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Cancer Medicine :: Rhabdomyosarcoma - Childhood

Rhabdomyosarcoma - Childhood

General approach to treatment

Treating rhabdomyosarcoma (RMS) is complex and requires the expertise of many different doctors, nurses, and other health professionals. Pediatric (or medical) oncologists, surgeons, radiation oncologists, and oncology nurses will get together to plan the most effective treatment.

The treatment and prognosis (outlook) for patients with RMS depend to a large extent on the type of RMS and on how much of it can be removed with surgery. This is why it’s very important for patients to be diagnosed and treated by doctors who have experience with rhabdomyosarcoma. Children with RMS are best treated in a cancer center such as those who are members of the Children's Oncology Group, where there is experience and expertise in treating childhood cancers.

All children and adults with RMS will be treated with surgery to remove the tumor if it is possible to do so without causing major damage or disfigurement. In some cases, chemotherapy and/or radiation therapy may be used first to try to shrink the tumor. If it shrinks enough, surgery may be done at this point. The goal is to completely remove the tumor, but this is often not possible.

Whether the tumor appears to have been completely removed or not, all patients with RMS should get chemotherapy. Without it, it is very likely that the cancer will come back at distant sites in the body because small amounts of cancer are almost always present in other parts of the body when the cancer is first diagnosed.

If cancer is left behind after surgery or if the cancer has some less favorable traits and it hasn't spread to distant sites (as is the case most of the time), radiation therapy will also be given.

All of these treatments may have side effects, but many of them can be made less troublesome. Your medical team will help you take care of the side effects and will work closely with nutritionists, psychologists, and social workers to help you understand and deal with the medical problems, stress, and other issues related to the treatment.

Because many of these things can be more complicated for cancer in children, many people will be involved in your child's overall care. As a parent, taking care of a child with cancer can be a very big job. It is important to remember that you will have a lot of help. It is also important for you to know that the health care professionals who treat children with RMS are using the experience and knowledge gained from more than 30 years of detailed scientific study of treating this disease.

The next few sections describe in more detail the types of treatments used for RMS.

Surgery for rhabdomyosarcoma

Unless the cancer has clearly spread to distant parts of the body, surgery is usually the first step in treating rhabdomyosarcoma (RMS). Complete resection (removal) of the main tumor, along with some surrounding normal tissue, is the goal whenever possible. If there are cancer cells at the edges (margins) of the removed specimen (meaning that some cancer cells may remain in the body at the tumor site), the surgeon may operate again to try to remove the remaining cancer.

In some cases, surgery may be done even if it is clear that all of the cancer can't be removed because it may help other treatments (chemotherapy and radiation) to work better.

During surgery, nearby lymph nodes may be biopsied to determine if the cancer has spread to these areas, especially if the main tumor is near the testicles in older boys or is on an arm or leg.

Completely removing head and neck tumors may require special surgical teams with ENT (ear, nose, and throat) surgeons, plastic surgeons, maxillofacial surgeons, and neurosurgeons. If the tumor is large or is in a spot where removing it completely would severely affect the child's appearance or cause other problems, then surgery may be delayed until after a few courses of chemotherapy and possibly radiation therapy to try to shrink it, or surgery may not be done at all.

What to expect during surgery

The type and extent of surgery can vary a great deal based on the location and size of the tumor.

The biopsy is generally the first surgery done for RMS. How it is done, how long recovery takes, and how it affects later treatment depend on many factors. The type of biopsy used is based on imaging test results, location and size of the tumor, the child's age and health, and the expertise of the doctor. 

The main goal of surgery is to completely remove the cancer in an effort to avoid future surgery. But as mentioned before, this is not always an option.

Before any surgery, several issues must be addressed. Someone from the surgical team will talk with your family and examine your child to make sure he or she is physically ready for surgery. Blood will be drawn for lab tests to make sure the bone marrow and other organs are working well and to ensure matched units of blood will be available in case your child needs a transfusion during surgery. A parent or guardian will need to sign consent forms, giving permission for the surgery, anesthesia, and possible blood transfusions.

You will be given instructions about what your child can eat and do before and after surgery. The medical team will need to know if your child has any allergies, especially to medicines. The team will calculate the dose of anesthesia based on your child's body size.

Your child may not be allowed to eat or drink for several hours before the surgery. This is to avoid potential complications that might result from having food in the stomach while under anesthesia.

An intravenous (IV) access line will be started in a vein (usually in the arm). Your child will be given a hospital gown and will lie down on a special table, which is then taken to the pre-operative holding area. Anesthesia may be started in this area or the operating room.

