Unusual Cancers of Childhood Treatment (PDQ®)–Patient Version - National Cancer Institute

Unusual Cancers of Childhood Treatment (PDQ®)–Patient Version

General Information About Unusual Cancers of Childhood

KEY POINTS

Unusual cancers of childhood are cancers rarely seen in children.
Tests are used to detect (find), diagnose, and stage unusual cancers of childhood.
There are three ways that cancer spreads in the body.
Cancer may spread from where it began to other parts of the body.

Unusual cancers of childhood are cancers rarely seen in children.

Cancer in children and adolescents is rare. Since 1975, the number of new cases of childhood cancer has slowly increased. Since 1975, the number of deaths from childhood cancer has decreased by more than half.

The unusual cancers discussed in this summary are so rare that most children's hospitals are likely to see less than a handful of some types in several years. Because the unusual cancers are so rare, there is not a lot of information about what treatment works best. A child's treatment is often based on what has been learned from treating other children. Sometimes, information is available only from reports of the diagnosis, treatment, and follow-up of one child or a small group of children who were given the same type of treatment.

Many different cancers are covered in this summary. They are grouped by where they are found in the body.

Tests are used to detect (find), diagnose, and stage unusual cancers of childhood.

Tests are done to detect, diagnose, and stage cancer. The tests used depend on the type of cancer. After cancer is diagnosed, tests are done to find out if cancer cells have spread from where the cancer began to other parts of the body. The process used to find out if cancer cells have spread to other parts of the body is called staging. The information gathered from the staging process determines the stage of the disease. It is important to know the stage in order to plan the best treatment.

The following tests and procedures may be used to detect, diagnose, and stage cancer:

Physical exam and health history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient’s health habits and past illnesses and treatments will also be taken.
Blood chemistry studies: A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease.
X-ray: An x-ray is a type of energy beam that can go through the body and onto film.
CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.
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Computed tomography (CT) scan of the abdomen. The child lies on a table that slides through the CT scanner, which takes x-ray pictures of the inside of the abdomen.
PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do.
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Positron emission tomography (PET) scan. The child lies on a table that slides through the PET scanner. The head rest and white strap help the child lie still. A small amount of radioactive glucose (sugar) is injected into the child's vein, and a scanner makes a picture of where the glucose is being used in the body. Cancer cells show up brighter in the picture because they take up more glucose than normal cells do.
MRI (magnetic resonance imaging): A procedure that uses a magnet and radio waves to make a series of detailed pictures of areas inside the body. The pictures are made by a computer. This procedure is also called nuclear magnetic resonance imaging (NMRI).
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Magnetic resonance imaging (MRI) of the abdomen. The child lies on a table that slides into the MRI scanner, which takes pictures of the inside of the body. The pad on the child’s abdomen helps make the pictures clearer.
Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later.
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Abdominal ultrasound. An ultrasound transducer connected to a computer is pressed against the skin of the abdomen. The transducer bounces sound waves off internal organs and tissues to make echoes that form a sonogram (computer picture).
Endoscopy: A procedure to look at organs and tissues inside the body to check for abnormal areas. An endoscope is inserted through an incision (cut) in the skin or opening in the body, such as the mouth or rectum. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of disease.
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Upper endoscopy. A thin, lighted tube is inserted through the mouth to look for abnormal areas in the esophagus, stomach, and first part of the small intestine.
Bone scan: A procedure to check if there are rapidly dividing cells, such as cancer cells, in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones with cancer and is detected by a scanner.
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Bone scan. A small amount of radioactive material is injected into the child's vein and travels through the blood. The radioactive material collects in the bones. As the child lies on a table that slides under the scanner, the radioactive material is detected and images are made on a computer screen.
Biopsy: The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. There are many different types of biopsy procedures. The most common types include the following:
- Fine-needle aspiration (FNA) biopsy: The removal of tissue or fluid using a thin needle.
- Core biopsy: The removal of tissue using a wide needle.
- Incisional biopsy: The removal of part of a lump or a sample of tissue that doesn’t look normal.
- Excisional biopsy: The removal of an entire lump or area of tissue that doesn’t look normal.

There are three ways that cancer spreads in the body.

Cancer can spread through tissue, the lymph system, and the blood:

Tissue. The cancer spreads from where it began by growing into nearby areas.
Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body.
Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body.

Cancer may spread from where it began to other parts of the body.

When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood.

Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body.
Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body.

The metastatic tumor is the same type of cancer as the primary tumor. For example, if thyroid cancer spreads to the lung, the cancer cells in the lung are actually thyroid cancer cells. The disease is metastatic thyroid cancer, not lung cancer.

Many cancer deaths are caused when cancer moves from the original tumor and spreads to other tissues and organs. This is called metastatic cancer. This animation shows how cancer cells travel from the place in the body where they first formed to other parts of the body.

