Patient Information

What is Rhabdomyosarcoma?

 

Rhabdomyosarcoma is a type of soft tissue sarcoma (tumour). Most children with rhabdomyosarcoma are younger than 10 years old. It's more common in boys than girls.

More children than ever are surviving childhood cancer. There are new and better drugs and treatments, and we can now also work to reduce the after-effects of having had cancer in the past.

It's devastating to hear that your child has cancer, and at times it can feel overwhelming. There are many healthcare professionals and support organisations to help you through this difficult time.

Understanding more about the cancer your child has and the treatments that may be used can often help parents to cope. Your child’s specialist will give you more detailed information and if you have any questions it is important to ask the specialist doctor or nurse who knows your child’s individual situation.

Sarcomas are rare types of tumour that develop in the supporting tissues of the body, such as bone, muscle or cartilage. There are 2 main types of sarcomas:

• soft tissue sarcomas can develop in muscle, fat, blood vessels, or in any of the other tissues that support, surround and protect the organs of the body

• bone sarcomas can develop in any of the bones of the skeleton

Rhabdomyosarcoma is the most common of soft tissue sarcomas in children. These tumours develop from muscle or fibrous tissue and can grow in any part of the body.

The most common areas of the body to be affected are around the head and neck, bladder, testes, womb, or vagina.

Sometimes tumours are also found in a muscle or a limb, in the chest or in the abdominal wall. If the tumour is in the head or neck area, it can occasionally spread into the brain or the fluid around the spinal cord.

Causes

The causes of rhabdomyosarcoma are unknown but research is going on all the time. Children with certain rare genetic disorders, such as Li-Fraumeni syndrome, have a higher risk of developing rhabdomyosarcoma.

Symptoms

The most common symptom is a lump or swelling. Other symptoms will depend on the part of the body that’s affected by the rhabdomyosarcoma:

• a tumour in the head or neck area can sometimes cause a blockage (obstruction) and discharge from the nose or throat; occasionally, an eye may appear swollen and protruding

• a tumour in the abdomen (tummy) can cause pain or discomfort in the abdomen and difficulty going to the toilet (constipation)

• a tumour in the bladder may cause symptoms such as blood in the urine and difficulty passing urine (peeing)

How rhabdomyosarcoma is diagnosed

Different tests are usually needed to diagnose a rhabdomyosarcoma. Your child may need a small operation to remove a sample from the tumour (a biopsy) so that it can be examined under a microscope. This is usually done under a general anaesthetic.

Tests may be done to check the exact size of the tumour and to find out if it has spread to any other part of the body. These may include:

• a chest X-ray to check the lungs

• an ultrasound

• CT or MRI scans

• blood tests and bone marrow

Any tests and investigations that your child needs will be explained to you

Staging

The ‘stage’ of a cancer is a term used to describe its size and whether it has spread from where it first started. Knowing the stage helps the doctors decide on the most effective treatment for your child.

The staging system for rhabdomyosarcoma is based on:

• where in the body the tumour started

• if it is in only one part of the body (localised disease), or if it has spread to another part of the body (metastatic disease).

There are different ways of staging rhabdomyosarcoma, and your child’s specialist doctor will explain more about the system they are using.

Treatment

Rhabdomyosarcomas are rare tumours and should be treated at specialist centres. Treatment will depend on the size of the tumour, its position in the body and whether it has spread.

The 3 main types of treatment for soft tissue sarcomas are chemotherapy, surgery and radiotherapy. Your child may have a combination of treatments.

Chemotherapy

Chemotherapy is the use of anti-cancer (cytotoxic) drugs to destroy cancer cells. It can be given:

• to shrink the tumour before surgery

• after surgery to reduce the risk of rhabdomyosarcoma coming back

The drugs used and the length of treatment depends on the type and stage of the rhabdomyosarcoma.

Surgery

If it is possible, your child will have an operation to remove all or as much as possible of the tumour, without damaging surrounding tissue or organs. The operation will depend on the size of the tumour and where it is in your body. The surgeon will explain what is involved. Chemotherapy is usually given before surgery to shrink the tumour and make it easier to remove with surgery. If an operation isn’t possible, both chemotherapy and radiotherapy are given.