If the diagnosis of RMS was not confirmed by a biopsy before the main operation, the surgeon may first take only a small sample of the tumor. The sample is given to a pathologist right away to look at and determine if it is cancer or not. If the pathologist can determine that it is cancer while your child is still on the operating table, the surgeon may remove the entire tumor and may also remove some of the nearby lymph nodes to check for spread of the cancer. If the surgeon suspects the disease has spread to another part of the body, a piece of the possible metastatic tumor may be removed as well.

A bone marrow aspiration and biopsy may also be done, and a central venous access line (a thin catheter) may be inserted into one of the large vessels in the chest to help deliver chemotherapy and other medicines later.

Once the procedure is finished, your child will be taken to the recovery area and closely monitored until fully awake. Your child will then be returned to his or her hospital room.

Possible risks and side effects

Possible complications of surgery depend on the location and extent of the operation and the child's health beforehand. Serious complications, although rare, can include problems with anesthesia, excessive bleeding, wound infections, and pneumonia. Most children will have some pain for a while after the operation, although this can usually be helped with medicines if needed.

Some operations may result in few physical changes other than a scar, while more involved operations may lead to changes in appearance or in how some parts of the body function, and may require physical rehabilitation.

Chemotherapy for rhabdomyosarcoma

All children with rhabdomyosarcoma (RMS) will get chemotherapy at some point. Even if it is thought that the cancer was completely removed by surgery, without chemotherapy it is likely to come back.

Chemotherapy (chemo) is the use of drugs to treat cancer. Chemotherapy is systemic therapy, meaning that the drugs enter the bloodstream and reach throughout the body to destroy cancer cells. This makes chemotherapy useful for killing RMS cells that have spread away from the main tumor to other parts of the body, even if they can't be seen.

Following surgery, any tiny deposits of RMS that remain can often be destroyed by chemotherapy. If larger areas of tumor remain after surgery (or if surgery couldn't be done for some reason), chemotherapy (along with radiation) can often shrink these areas. In some cases this may allow further surgery to completely remove the remaining tumor.

Drugs used to treat rhabdomyosarcoma

There are many chemotherapy drugs. Some can be taken by mouth, but most are injected into a vein. The drugs used depend to some extent on which risk group the child is in .

The main drugs used to treat children in the low-risk group are vincristine and dactinomycin (also known as actinomycin-D). This combination is often referred to as VA. In some cases cyclophosphamide may be added as well. This 3-drug combination is referred to as VAC.

The VAC regimen is the most common combination used for the intermediate-risk group. Irinotecan or topotecan may be added as well. Other drugs used to treat RMS include ifosfamide, etoposide, and doxorubicin.

The same drugs are also used for children in the high-risk group (which includes children with metastatic disease), but these drugs have not been shown to be as successful in this group. New drugs and drug combinations are continually being studied by the Soft Tissue Sarcoma Committee of the Children's Oncology Group and other researchers. It is hoped that they will improve the survival rate in the high-risk group.

Doctors give chemotherapy in cycles, which is usually treatment on 1 or 2 days in a row, followed by days off to give the body time to recover. For RMS, chemotherapy is typically given once a week for the first few months, and then less often. The total length of chemotherapy is usually in the range of 6 months to a year.

Possible side effects

Chemo drugs attack cells that are dividing quickly, which is why they often work against cancer cells. But other cells in the body, such as those in the bone marrow, the lining of the mouth and intestines, and the hair follicles, also divide quickly. These cells are also likely to be affected by chemotherapy, which can lead to side effects.

Children seem to have an advantage over adults when it comes to chemotherapy. They tend to have less severe side effects and recover from side effects more quickly. This is why doctors can often give them higher doses of chemotherapy to kill the tumor.

The side effects of chemotherapy depend on the type of drugs, the doses, and how long they are taken. Possible side effects can include:

Hair loss

Mouth sores

Loss of appetite

Nausea and vomiting

Diarrhea

Increased chance of infections (from low white blood cell counts)

Easy bruising or bleeding (from low blood platelet counts)

Fatigue (from low red blood cell counts)

These side effects are usually short-term and go away once treatment is finished. Your child's doctor and treating team will watch closely for any side effects that develop. There are often ways to lessen these side effects. For example, drugs can be given to help prevent or reduce nausea and vomiting. Be sure to ask your doctor or nurse about medicines to help reduce side effects, and report any side effects your child has so they can be managed effectively.

