Neuroblastoma

Neuroblastoma is a type of cancer that can affect babies and young children. “Neuro” refers to nerves, and “blastoma” is the medical word for cancer that begins in immature cells. Neuroblastoma grows in immature nerve cells called neuroblasts. This cancer specifically affects neuroblasts in your child’s sympathetic nervous system.

Neuroblastoma usually starts in nerve cells in your child’s adrenal glands. These glands are located in your child’s upper belly (abdomen), just above their kidneys. But neuroblastoma can start in other nerve cells, too — usually those located elsewhere in your child’s belly or in their chest or neck.

Once the initial tumor forms, its cancer cells can spread (metastasize) to other parts of your child’s body, including their:

• Bone marrow
• Bones
• Lymph nodes
• Liver
• Skin
• Brain

Neuroblastoma almost always develops before age 5. The average age at diagnosis is 1 to 2 years. Around 700 to 800 children receive a neuroblastoma diagnosis each year in the U.S. Providers rarely diagnose neuroblastoma in children over age 10 or adults.

Signs and symptoms of neuroblastoma

Neuroblastoma has many possible signs and symptoms. These can vary based on:

• The tumor’s location in your child’s body
• The size of the tumor
• The locations where tumors have spread
• Whether or not the tumors are releasing hormones (when they do, they can cause paraneoplastic syndromes)

A lump is the most common sign parents or caregivers notice. You might feel the lump in your child’s belly area, neck or chest. The lump might feel hard to the touch. If you press on it, your child likely won’t feel any pain.

Tumors may press on nearby structures inside your child’s body, like their windpipe, esophagus, nerves, blood vessels or lymph vessels. This can lead to a wide range of neuroblastoma signs and symptoms, such as:

• Swelling in different parts of your child’s body, like their belly, chest, neck or face
• Lack of interest in eating or feeling, like their belly is too full to eat
• Trouble peeing or pooping
• Problems with balance, coordination or movement
• Coughing or difficulty breathing
• Difficulty swallowing
• Symptoms of Horner syndrome, including a droopy upper eyelid and one pupil that’s smaller than the other

Signs of tumor spread

If tumors spread to nearby tissues (like lymph nodes) or farther away, signs and symptoms might include:

• Swollen lymph nodes, typically above your child’s collarbone, in their armpits or in their groin area
• Bone pain that makes it hard for your child to move or walk, or wakes them up at night
• A feeling of weakness or numbness in your child’s arms or legs
• Dark circles around your child’s eyes
• Bulging eyes
• Tiredness, weakness or irritability
• Frequent illness
• Fever that doesn’t go away
• Bruising or bleeding more than expected from minor bumps or cuts
• Skin that’s paler than usual

Signs of paraneoplastic syndromes

If the tumors release hormones, your child may experience:

• Constant diarrhea
• Fever
• Fast heart rate
• Sweating
• Flushed or discolored skin
• High blood pressure

Neuroblastoma causes

Experts don’t fully understand what causes neuroblastoma. They do know this type of cancer occurs when immature nerve cells (neuroblasts) keep growing and dividing when they shouldn’t. These abnormal cells build up and form a tumor. But experts are still trying to figure out what causes this process to start in the first place. They believe gene mutations likely play a role.

Gene mutations are changes to your child’s genes that affect how they work. Certain genes help cells in your child’s body grow and divide normally. Other genes prevent cells from growing out of control or fix mistakes that happen when cells divide.

Any changes to those genes can keep them from doing their jobs. Experts believe certain gene mutations may happen during fetal development or soon after birth that allow some neuroblasts to grow out of control.

In the vast majority of cases, the gene changes happen randomly. Experts haven’t found any specific chemicals or exposures that raise a person’s risk of having a baby with neuroblastoma.

Rarely, neuroblastoma runs in families. About 1 or 2 out of every 100 children with neuroblastoma inherit the gene change from a biological parent. Providers call this hereditary or familial neuroblastoma.

