from another individual is called allogeneic transplantation. Stem cells collected from patients themselves prior to intensive treatment can also be used to supplement the recovery of the patient's own cells after particularly aggressive course of chemotherapy or radiation therapy. The use of a patient's own cells is referred to as autologous transplantation.
Stem cell transplantation and the treatment needed to manage its effects are complex. Your physician will give you more detailed information on what to expect.
- supportive care - any type of treatment to prevent and treat infections, side effects of treatments, and complications, and to keep your child comfortable during treatment
- continuous follow-up care - a schedule of follow-up care determined by your child's physician and other members of your care team to monitor ongoing response to treatment and possible late effects of treatment
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Prognosis greatly depends on:
- the extent of the disease.
- the size and location of the tumor.
- presence or absence of metastasis.
- the tumor's response to therapy.
- the age and overall health of your child.
- your child's tolerance of specific medications, procedures, or therapies.
- new developments in treatment.
As with any cancer, prognosis and long-term survival can vary greatly from child to child. Prompt medical attention and aggressive therapy are important for the best prognosis. Continuous follow-up care is essential if your child is diagnosed with rhabdomyosarcoma. Side effects of radiation and chemotherapy, as well as second malignancies, can occur in survivors of rhabdomyosarcoma. New methods are continually being discovered to improve treatment and to decrease side effects.
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Children's Hospital and Dana-Farber Cancer Institute are conducting numerous research studies that will help clinicians better understand and treat rhabdomyosarcoma.
In the area of stem cell transplantation, the Dana-Farber/Children's Transplant Program is one of only eight institutions around the country that are investigating the use of umbilical cord transplantation. Also under study is a novel method for preventing graft versus host disease, a serious complication that occurs when transplanted cells do not recognize the tissues and organs of the recipient's body and react against the recipient's tissue. The result of this treatment approach, if it continues to be as successful, will be that the degree of match between donor and the recipient will not need to be particularly close, greatly increasing the pool of potential donors for each patient. This could also eliminate the need for long-term drug therapy traditionally needed to treat graft versus host disease.
Other types of treatment currently being studied include:
- angiogenesis inhibitors - substances that may be able to prevent the growth of tumors by blocking the formation of new blood vessels that feed the tumors
- biological therapies - a wide range of substances that may be able to involve the body's own immune system to fight cancer or lessen harmful side effects of some treatments
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The information on this website should not be taken as medical advice,
which can only be given to you by your personal health care professional. |
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