Treatment Option Overview

KEY POINTS

There are different types of treatment for children with unusual cancers.
Children with unusual cancers should have their treatment planned by a team of health care providers who are experts in treating cancer in children.
Nine types of standard treatment are used:
- Surgery
- Radiation therapy
- Chemotherapy
- High-dose chemotherapy with autologous stem cell rescue
- Hormone therapy
- Immunotherapy
- Watchful waiting
- Targeted therapy
- Embolization
New types of treatment are being tested in clinical trials.
- Gene therapy
Patients may want to think about taking part in a clinical trial.
Patients can enter clinical trials before, during, or after starting their cancer treatment.
Follow-up tests may be needed.
Treatment for unusual cancers of childhood may cause side effects.

There are different types of treatment for children with unusual cancers.

Different types of treatments are available for children with cancer. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.

Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.

Children with unusual cancers should have their treatment planned by a team of health care providers who are experts in treating cancer in children.

Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with cancer and who specialize in certain areas of medicine. These may include the following specialists:

Nine types of standard treatment are used:

Surgery

Surgery is a procedure used to find out whether cancer is present, to remove cancer from the body, or to repair a body part. Palliative surgery is done to relieve symptoms caused by cancer. Surgery is also called an operation.

After the doctor removes all the cancer that can be seen at the time of the surgery, some patients may be given chemotherapy or radiation therapy after surgery to kill any cancer cells that are left. Treatment given after the surgery, to lower the risk that the cancer will come back, is called adjuvant therapy.

Radiation therapy

Radiation therapy is a cancer treatment that uses high energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are different types of radiation therapy:

External radiation therapy uses a machine outside the body to send radiation toward the cancer.
Proton beam radiation therapy is a type of high-energy, external radiation therapy. A radiation therapy machine aims streams of protons (tiny, invisible, positively-charged particles) at the cancer cells to kill them. This type of treatment causes less damage to nearby healthy tissue.
Internal radiation therapy uses a radioactive substance that is injected into the body or sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer.
131I-MIBG (radioactive iodine) therapy is a type of internal radiation therapy used to treat pheochromocytoma and paraganglioma. Radioactive iodine is given by infusion. It enters the bloodstream and collects in certain kinds of tumor cells, killing them with the radiation that is given off.

The way the radiation therapy is given depends on the type of cancer being treated.

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can affect cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, a body cavity such as the abdomen, or an organ, the drugs mainly affect cancer cells in those areas. Combination chemotherapy is treatment using more than one anticancer drug. The way the chemotherapy is given depends on the type and stage of the cancer being treated.

High-dose chemotherapy with autologous stem cell rescue

High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell rescue is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

Hormone therapy

Hormone therapy is a cancer treatment that removes hormones or blocks their action and stops cancer cells from growing. Hormones are substances that are made by glands in the body and flow through the bloodstream. Some hormones can cause certain cancers to grow. If tests show that the cancer cells have places where hormones can attach (receptors), drugs, surgery, or radiation therapy is used to reduce the production of hormones or block them from working. Hormone therapy with drugs called corticosteroids may be used to treat thymoma or thymic carcinoma.

Hormone therapy with a somatostatin analogue (octreotide or lanreotide) may be used to treat neuroendocrine tumors that have spread or cannot be removed by surgery. Octreotide may also be used to treat thymoma that does not respond to other treatment. This treatment stops extra hormones from being made by the neuroendocrine tumor. Octreotide or lanreotide are somatostatin analogues which are injected under the skin or into the muscle. Sometimes a small amount of a radioactive substance is attached to the drug and the radiation also kills cancer cells. This is called peptide receptor radionuclide therapy.

Immunotherapy

Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This type of cancer treatment is also called biotherapy or biologic therapy.

