What is Neuroblastoma?

Neuroblastoma is a cancer that develops from immature nerve cells found in several areas of the body.

Neuroblastoma most commonly arises in and around the adrenal glands, which have similar origins to nerve cells and sit atop the kidneys. However, neuroblastoma can also develop in other areas of the abdomen and in the chest, neck and near the spine, where groups of nerve cells exist.

Neuroblastoma most commonly affects children age 5 or younger, though it may rarely occur in older children.

Some forms of neuroblastoma go away on their own, while others may require multiple treatments. Your child's neuroblastoma treatment options will depend on several factors.

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Before Neuroblastoma Cancer Treatment


Signs and symptoms of neuroblastoma vary depending on what part of the body is affected.

Neuroblastoma in the abdomen — the most common form — may cause signs and symptoms such as:

. Abdominal pain

. A mass under the skin that isn't tender when touched

. Changes in bowel habits, such as diarrhea or constipation

Neuroblastoma in the chest may cause signs and symptoms such as:

. Wheezing

. Chest pain

. Changes to the eyes, including drooping eyelids and unequal pupil size

Other signs and symptoms that may indicate neuroblastoma include:

. Lumps of tissue under the skin

. Eyeballs that seem to protrude from the sockets (proptosis)

. Dark circles, similar to bruises, around the eyes

. Back pain

. Fever

. Unexplained weight loss

. Bone pain

When to see a doctor

Contact your child's doctor if your child has any signs or symptoms that worry you. Mention any changes in your child's behavior or habits.


In general, cancer begins with a genetic mutation that allows normal, healthy cells to continue growing without responding to the signals to stop, which normal cells do. Cancer cells grow and multiply out of control. The accumulating abnormal cells form a mass (tumor).

Neuroblastoma begins in neuroblasts — immature nerve cells that a fetus makes as part of its development process.

As the fetus matures, neuroblasts eventually turn into nerve cells and fibers and the cells that make up the adrenal glands. Most neuroblasts mature by birth, though a small number of immature neuroblasts can be found in newborns. In most cases, these neuroblasts mature or disappear. Others, however, form a tumor — a neuroblastoma.

It isn't clear what causes the initial genetic mutation that leads to neuroblastoma.

Risk factors

Children with a family history of neuroblastoma may be more likely to develop the disease. Yet, familial neuroblastoma is thought to comprise a very small number of neuroblastoma cases. In most cases of neuroblastoma, a cause is never identified.


Complications of neuroblastoma may include:

. Spread of the cancer (metastasis). Neuroblastoma may spread (metastasize) to other parts of the body, such as the lymph nodes, bone marrow, liver, skin and bones.

. Spinal cord compression. Tumors may grow and press on the spinal cord, causing spinal cord compression. Spinal cord compression may cause pain and paralysis.

. Signs and symptoms caused by tumor secretions. Neuroblastoma cells may secrete certain chemicals that irritate other normal tissues, causing signs and symptoms called paraneoplastic syndromes. One paraneoplastic syndrome that occurs rarely in people with neuroblastoma causes rapid eye movements and difficulty with coordination. Another rare syndrome causes abdominal swelling and diarrhea.

Can I prevent neuroblastoma?

It isn’t possible to prevent neuroblastoma. If you or your partner had neuroblastoma as a child or have a family history of the disease, talk to your provider. All children diagnosed with neuroblastoma should receive genetic counseling to see if genetic testing is needed. Genetic testing can tell you if your child has the gene markers for familial (inherited) neuroblastoma. However, it is important to remember that the inherited form of neuroblastoma is very rare – only 1% to 2% of cases. Additionally, some gene mutations that increase the risk of getting neuroblastoma are passed down through families – for instance, patients with Li Fraumeni syndrome (or a mutation in the p53 gene, which forms a tumor suppressor protein) are at increased risk for many types of cancers, including neuroblastoma.


Tests and procedures used to diagnose neuroblastoma include:

. Physical exam. Your child's doctor conducts a physical exam to check out any signs and symptoms. The doctor will ask you questions about your child's habits and behaviors.

. Urine and blood tests. These may indicate the cause of any signs and symptoms your child is experiencing. Urine tests may be used to check for high levels of certain chemicals that result from the neuroblastoma cells producing excess catecholamines.

. Imaging tests. Imaging tests may reveal a mass that can indicate a tumor. Imaging tests may include an X-ray, ultrasound, computerized tomography (CT) scan, metaiodobenzylguanidine (MIBG) scan and magnetic resonance imaging (MRI), among others.

. Removing a sample of tissue for testing. If a mass is found, your child's doctor may want to remove a sample of the tissue for laboratory testing (biopsy). Specialized tests on the tissue sample can reveal what types of cells are involved in the tumor and specific genetic characteristics of the cancer cells. This information helps your child's doctor devise an individualized treatment plan.

. Removing a sample of bone marrow for testing. Your child may also undergo bone marrow biopsy and bone marrow aspiration procedures to see if neuroblastoma has spread to the bone marrow — the spongy material inside the largest bones where blood cells are formed. In order to remove bone marrow for testing, a needle is inserted into your child's hipbone or lower back to draw out the marrow.


Once neuroblastoma is diagnosed, your child's doctor may order further testing to determine the extent of the cancer and whether it has spread to distant organs — a process called staging. Knowing the cancer's stage helps the doctor decide what treatment is most appropriate.

Imaging tests used to stage cancer include X-rays, bone scans, and CT, MRI and MIBG scans, among others.

