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What Is Neuroblastoma?
What Are the Symptoms of Neuroblastoma?
How Do You Diagnose Neuroblastoma?
How Do You Treat Neuroblastoma?
What Is Neuroblastoma?
Neuroblastoma is a pediatric cancer that develops in immature nerve tissues, called neuroblasts, in the sympathetic nervous system.
“Neuro” means nerves, while “blastoma” means a cancer that starts in immature cells. Neuroblastoma is a solid tumor or cluster of malformed cells, most commonly found on the top of the kidneys in the adrenal glands. It can also be found in the pelvis, spine, chest, and neck, or spread to the skin or bone marrow.
As a baby develops in the womb, special cells called neural crest cells form in the adrenal gland and grow from an immature to a mature state.
Sometimes a mutation occurs during this transition, causing a neuroblastoma to develop. As a result of this mutation, the cells fail to complete the cycle into maturity. The cells then multiply rapidly and form a mass or tumor.
Neuroblastoma may be discovered in the developing baby during a fetal ultrasound, or it may be found in infants or young children presenting with symptoms.
Symptoms may also mean the cancer has spread, usually to other parts of the chest, bones, or bone marrow. Neuroblastoma is typically diagnosed in children under the age of 5 and makes up approximately 7.5% of all childhood cancers.
How common is neuroblastoma?
While neuroblastoma is a rare cancer, it is the most common cancer among infants, with about 800 young children being diagnosed in the United States every year. It is very rare for a child over 5 to be diagnosed with neuroblastoma.
What causes neuroblastoma?
Neuroblastoma is caused when neuroblasts, struggling to mature, grow rapidly and divide to create a tumor. A genetic mutation or mistake causes the cells to grow uncontrollably, although experts can’t identify exactly what causes these genes to mutate. The mutations are typically found in growth-regulating genes, such as the MYC, ALK, and PHOX2B genes.
What are neuroblastoma risk factors and complications?
It’s important to know your family history of neuroblastoma to help understand the possible need for screening for cancer.
Neuroblastoma risk factors
Your child may be at a higher risk for neuroblastoma if they have:
- A family history of cancer.
- Mutations in MYC, ALK, or PHOX2B genes.
- Other birth defects.
- Other genetic conditions.
Even still, most children with neuroblastoma do not have a family history or other underlying factors, so it can be more difficult to determine who is at higher risk.
What are the stages or risk groups of neuroblastoma?
Doctors determine the stage of neuroblastoma on several factors, including:
- How aggressive the tumor cells are.
- The age of the child — Ages greater than 15 months are associated with more aggressive neuroblastomas.
- Whether the cancer has spread to other places in the body.
Experts use the International Neuroblastoma Risk Group Staging System (INGRSS) to determine the cancer stages based on pretreatment imaging and tumor characteristics. They use image-defined risk factors (IDRFs), which are features of the tumor found on MRIs or CTs, to determine the difficulty of surgical removal.
Here’s what you need to know:
- Stage L1 — The tumor is localized to one body part and has not spread. The tumor does not interfere with any vital structures of the body and can be surgically removed without much concern, meaning there are no IDRFs. This is the lowest risk stage of neuroblastoma.
- Stage L2 — The tumor is still localized to one part of the body and has not spread, but there is one or more IDRFs. This could mean the tumor is interfering with vital structures in the body, such as major blood vessels or the spinal cord, which can make surgical removal more difficult.
- Stage M — This stage means the cancer has spread to other areas of the body, also called metastatic cancer. This stage is the riskiest because the cancer is in multiple parts of the body.
- Stage MS — This stage is categorized for children under 18 months of age whose cancer has metastasized to the bone marrow, liver, or skin. This stage of neuroblastoma is considered low-risk, compared to Stage M.
Risk categories of neuroblastoma
There are three risk categories for neuroblastoma:
- Low-risk neuroblastoma — For some young infants, their tumors may go away on their own without any intervention or treatment. In this risk category, children may still have a combination of surgery and chemotherapy to treat the cancer, but treatment may not be needed right away or perhaps not at all.