Interferon: Interferon affects the division of cancer cells and can slow tumor growth. It is used to treat nasopharyngeal cancer and papillomatosis.
Epstein-Barr virus (EBV)-specific cytotoxic T-lymphocytes: White blood cells (T-lymphocytes) are treated in the laboratory with Epstein-Barr virus and then given to the patient to stimulate the immune system and fight cancer. EBV-specific cytotoxic T-lymphocytes are being studied for the treatment of nasopharyngeal cancer.
Vaccine therapy: A cancer treatment that uses a substance or group of substances to stimulate the immune system to find the tumor and kill it. Vaccine therapy is used to treat papillomatosis.
Immune checkpoint inhibitor therapy: Some types of immune cells, such as T cells, and some cancer cells have certain proteins, called checkpoint proteins, on their surface that keep immune responses in check. When cancer cells have large amounts of these proteins, they will not be attacked and killed by T cells. Immune checkpoint inhibitors block these proteins and the ability of T cells to kill cancer cells is increased.
There are two types of immune checkpoint inhibitor therapy:
- CTLA-4 is a protein on the surface of T cells that helps keep the body’s immune responses in check. When CTLA-4 attaches to another protein called B7 on a cancer cell, it stops the T cell from killing the cancer cell. CTLA-4 inhibitors attach to CTLA-4 and allow the T cells to kill cancer cells. Ipilimumab is a type of CTLA-4 inhibitor. Ipilimumab may be considered for the treatment of high-risk melanoma that has been completely removed during surgery. Ipilimumab is also used with nivolumab to treat certain children with colorectal cancer.
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  Immune checkpoint inhibitor. Checkpoint proteins, such as B7-1/B7-2 on antigen-presenting cells (APC) and CTLA-4 on T cells, help keep the body’s immune responses in check. When the T-cell receptor (TCR) binds to antigen and major histocompatibility complex (MHC) proteins on the APC and CD28 binds to B7-1/B7-2 on the APC, the T cell can be activated. However, the binding of B7-1/B7-2 to CTLA-4 keeps the T cells in the inactive state so they are not able to kill tumor cells in the body (left panel). Blocking the binding of B7-1/B7-2 to CTLA-4 with an immune checkpoint inhibitor (anti-CTLA-4 antibody) allows the T cells to be active and to kill tumor cells (right panel).
- PD-1 is a protein on the surface of T cells that helps keep the body’s immune responses in check. When PD-1 attaches to another protein called PDL-1 on a cancer cell, it stops the T cell from killing the cancer cell. PD-1 inhibitors attach to PDL-1 and allow the T cells to kill cancer cells. Nivolumab is a type of PD-1 inhibitor. Nivolumab is used with ipilimumab to treat certain children with colorectal cancer. Pembrolizumab and nivolumab are used to treat melanoma that has spread to other parts of the body. Nivolumab and pembrolizumab are being studied in the treatment of melanoma for children and adolescents. Treatment with these two drugs has been mostly studied in adults.
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  Immune checkpoint inhibitor. Checkpoint proteins, such as PD-L1 on tumor cells and PD-1 on T cells, help keep immune responses in check. The binding of PD-L1 to PD-1 keeps T cells from killing tumor cells in the body (left panel). Blocking the binding of PD-L1 to PD-1 with an immune checkpoint inhibitor (anti-PD-L1 or anti-PD-1) allows the T cells to kill tumor cells (right panel).
  
  Immunotherapy uses the body’s immune system to fight cancer. This animation explains one type of immunotherapy that uses immune checkpoint inhibitors to treat cancer.
BRAF kinase inhibitor therapy: BRAF kinase inhibitors block the BRAF protein. BRAF proteins help control cell growth and may be mutated (changed) in some types of cancer. Blocking mutated BRAF proteins may help keep cancer cells from growing. Dabrafenib, vemurafenib, and encorafenib are used to treat melanoma. Oral dabrafenib is being studied in children and adolescents with melanoma. Treatment with these three drugs has mostly been studied in adults.

Watchful waiting

Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. Watchful waiting may be used when the tumor is slow-growing or when it is possible the tumor may disappear without treatment.

Targeted therapy

Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Types of targeted therapies used to treat unusual childhood cancers include the following:

Tyrosine kinase inhibitors: These targeted therapy drugs block signals needed for tumors to grow. Vandetanib and cabozantinib are used to treat medullary thyroid cancer. Sunitinib is used to treat pheochromocytoma, paraganglioma, neuroendocrine tumors, thymoma, and thymic carcinoma. Crizotinib is used to treat tracheobronchial tumors.
mTOR inhibitors: A type of targeted therapy that stops the protein that helps cells divide and survive. Everolimus is used to treat cardiac, neuroendocrine, and islet cell tumors.
Monoclonal antibodies: This targeted therapy uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Bevacizumab is a monoclonal antibody used to treat papillomatosis.
Histone methyltransferase inhibitors: This type of targeted therapy slows down the cancer cell's ability to grow and divide. Tazemetostat is used to treat ovarian cancer. Tazemetostat is being studied in the treatment of chordomas that have recurred after treatment.
MEK inhibitors: This type of targeted therapy blocks signals needed for tumors to grow. Trametinib and binimetinib are used to treat melanoma that has spread to other parts of the body. Treatment with trametinib or binimetinib has been mostly studied in adults.

Targeted therapies are being studied in the treatment of other unusual cancers of childhood.

Embolization

Embolization is a treatment in which contrast dye and particles are injected into the hepatic artery through a catheter (thin tube). The particles block the artery, cutting off blood flow to the tumor. Sometimes a small amount of a radioactive substance is attached to the particles. Most of the radiation is trapped near the tumor to kill the cancer cells. This is called radioembolization.

New types of treatment are being tested in clinical trials.

This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.

Gene therapy

Gene therapy is a treatment in which foreign genetic material (DNA or RNA) is inserted into a person's cells to prevent or fight disease. Gene therapy is being studied in the treatment of papillomatosis.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their cancer treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.

Treatment for unusual cancers of childhood may cause side effects.

For information about side effects that begin during treatment for cancer, see our Side Effects page.

Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following:

Physical problems.
Changes in mood, feelings, thinking, learning, or memory.
Second cancers (new types of cancer).

Some late effects may be treated or controlled. It is important to talk with your child's doctors about the possible late effects caused by some cancers and cancer treatments. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information).