During Neuroblastoma Cancer Treatment

What are the stages of neuroblastoma?

Healthcare providers classify neuroblastoma in children based on how advanced the cancer is and how fast it’s growing. They also consider whether it has spread (metastasized) to other parts of the body.

Using this information, providers determine the cancer’s risk level. Then they choose the most appropriate treatments. The stage of neuroblastoma used to be determined by how much neuroblastoma was found in the body after surgery. Now, the International Neuroblastoma Risk Group Staging System (INRGSS) is used. The stage of neuroblastoma is determined by how much tumor spread is seen on initial imaging studies (such as CT scan or MRI, as discussed below), called “image-defined risk factors.” The INRG stages of neuroblastoma are:

. Stage L1: This is the stage with the lowest risk. L1 tumors are confined to one body compartment and have not spread. Also, the tumor does not involve vital structures of the body (no image-defined risk factors are present).

. Stage L2: In this stage, the tumor is confined to one body compartment, but cancer cells can spread to regional lymph nodes, for instance. Also, there is involvement of vital structures of the body, such as tumor wrapping around large blood vessels (i.e., at least one image-defined risk factor is present).

. Stage M: In this stage, the cancer cells have spread to more than one body compartment – called “distant metastatic disease.” This stage carries the highest risk.

. Stage MS: This is a “special” category of neuroblastoma, affecting children younger than 18 months of age. In this stage, the cancer cells have spread (or metastasized) to either the skin, liver or bone marrow only. Children with stage MS neuroblastoma generally have an excellent prognosis. Stage MS neuroblastoma is usually considered low-risk disease.


Your child's doctor selects a treatment plan based on several factors that affect your child's prognosis. Factors include your child's age, the stage of the cancer, the type of cells involved in the cancer, and whether there are any abnormalities in the chromosomes and genes.

Your child's doctor uses this information to categorize the cancer as low risk, intermediate risk or high risk. What treatment or combination of treatments your child receives for neuroblastoma depends on the risk category.


Surgeons use scalpels and other surgical tools to remove cancer cells. In children with low-risk neuroblastoma, surgery to remove the tumor may be the only treatment needed.

Whether the tumor can be completely removed depends on its location and its size. Tumors that are attached to nearby vital organs — such as the lungs or the spinal cord — may be too risky to remove.

In intermediate-risk and high-risk neuroblastoma, surgeons may try to remove as much of the tumor as possible. Other treatments, such as chemotherapy and radiation, may then be used to kill remaining cancer cells.


Chemotherapy uses chemicals to destroy cancer cells. Chemotherapy targets rapidly growing cells in the body, including cancer cells. Unfortunately, chemotherapy also damages healthy cells that grow quickly, such as cells in the hair follicles and in the gastrointestinal system, which can cause side effects.

Children with intermediate-risk neuroblastoma often receive a combination of chemotherapy drugs before surgery to improve the chances that the entire tumor can be removed.

Children with high-risk neuroblastoma usually receive high doses of chemotherapy drugs to shrink the tumor and to kill any cancer cells that have spread elsewhere in the body. Chemotherapy is usually used before surgery and before bone marrow transplant.

Radiation therapy

Radiation therapy uses high-energy beams, such as X-rays, to destroy cancer cells.

Children with low-risk or intermediate-risk neuroblastoma may receive radiation therapy if surgery and chemotherapy haven't been helpful. Children with high-risk neuroblastoma may receive radiation therapy after chemotherapy and surgery, to prevent cancer from recurring.

Radiation therapy primarily affects the area where it's aimed, but some healthy cells may be damaged by the radiation. What side effects your child experiences depends on where the radiation is directed and how much radiation is administered.

Bone marrow transplant

Children with high-risk neuroblastoma may receive a transplant using stem cells collected from bone marrow (autologous stem cell transplant).

Before the bone marrow transplant, also known as stem cell transplant, your child undergoes a procedure that filters and collects stem cells from his or her blood. The stem cells are stored for later use. Then high doses of chemotherapy are used to kill any remaining cancer cells in your child's body. Your child's stem cells are then injected into your child's body, where they can form new, healthy blood cells.


Immunotherapy uses drugs that work by signaling your body's immune system to help fight cancer cells. Children with high-risk neuroblastoma may receive immunotherapy drugs that stimulate the immune system to kill the neuroblastoma cells.

Newer treatments

Doctors are studying a newer form of radiation therapy that may help control high-risk neuroblastoma. The treatment uses a radioactive form of the chemical metaiodobenzylguanidine (MIBG). When injected in to the bloodstream, the MIBG travels to the neuroblastoma cells and releases the radiation.

MIBG therapy is sometimes combined with chemotherapy or bone marrow transplant. After receiving an injection of the radioactive MIBG, your child will need to stay in a special hospital room until the radiation leaves his or her body in the urine. MIBG therapy usually takes a few days.

After Neuroblastoma Cancer Treatment

What is the outlook for people with neuroblastoma?

The outlook for children with neuroblastoma varies. Cancer specialists measure cancer outlook by the five-year survival rate. Younger children with low- or intermediate-risk neuroblastoma have a good prognosis and a 90% to 95% survival rate. Older kids and those with high-risk neuroblastoma live cancer-free around 60% of the time. Healthcare providers and researchers continue to search for better treatments for this group of patients, and there are promising treatments on the horizon.

The prognosis depends on several factors, including:

. How old the child was at the time of diagnosis.

. The biological makeup of the tumor.

. If the tumor has spread to lymph nodes or other parts of the body.




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