Rhabdomyosarcoma
is a cancerous tumor that grows in the body's soft
tissues (which connect, support or
surround organs and other body structures), particularly in the muscles that
attach to bone and help the body to move. Just weeks into the life of a
developing embryo, rhabdomyoblast cells (which grow into muscle over time)
begin to form. These are the cells that can develop into rhabdomyosarcoma.
Because this is a cancer of embryonal cells, it is much more common in
children, although it can occur in adults.
Patients with rhabdomyosarcoma are treated at
Dana-Farber/Boston Children's through our Bone & Soft Tissue Tumor Program. Because rhabdomyosarcoma can develop anywhere in a child’s body and will
require surgery as part of treatment, it is important that your child be
treated at a center that offers surgical expertise in the part of the body
where your child’s tumor appears. At Dana-Farber/Boston Children’s we have
urology, gynecology, head and neck, and general surgeons who have specialized
expertise in treating these types of tumors in children.
Our center also offers a range of clinical trials for
rhabdomyosarcoma, and we are New England's hub for the Children's Oncology
Group – an international consortium of cancer treatment centers that conducts
studies of pediatric cancers.
Successfully diagnosing and treating
your child’s rhabdomyosarcoma involves staging and classifying the disease,
which will help your child's doctor determine treatment options and prognosis.
Staging is the process of determining whether the cancer has spread and, if so,
how far.
Rhabdomyosarcoma
is typically divided into two groups. These are:
- Embryonal
rhabdomyosarcoma (ERMS). This is the most common form of the cancer, usually occurring
in children under 6 years of age.
- Alveolar
rhabdomyosarcoma (ARMS). This type is found more frequently in older children
and accounts for about 20 percent
of all cases.
As
a parent, you undoubtedly want to know what may have caused your child’s tumor.
In most cases doctors don't know what leads to rhabdomyosarcoma.
Rhabdomyosarcoma is not associated with environmental factors, and it is
important to keep in mind that there is nothing you could have done, or avoided
doing, that would have prevented the tumor from developing.
Some rhabdomyosarcoma tumors are
thought to 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. Rhabdomyosarcomas usually have some type of chromosome
abnormality or genetic mutation in the cells of the tumor.
Your child
may not experience any symptoms of rhabdomyosarcoma until the tumor is very
large, especially if it is located deep in the muscles or stomach. Common
symptoms may include:
- A
mass that can be seen or felt and may or may not be painful
- Bleeding
from the nose, vagina, rectum or throat (if the tumor is located in these
areas)
- Tingling,
numbness or pain if the tumor compresses a nerve in the affected area
- Protrusion
of the eye or drooping of the eyelid, which may indicate a tumor behind
the eye
Keep
in mind that some similar symptoms can be associated with more common medical
problems and conditions. It is important to consult your child's physician for
a diagnosis.
The first
step in treating your child is forming an accurate and complete diagnosis. A
physician may order a number of different tests to diagnose rhabdomyosarcoma.
In addition to a medical history and physical exam, these may include:
- Blood and urine tests, including a complete
blood count.
- Computerized tomography (CT or CAT) scan –
Detailed images of any part of the body, including the bones, muscles and
fat.
- Magnetic resonance imaging (MRI) – A test
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 that uses
invisible electromagnetic energy beams to produce images of
internal tissues, bones, and organs.
- Ultrasound (also called sonography) – 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 biopsy and/or aspiration – A
procedure that involves taking a small amount of bone marrow fluid
and tissue, usually from part of the hip bones, to examine the number,
size and maturity of blood cells and/or abnormal cells.
- Biopsy – A sample taken of the primary
tumor and/or metastatic lesions
- Lumbar
puncture (spinal tap) – Inserting a special needle through the lower back into
the spinal canal, 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.
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),
now a part of the Children’s Oncology Group which has outlined the treatment of
rhabdomyosarcoma. Dana-Farber/Boston Children’s plays an active role in this
organization.
Treatment
for rhabdomyosarcoma may include:
- Surgery: Surgery is often a first step, allowing
doctors to form a complete diagnosis of the tumor type and providing
information on the stage of the disease. Complete surgical removal of a
rhabdomyosarcoma is often not possible.
- Radiation therapy: This treatment can help stop the
growth of abnormal cells in specific areas of the body. Radiation therapy uses
high-energy rays from a specialized machine to damage or kill abnormal cells.
- Chemotherapy: These medicines can help stop the
growth of abnormal cells throughout the body. Chemotherapy is systemic
treatment, meaning it is introduced to the bloodstream and travels throughout
the body to kill or slow the growth of targeted cells. Different groups of
chemotherapy drugs work in different ways, and can be taken in a variety of
ways including orally or intravenously.
While
chemotherapy can be quite effective in treating certain cancers, the agents
don't completely differentiate normal healthy cells from abnormal cells.
Because of this, your child could have adverse side effects during treatment.
Deciding on which of these
approaches to use depends largely on the tumor's site. For instance, a sarcoma
in the muscles of the arms or legs is often initially treated with surgical
removal, which may be followed by chemotherapy with or without radiation. A
tumor in the bladder or prostate requires chemotherapy prior to attempts at
surgical removal or treatment with radiation. Tumors around the eye are
very responsive to chemotherapy and radiation, so they rarely require surgical
removal.
Receiving
care at the Dana-Farber/Boston Children's means that your child will have
access to cutting-edge therapies being tested by the nation's top researchers
in pediatric cancer. We are the New England Phase I Center for the Children's
Oncology Group, a group of cancer researchers from around the world dedicated
to finding new treatments for pediatric cancer.
Our
scientists are conducting numerous research studies to help doctors better
understand and treat soft-tissue sarcomas. Treatments being evaluated include angiogenesis
inhibitors, which are substances that might be able to prevent the growth of
tumors; and biological therapies, which harness the body's immune system to
fight cancer or lessen side effects.
Clinical
trials
For
many children with rare or hard-to-treat conditions, clinical trials provide
new options
Contact
us: If you’re not sure which clinical trials might be right for your child,
email us at clinicaltrials@danafarberbostonchildrens.org.
We can help you navigate your options.
The prognosis for children with rhabdomyosarcoma varies, but
more than 70% survive five years after diagnosis if they have localized disease
and receive combination therapy. After
five years of disease-free survival, relapses are rare. Prompt medical
attention and aggressive therapy are critical for the best prognosis.
Your child's outcome will likely depend on a number of
factors, including:
- the
extent of the disease
- the size and location of the tumor
- the tumor's characteristics when examined under
a microscope
- the 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
You and
your child will need to monitor the late effects of treatment and continue to
consult with your medical team after your child's cancer is in remission. This
will likely include visiting a cancer
survivorship clinic every year to screen for recurrence and manage disease
complications.
Through the David
B. Perini Jr. Quality of Life Clinic, our cancer survivorship clinic, childhood cancer survivors receive a comprehensive follow-up evaluation from
their cancer care team. In addition to meeting with your pediatric oncologists, your child may see one of our endocrinologists, cardiologists,
neurologists, neuro-psychologists or alternative/complementary therapy specialists. We also offer patient and family education, psychosocial assessment, genetic counseling, reproductive counseling and
opportunities to speak with other childhood cancer survivors.