Along with the risks above, some chemo drugs can have specific side effects (although these are relatively uncommon). For example:

Cyclophosphamide and ifosfamide can damage the bladder, which can cause blood in the urine. The chance of this happening can be lowered by giving the drugs with plenty of fluids and with a drug called mesna, which helps protect the bladder. These drugs can also damage the ovaries or testicles, which might affect fertility (the ability to have children).

Vincristine can damage nerves. Some patients may notice tingling and numbness, particularly in the hands and feet.

Recent studies have shown that children under the age of 3 years are more likely to have liver damage from chemotherapy. Doctors now use lower and very specific doses for any child younger than 3 years old.

Some chemo drugs may also increase the risk of developing a second type of cancer, usually a form of leukemia, years after the RMS is cured. But this is rare, and the importance of chemotherapy in treating RMS far outweighs this risk.

Radiation therapy for rhabdomyosarcoma

Radiation therapy (radiotherapy) uses high-energy radiation to kill cancer cells. It is often an effective way to kill cancer cells that cannot be removed during surgery. When radiation therapy is used to help treat rhabdomyosarcoma (RMS), it is typically given along with chemotherapy.

Radiotherapy is most useful if some of the main tumor is still left after surgery (group II or III) or if completely removing the tumor would mean loss of an important organ, like the eye or bladder, or would be disfiguring. It is not usually needed for children with embryonal rhabdomyosarcoma (ERMS) that can be completely removed by surgery (group I).

Usually radiation therapy is given to any area of remaining disease after 6 to 12 weeks of chemotherapy. An exception is when a tumor near the meninges (linings of the brain) has grown into the skull bones, into the brain itself, or into the spinal cord. In these patients radiation therapy is started right away (along with chemotherapy).

Radiotherapy cannot be given to the whole body to treat metastases, but it can be given to certain areas of known disease to reduce any symptoms the cancer may be causing.

Radiation therapy is much like getting an x-ray, although the dose of radiation is much higher. Radiation is usually given daily (5 days a week) over many weeks. Before treatments start, the radiation team takes careful measurements with imaging tests such as MRI scans to determine the correct angles for aiming the radiation beams and the proper dose of radiation.

For each session, your child will lie on a special table while a machine delivers the radiation from a precise angle. The treatment is not painful. Each session lasts about 15 to 30 minutes, with most of the time being spent making sure the radiation is aimed correctly. The actual treatment time each day is much shorter. Some younger children may be given medicine to make them drowsy before each treatment.

Newer radiation techniques

Some newer techniques may help doctors to more accurately aim treatment at the tumor while reducing the radiation exposure to nearby healthy tissues. These techniques may help increase the success rate and reduce side effects. Many doctors now recommend using these approaches when they are available.

Three-dimensional conformal radiation therapy (3D-CRT): 3D-CRT uses special computers to precisely map the location of the tumor. Depending on where the tumor is, your child may be fitted with a plastic mold resembling a body cast to keep him or her in the same position so that the radiation can be aimed more accurately. Radiation beams are then shaped and aimed at the tumor from several directions, which makes the radiation less likely to damage nearby normal tissues.

Intensity modulated radiation therapy (IMRT): IMRT is an advanced form of 3D therapy. In addition to shaping the beams and aiming them at the tumor from several angles, the intensity (strength) of the beams can be adjusted to limit the dose reaching the most sensitive normal tissues. This lets doctors deliver an even higher dose to the cancer areas. Many major hospitals and cancer centers now use IMRT.

Brachytherapy (internal radiation therapy): Another newer approach is to insert a radioactive pellet into or near the tumor. The radiation from the pellet travels only a short distance, so the tumor gets most of the radiation. This approach may be especially useful in treating some bladder, vaginal, and head and neck area tumors. Some early studies suggest that this may be a good way to preserve the function of these organs in many children.

Possible side effects

The side effects of radiation therapy depend on the dose of radiation and where it is aimed.

Short-term side effects can include fatigue and increased numbers of infections. Effects on skin areas that receive radiation can range from hair loss and mild sunburn-like changes to more severe skin reactions. If the abdomen or pelvis gets radiation, nausea, vomiting, and diarrhea are common. In some cases there may be damage to the bladder or bowel. Radiation to the head and neck can cause mouth sores and loss of appetite.

Small children's brains are very sensitive to radiation, so doctors try to avoid using radiation to the head whenever possible. Side effects of brain radiation therapy usually become most serious 1 or 2 years after treatment and may include headaches and problems such as memory loss, personality changes, and trouble learning at school.