Radiotherapy

Radiotherapy treats cancer by using high energy rays, which destroy the cancer cells while doing as little harm as possible to normal cells. It may be given after surgery to the area where the rhabdomyosarcoma started.

Side effects of treatment

The side effects will depend on the treatment being given and the part of the body that is being treated. Your child’s doctor will discuss this with you before treatment starts. Most side effects are short-term (temporary) and gradually disappear once treatment stops.

Chemotherapy may cause side effects such as feeling sick, hair loss, tiredness, and an increased risk of infection. But it can also make your child feel better by relieving any symptoms the tumour is causing.

Radiotherapy can make your child feel tired, and the skin in the area that’s being treated may go red or get darker. Other side effects will depend on the area of the body that is being treated. Your child’s specialist doctor or nurse will explain this.

Late side effects

A small number of children may develop long-term side effects many years after treatment for rhabdomyosarcoma. This depends on the type of treatment your child had. Your child’s doctor or nurse will talk to you about any possible risk of late side effects. Follow-up for children who’ve had cancer includes close monitoring for any signs of any late effects.

Late effects may include a possible reduction in bone growth, infertility, a change in the way the heart and the kidneys work, and a slight increase in the risk of developing another cancer in later life.

Clinical trials

Many children have their treatment as part of a clinical research trial. Trials aim to improve our understanding of the best way to treat an illness, usually by comparing the standard treatment with a new or modified version. Specialist doctors carry out trials for children’s cancer. If appropriate, your child’s medical team will talk to you about taking part in a clinical trial, and will answer any questions you have. Written information is provided to help explain things.

Taking part in a research trial is completely voluntary, and you’ll be given plenty of time to decide if it’s right for your child.

Before any trial is allowed to take place, it must be approved by an ethics committee, which protects the interests of the patients taking part. If you decide to take part in a trial, your doctor or a research nurse must discuss the treatment with you so that you understand the trial and what it means for your child to take part. You may decide not to take part, or you can withdraw from a trial at any stage. You will then receive the best standard treatment available.

Treatment guidelines

These are just for your guidelines only. Sometimes, clinical trials are not available for your child’s tumour. This may be because a recent trial has just finished, or because the tumour is very rare. In these cases, you can expect your doctors and nurses to offer treatment which is agreed to be the most appropriate, using guidelines which have been prepared by experts across the country. The Children’s Cancer and Leukaemia Group (CCLG) is an important organisation which helps to produce these guidelines.

Follow-up care

After treatment, the doctors will regularly check your child to be sure that the cancer has not come back and there are no complications. After a while, you will not need to visit the clinic so often.

If you have specific concerns about your child’s condition and treatment, it’s best to discuss them with your child’s doctor, who knows their situation in detail.

Soft-tissue sarcoma (paediatric)

A Soft-tissue sarcoma is a tumour that develops from muscle or soft tissue. These are sometimes called malignant mesenchymal tumours.

 

Rhabdomyosarcoma is the most common type of soft-tissue sarcoma in childhood. This is a cancer of primitive muscle cells and can develop anywhere in the body. The most common sites are around the head and neck, bladder, or testes in boys.

Who gets soft-tissue sarcoma?

 

About 60 children develop a soft-tissue sarcoma in the UK each year. It is slightly more common in boys than girls. About two-thirds of rhabdomyosarcomas occur in children under six years of age.

The cause of soft-tissue sarcomas is unknown. There may be genetic factors in some children, for example, those with Li Fraumeni syndrome, have an increased risk of developing rhabdomyosarcoma.

Signs and symptomsThe signs and symptoms will depend on where the tumour develops. The most usual sign is a swelling or lump. Other symptoms may include:

• Blocked nose or discharge

• Swollen or protruding eye

• Tummy (abdomen) swelling, discomfort or difficulty going to the toilet (constipation)

• Blood in the urine or difficulty passing urine

 

Tests / Investigations

 

We will need to carry out some tests to find out as much as possible about the type position and size of the tumour. These tests include:

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• Chest X-ray – to see whether the tumour has spread to the lungs.

• Ultrasound scan – this will tell us the position and size of the tumour.

• CT scan or MRI scan – a scan of the affected tissue and chest will be carried out. This will give us more information about the tumour and whether it has spread.

