Rhabdomyosarcoma

Each year, more than 400 children, adolescents, and young adults up to age 40 are diagnosed with rhabdomyosarcoma. Our team of pediatric sarcoma experts treats more than 25 newly diagnosed patients annually.

Rhabdomyosarcoma is a tumor that arises from muscle. Any muscle in the body can be involved. Pain and swelling are the most common symptoms related to this disease.

There are two main types of rhabdomyosarcoma that affect children and young adults: the more common embryonal form and the rarer alveolar form. The type of therapy recommended for a young patient depends on the form of rhabdomyosarcoma.

Diagnosis

When rhabdomyosarcoma is suspected, a biopsy of the affected tissue is required to confirm the diagnosis. A biopsy typically involves removing a small portion of the tumor under anesthesia to make a diagnosis. In rare situations, this procedure can result in the removal of the entire tumor.

Our pathologists perform tests on the biopsied tissue to look for specific abnormalities associated with this disease. One particular test can identify a genetic abnormality only found in alveolar rhabdomyosarcoma.

Because rhabdomyosarcoma can spread widely throughout the body, our experts may also perform bone marrow tests and imaging studies such as MRI, CT scans, PET scans, and bone scans to determine how far the tumor has spread.

The results of these tests allow Memorial Sloan Kettering doctors to categorize children and young adults with rhabdomyosarcoma into different risk groups – low, intermediate, or high – based on how much of the body is involved and the patient’s expected treatment response.

Treatment

Initial treatment for rhabdomyosarcoma typically lasts between five and 12 months. We tailor our treatments to each individual patient’s risk group and type of disease. In general, patients in the low-risk group will require less therapy than those in the high-risk group, and alveolar rhabdomyosarcoma may require more-intensive therapy than embryonal rhabdomyosarcoma.

The typical treatment includes chemotherapy to shrink the tumor and prevent new tumors from forming. In only a small number of patients, surgery is performed after chemotherapy to remove the entire tumor. Typically, though, radiation therapy begins several months after chemotherapy starts, as a highly effective additional way to shrink the tumor.

For some patients with certain types of rhabdomyosarcoma, we use an advanced, more targeted form of radiation therapy called intensity-modulated radiotherapy (IMRT), which can be quite effective in controlling the tumor.(1) Varying the dose of IMRT in different areas of the tumor has been shown to decrease side effects in some younger patients as well.(2)

Often a PET scan is performed after the initial chemotherapy and radiation therapy. Our investigators led a study that showed that the results of the scan can be used to predict the chance that the rhabdomyosarcoma will be permanently controlled by the combination of chemotherapy and radiation therapy.(3)

We also conducted a clinical trial that demonstrated the effectiveness of the chemotherapy combination of irinotecan and carboplatin in newly diagnosed patients with intermediate- and high-risk rhabdomyosarcoma, both alone and in combination with radiation therapy.(4)

Most children with rhabdomyosarcoma are cured. In situations in which a child’s tumor recurs or spreads to other areas of the body, Memorial Sloan Kettering offers several innovative treatment strategies, including novel combinations of chemotherapy agents, biologically targeted agents, radiation therapy, and surgery. Your child may also be eligible to participate in a clinical trial.