Other long-term problems can include scar tissue formation and the slowing of bone growth. Depending on the age of the child and what parts of the body get the radiation, this could result in deformities or a lack of growth to full height. Radiotherapy may also raise the risk of cancer many years later in the areas that got radiation.

To lower the risk of serious long-term effects from radiation, doctors use the lowest dose of radiation therapy that is still effective.

High-dose chemotherapy and stem cell transplants for rhabdomyosarcoma

A stem cell transplant (sometimes referred to as a bone marrow transplant) makes it possible to use much larger doses of chemotherapy than would normally be possible. Chemotherapy drugs kill rapidly dividing normal cells (such as those in the bone marrow, where new blood cells are made) as well as cancer cells. Higher doses of these drugs might be more effective in treating some cancers, but they are not given because the severe damage to the bone marrow would cause life-threatening shortages of blood cells.

A stem cell transplant gets around this problem by taking out and saving some of the patient's own blood-forming stem cells (either from the blood or bone marrow) before high-dose chemotherapy and then putting them back into the blood after chemotherapy is over, where they will travel to the bone marrow. This allows the normal marrow to regrow.

Stem cell transplants are used to treat some aggressive childhood cancers, but so far it is not clear if they can help rhabdomyosarcoma patients. Because of the severe side effects they can cause, most doctors recommend they be used only as part of a clinical trial.

Rhabdomyosarcoma that progresses or recurs after initial treatment

Rhabdomyosarcoma (RMS) that continues to grow during treatment or that comes back once treatment is finished is often hard to treat. The type of treatment will depend on a number of factors, including the site of the recurrence, type of tumor, and previous treatments used.

For tumors that recur in the same spot as the original tumor, surgery may be used if it is feasible. If radiation therapy wasn't part of the initial treatment, it may be tried as well.

In rare cases, surgery may be used for cancers that recur at distant sites, such as if there is a small recurrence in a lung.

What happens during and after treatment for rhabdomyosarcoma?

During and after treatment for rhabdomyosarcoma (RMS), the main concerns for most families are the immediate and long-term effects of the tumor and its treatment, and concerns about the possibility of the tumor coming back.

It is certainly normal to want to put the tumor and its treatment behind you and to get back to a life that doesn't revolve around cancer. But it's important to realize that close follow-up care is a central part of this process that offers your child the best chance for recovery and long-term survival.

Possible long-term side effects of treatments for rhabdomyosarcoma

Because more children with rhabdomyosarcoma (RMS) are surviving longer, it is now possible to look at the long-term effects of their treatment. It's important to discuss what these possible effects might be with your child's medical team before starting treatment. Doctors try to limit these potential side effects as much as possible when planning treatment.

The long-term effects of surgery depend a great deal on the location and extent of the tumor(s). Some operations may result in few physical changes other than a scar, while more involved operations may lead to changes in appearance or in how some parts of the body function, and may require physical rehabilitation.

Some chemotherapy drugs may damage cells in the ovaries or testicles, which can affect a patient's ability to have children later on. For parents, it's important to discuss this with your child's health care team before treatment. In some cases there may be ways to help preserve fertility.

The long-term side effects of radiation therapy may be significant, especially for young children. Bones and soft tissues that are irradiated do not grow very well. Depending on the area getting radiation, this may result in curvature of the spine, a shortened arm or leg, limited motion of a joint, hardening of the surrounding soft tissue, stiffening of the lungs, poor development of the facial bones, cataracts and poor vision of the involved eye, later problems with sexual function, and other problems. Young children's brains are especially sensitive to radiation to the head, which can lead to learning problems or other issues, so doctors do their best to avoid this when possible.

Another unwanted long-term result is the small, but definitely increased, risk of second cancers in survivors who had chemotherapy and radiation therapy. These cancers include bone cancer, leukemia, or other soft tissue tumors. The bone cancers seem to be linked with radiotherapy, while the leukemias are more often seen after treatment with cyclophosphamide and related drugs.

It is important to remember that these second cancers affect only a small number of rhabdomyosarcoma survivors, and these are children who most likely would not have survived without these treatments.

Long-term follow-up care for children

To help increase awareness of late effects and improve follow-up care of childhood cancer survivors throughout their lives, the Children's Oncology Group (COG) has developed long-term follow-up guidelines for survivors of childhood cancers. These guidelines can help you know what to watch for, what screening tests should be done to look for problems, and how late effects may be treated.

It is very important to discuss possible long-term complications with your child's health care team, and to make sure there is a plan in place to watch for these problems and treat them, if needed. 