• Biopsy – this is a small operation, usually under general anaesthetic, to remove some tissue.

• Bone marrow aspirate and trephine – these tests will tell us whether the tumour has spread to the bone marrow.

• PET/CT scan – this will tell us whether there is spread to other parts of the body

 

Staging

 

Staging refers to the size of the tumour and whether it has spread. Knowing the stage of the cancer helps the doctors decide on the most appropriate treatment for your child. We use TNM (tumour, node, metastases) staging for soft-tissue tumours with a simplified version as follows:
 

• Stage I: Tumour confined to the organ or tissue of origin with no evidence of lymph node involvement or distant spread (metastases).

• Stage II: Tumour involving one or more adjacent organs or tissues, a collection of fluid (malignant effusion) or multiple tumours in the same organ, with no evidence of lymph node involvement or distant metastases.

• Stage III: Any of the above with lymph node involvement.

• Stage IV: Evidence of distant metastases.

 

Treatment of soft-tissue sarcoma

 

The treatment for soft-tissue sarcoma involves chemotherapy, surgery and radiotherapy. This depends on the site, stage, appearance down the microscope (histology) and response to treatment.

Chemotherapy

 

A combination of anti-cancer drugs is used to destroy the cancer cells. The treatment is normally given every one to three weeks. The exact combination and number of courses depends on the response of the tumour and the risk group. The risk group takes into account the stage, type of tumour, where it is and the age of your child. Some children will need high-dose chemotherapy with a stem cell transplant.

If your child takes part in a clinical trial, the treatment is explained in more detail in the specific trial information sheet. An outline of the treatment will be provided in the form of a ‘road map’.

Surgery

 

An operation may be needed to remove residual tumour following chemotherapy, if the tumour can be removed easily without damaging other tissues.

Radiotherapy

 

Radiotherapy may be needed if surgery is not possible, where there is tumour left behind after surgery, in some limb tumours and those which show certain histological features. See the Children’s Unit booklet Radiotherapy treatment for more details on this type of treatment.

Clinical Trials

 

Clinical trials are medical research trials involving patients that are carried out to try and find new and better treatments. In cancer, clinical trials are most commonly used to try and improve different forms of treatment such as surgery, radiotherapy or chemotherapy.

The treatment being tested may be aimed at:
 

• improving the number of people cured (for example, trying new types of surgery or chemotherapy)

• improving survival

• relieving symptoms of the cancer

• relieving the side effects of treatment

• improving the quality of life or sense of well-being for people with cancer.
   

Clinical trials may also involve research aimed at understanding more about the tumour’s biology. You may be asked to allow us to do research on the tumour sample removed at surgery, or on blood samples.

Well-run clinical trials have led to a significant improvement in the treatment of children with cancer. If you are asked to consider entering your child into a clinical trial you will be given specific information about the trial before deciding whether to take part.

Effects of treatmentAbout two thirds of children with soft-tissue sarcomas are cured, however for the individual child this depends on the risk group and whether the tumour has spread.

General side effects of chemotherapy

 

• Bone marrow suppression (myelosuppression)

• Chemotherapy drugs decrease the production of blood cells by the bone marrow for a variable period of time. This results in low red blood cells (anaemia), low white blood cells (neutropenia) and platelets (thrombocytopenia). Your child may need blood or platelet transfusions and will be at increased risk of infections. The doctors and nurses caring for your child will tell you more about these side effects.

• Nausea and vomiting

• Some of the chemotherapy drugs used may make your child feel sick or vomit. We will give anti-sickness drugs at the same time to stop nausea and vomiting. These are usually very effective.

• Hair loss

• Temporary hair loss is common.

 

Specific Drugs

 

For details of the side effects of individual drugs please see Macmillan specific drug information leaflets.

 

What are the possible long-term effects of treatment?

 

A small number of children develop side effects many years later because of the treatment they have received. These include growth problems, impaired heart and kidney function, impaired fertility and a small risk of developing a second cancer later in life. Teenage boys have the option to store sperm for use in later years.

About five years after treatment finishes we will transfer your child’s care to our long-term follow-up clinic. Your child will be seen at regular intervals in this clinic, indefinitely, so that we can help with any long-term effects of the treatment.

​by the Royal Marsden NHS Foundation Trust