No matter the exact cause, it’s not your fault that your child has neuroblastoma. Experts haven’t identified any ways to prevent the condition, and there’s nothing you can or should have done differently.

What genes are associated with neuroblastoma?

Children with hereditary neuroblastoma typically have changes to one of these genes:

• ALK gene (most common)
• PHOX2B gene

Experts continue to look into which gene mutations might occur in nonhereditary forms of neuroblastoma.

Some genes within tumor cells affect how the cancer progresses. For example:

• The MYCN gene is a type of oncogene, or a gene that may lead to cancer if it undergoes any changes. One important change is called amplification. It’s when there are too many copies of the same gene within a single cell. Experts know that amplification of MYCN within tumor cells can make the tumor grow fast. It can also make treatment more challenging.
• Changes to the ATRX gene cause the tumor to grow at a slower rate. But the tumor may be more difficult to cure.
• Sometimes, the NTRK1 gene is more active than usual in the tumor cells. If this happens, your child may have a better prognosis.

Changes in the tumor cells’ chromosomes also matter. Chromosomes are threadlike structures in cells that are made of proteins and DNA. Experts can examine:

• The number of chromosomes in the tumor cells
• Whether or not whole or partial chromosomes are gained or lost inside the tumor cells

This information helps experts define how aggressive the tumor is and what risk category your child will be placed in.

How doctors diagnose this condition

Healthcare providers diagnose neuroblastoma by doing physical exams and running tests. During the physical exam, your child’s provider will:

• Talk to you about what you’ve noticed: They’ll ask about your child’s symptoms and when they began. They’ll also want to learn more about your child’s medical history and their biological family history, including whether someone has had neuroblastoma.
• Look for signs of neuroblastoma: They’ll gently feel different areas, like your child’s belly, chest and neck, to see if there are any lumps or swelling. They’ll examine your child’s eye area to look for issues like bulging or bruising.
• Check nerve function: They’ll see how your child’s nervous system is working by checking their reflexes and coordination. Tumors near your child’s spinal cord may affect how well they can move.

Providers can learn a lot from the physical exam. If they think your child may have neuroblastoma, they’ll run tests to confirm a diagnosis and see if the cancer has spread. Possible tests include:

• Blood tests: These check blood cell counts and help providers understand your child’s liver and kidney function. Providers may also check general proteins that can be elevated in advanced neuroblastoma, called lactate dehydrogenase (LDH), ferritin or uric acid.
• Pee (urine) tests: These tests can check for elevated levels of substances called homovanillic acid (HVA) and vanillylmandelic acid (VMA) in your child’s urine. The levels will be higher than normal about 90% of the time if tumors are present.
• Imaging tests: These show tumor location. An ultrasound may be the first imaging test your child receives, especially if they’re very young. Sometimes, providers start with a CT scan or an MRI scan. They may also do an MIBG scan to see if tumors have spread.
• Tumor biopsy: Providers take samples from tumors for testing in a lab. A biopsy test lets providers know for sure that what they’re seeing on imaging tests is neuroblastoma.
• Bone marrow aspiration and biopsy: Providers remove bone marrow (usually from pelvic bones) for lab testing. These procedures can confirm a diagnosis in place of a tumor biopsy or, if there’s already a diagnosis, show if cancer has spread to the bone marrow.

Providers may give your child anesthesia so they’re asleep during certain tests, like:

• Biopsies
• Scans (for babies or young toddlers)

This makes the tests easier for the providers to do, and your child won’t feel any discomfort.

Neuroblastoma stages

Healthcare providers classify neuroblastoma 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 your child’s body.

The stages of neuroblastoma include:

• Stage L1 (localized): The tumors are confined to one body area (like your child’s belly, chest or neck) and haven’t spread. Also, the tumor doesn’t affect vital structures, like major blood vessels.
• Stage L2 (locoregional): Tumors have spread, but they’re still limited to the same initial body area. They may have spread to nearby tissues, like lymph nodes. In addition, tumors affect a vital body structure. For example, a tumor might wrap around a large blood vessel.
• Stage M (metastatic): Cancer cells have spread to more than one body area. This is sometimes called “distant metastatic disease.”
• Stage MS: This is a special category of neuroblastoma. It affects some children younger than 18 months of age. In this stage, the cancer cells have spread to either their skin, liver or bone marrow only. Children with this stage generally have an excellent prognosis.