Keeping good medical records

As much as you might want to put the experience behind you once treatment is completed, it is very important to keep good records of your child's medical care during this time. Gathering these details soon after treatment may be easier than trying to get them at some point in the future. There are certain pieces of information that your child's doctors should have, even after your child has become an adult. These include:

A copy of the pathology report(s) from any biopsies or surgeries.

If there was surgery, a copy of the operative report(s).

If your child stayed in the hospital, a copy of the discharge summaries that doctors prepare when patients are sent home.

If chemotherapy was given, a list of the final doses of each chemotherapy drug or other drug your child received. (Certain chemotherapy drugs have specific long-term side effects. If you can get a list of these from the pediatric oncologist, this might also help any new primary care doctor.)

If radiation therapy was given, a summary of the type and dose of radiation and when and where it was given.

It is also very important to keep your health insurance. Tests and doctor visits cost a lot, and even though no one wants to think of the tumor coming back, this could happen.

What`s new in rhabdomyosarcoma research and treatment?

The treatment of rhabdomyosarcoma (RMS) has come a long way in the past few decades, largely due to the work of the Intergroup Rhabdomyosarcoma Study Group (now known as the Soft Tissue Sarcoma Committee of the Children's Oncology Group). However, more work needs to be done.

Better classification of rhabdomyosarcomas

Newer molecular techniques may help better categorize RMS and predict which patients will respond best to certain treatments. For example, rather than just looking at the cancer cells under a microscope, researchers have begun to use special genetic tests to help classify RMS. About 1 out of 4 cancers that doctors would usually classify as alveolar rhabdomyosarcoma (ARMS) have been found to lack the typical gene change (the PAX/FOXO1 fusion gene) seen in ARMS. Some early studies have shown that these cancers seem to act more like embryonal rhabdomyosarcoma (ERMS) than ARMS. ERMS generally requires less intensive treatment than ARMS. If this finding is confirmed in other studies, it may allow doctors to use less intensive treatments on these cancers and still achieve the same results.

Improving standard treatments

A major goal of current research is to treat all patients more effectively, while reducing exposure to intensive treatments (and their side effects) when possible. For example, researchers are studying whether children who have a low risk of the tumor recurring can be successfully treated without using potentially harmful treatments such as radiation therapy and the chemotherapy drug cyclophosphamide.

Because children's bodies are very sensitive to radiation, doctors are looking for ways to limit the doses as much as possible. Newer radiation therapy techniques allow doctors to aim the radiation more precisely, limiting the amount that reaches normal body tissues. Some of these techniques were described in the section "Radiation therapy for rhabdomyosarcoma", and other approaches are now being studied. For example, in stereotactic radiation therapy, a special machine allows the radiation to be aimed at the tumor from many different angles.

Proton beam radiation is another new approach. Standard radiation beams give off the same amount of radiation at all points in the body as they pass through it. Proton beam radiation uses radioactive particles that travel only a certain distance before releasing most of their energy. Doctors can use this property to limit the radiation reaching normal body tissues. This new approach seems promising, but it is not yet clear if it is better than other newer forms of radiation therapy. It is also expensive and is only available in a handful of centers around the country at this time.

Doctors are studying adding newer chemotherapy drugs such as irinotecan and temozolomide to the standard chemotherapy regimens in those who have a higher risk of the tumor recurring.

For patients at a high risk of tumor recurrence, doctors are looking at maximizing the early treatment with drugs such as cyclophosphamide and ifosfamide by giving them more frequently (a concept called interval compression).

Newer treatment approaches

Drugs that target specific parts of cancer cells (as opposed to just attacking fast-growing cells, as chemotherapy drugs do) are now being studied for use in RMS. Some of these drugs are already being used to treat certain adult cancers. Examples of newer targeted drugs being studied for use against RMS include:

IGF-1 receptor inhibitors, such as cixutumumab (IMC-1A2)

Drugs that affect a tumor's ability to make new blood vessels, such as bevacizumab (Avastin) and sorafenib (Nexavar)

Drugs that target the mTOR protein, such as temsirolimus (Torisel) and everolimus (Afinitor)

Drugs that target the ALK protein, such as crizotinib (Xalkori)

Dasatinib (Sprycel)

Researchers are also testing other new ways to treat RMS. For example, some researchers are looking at exposing some of the body's own immune system cells, called dendritic cells, to the abnormal PAX-FOXO1 protein that is found in many ARMS cells. The hope is that the dendritic cells will then cause the immune system to attack these cells, no matter where they are in the body.

Eventually, a combination of these approaches may prove to be the best way to treat this disease.

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