Testing helps providers identify the stage of neuroblastoma for your child. Then, they use the stage and other information to identify the risk group.

Neuroblastoma risk groups

Risk groups describe the chances of a tumor responding to treatment. Knowing the risk group helps providers plan the most effective treatment for your child.

The three risk groups are:

• Low-risk neuroblastoma: These tumors are least likely to pose a danger after treatment. There’s a good chance treatment will cure the cancer. Sometimes, no treatment is needed at all. Providers may just watch the tumor with serial ultrasounds or MRIs, and the tumor will shrink over time on its own.
• Intermediate-risk neuroblastoma: There’s still a good chance treatment can cure the cancer.
• High-risk neuroblastoma: These tumors are hard to cure. They may not respond to treatment. Or they may go away but then come back (recur).

Your child might need multiple and more aggressive treatments if they have high-risk neuroblastoma. Treatment may be more conservative if they have low-risk neuroblastoma.

Your child’s care team will assign a risk group based on:

• Your child’s age
• The cancer stage (stage L1 carries the lowest risk, while stage M carries the highest risk)
• The tumor’s appearance under a microscope
• Any gene changes in the tumor cells

How is neuroblastoma treated?

Neuroblastoma treatment depends on your child’s risk group. Your child may need one or more of the following:

• Chemotherapy, possibly in high doses and/or with autologous stem cell transplantation
• Surgery to remove the tumor and any cancer cells that have spread
• Radiation therapy
• Immunotherapy
• Targeted therapy, like dinutuximab
• Medications (like 13-cis-retinoic acid) for several months after treatment

Your child’s care team will include a pediatric oncologist, as well as many other specialists. They’ll explain which treatments are necessary and the best timing for them. They’ll also explain the benefits and risks and what you can expect as your child recovers from treatment.

Clinical trials may be an option for your child. Your child’s care team can tell you more about possible trials and what they involve.

Is neuroblastoma cancer curable?

Treatment can often cure low-risk and intermediate-risk neuroblastomas, and some high-risk neuroblastomas. But each child is different, and it’s hard to predict treatment outcomes. Your child’s healthcare team can give you a better idea of what to expect.

What questions should I ask my child’s healthcare provider?

Here are some questions that may help you learn more about your child’s condition:

• Where is the tumor located?
• Has the cancer spread?
• What’s the cancer’s risk group?
• What treatment do you recommend?
• Are there clinical trials my child can participate in?
• Who will lead my child’s medical team?
• Who can I talk to if I’m worried about managing the cost of treatment?
• Is there a local support group I can join?

What can I expect if my child has neuroblastoma?

Your child’s care team can give you the most accurate picture of how neuroblastoma may affect your child’s health and lifespan. They have detailed knowledge about the tumors and their likelihood of responding to treatment.

In general, providers use the neuroblastoma risk groups (low, intermediate or high) to plan treatment and develop a prognosis. The lower the risk group, the better the chances of a good prognosis.

What is the survival rate for neuroblastoma?

The five-year survival rate for neuroblastoma varies by risk group. Here’s what researchers have found as of 2025:

• More than 95 out of 100 children in the low-risk group are alive five years after their diagnosis.
• About 90 to 95 out of 100 children in the intermediate-risk group are alive five years after their diagnosis.
• Around 50 to 60 out of 100 children in the high-risk group are alive five years after their diagnosis. With new treatments called maintenance therapy, these numbers have increased. Researchers continue to study different treatments to improve the survival rate for children with high-risk neuroblastoma.

Remember that numbers only tell part of the story. Your child’s care team will tell you more about what you might expect based on your child’s unique medical history and how they respond to treatment.