- Intermediate-risk neuroblastoma — Children in this category may need a combination of chemotherapy and surgery to cure this tumor type. In some cases, chemotherapy comes before surgery to shrink the tumor. The duration and type of chemotherapy are determined by several factors considered by your child’s care team.
- High-risk neuroblastoma — In this risk category, doctors will want to treat the cancer right away with a combination of surgery, chemotherapy, radiation, and immunotherapy. Some children may need daily medication for several months after treatment.
If your child has extra copies of the MYCN gene, they will be treated as high-risk, regardless of their disease stage. MYCN genes can cause the rapid growth and spread of tumors in the body and must be handled quickly.
How can I prevent neuroblastoma?
Unfortunately, it is impossible to prevent neuroblastoma. If you or your partner have a family history of neuroblastoma, it’s important to talk with your doctor about the increased risk for your baby to be born with the cancer. However, genetic neuroblastoma only accounts for up to 2% of cases.
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What Are the Signs and Symptoms of Neuroblastoma?
Often, neuroblastoma is diagnosed with imaging tests without symptoms.
Neuroblastoma symptoms will likely appear only when the cancer has grown very large or has already spread to other parts of the body. Because of this, symptoms can range from mild to very severe and vary depending on the location of the tumor.
Symptoms of neuroblastoma include:
- Aching of the bones, back, or legs.
- Balance and coordination issues.
- Bulging eyes or droopy eyelids.
- Bloated, painful stomach.
- Dark circles under the eyes.
- Diarrhea or constipation.
- Fatigue.
- Fever.
- High blood pressure.
- Pale skin.
- Trouble breathing or shortness of breath.
- Weakness or paralysis in the arms or legs.
- Uncontrollable eye movement.
Because many conditions have similar symptoms, it is important to call your child’s pediatrician right away if you notice any signs of neuroblastoma. Early detection and diagnosis can impact your child’s prognosis and treatment plan.
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How Do You Diagnose Neuroblastoma?
Neuroblastoma is typically diagnosed before children turn 5. Sometimes doctors are even able to diagnose the cancer during a prenatal ultrasound.
To diagnose neuroblastoma, your child’s doctor will perform a physical and neurological examination to check their nerve function, coordination, and reflexes. Different tests will be ordered to diagnose the stage and condition of the disease.
This testing may include:
- Biopsy — A biopsy involves removing a tissue sample of the tumor and sending it to a lab for testing. This test will confirm a cancer diagnosis and help determine the stage and best treatment.
- CT scan — This scan may include an injected dye that goes into your child’s vein to make it easier to see the tumor in imaging.
- MRI — This scan uses large magnets and radio waves to help see soft tissues more clearly.
- Ultrasound — An ultrasound can help determine the location and size of the tumor.
- X-ray — An x-ray image may be ordered to determine the location of the tumor in the chest or abdomen.
Your child’s doctor may order other, more specialized tests depending on the presenting symptoms and location of the possible cancer, including an MIBG (iodine-123 meta-iodobenzylguanidine) scan.
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How Do You Treat Neuroblastoma?
Neuroblastoma treatment depends on many variables, including your child’s:
- Age.
- Overall health.
- Risk category.
- Stage of cancer.
- Tumor location.
The most common neuroblastoma treatments are chemotherapy, immunotherapy, prescribed medications, radiation, surgery, or other treatments.
Here’s what you need to know about each.
Surgery
Surgery to remove the tumor can be simple or complex, depending on the stage of cancer and the location of the tumor. Your child’s doctor may order chemotherapy before surgery to shrink the tumor, or they may order it after surgery to kill any remaining cancer cells. Some children, including those with low-risk neuroblastoma, will require surgery as their only treatment.
Chemotherapy
Chemotherapy uses drugs to kill cancer cells or stop them from growing. These drugs would most likely be administered through an IV in your child’s vein. These treatments can take weeks to months, depending on the cancer stage and the body’s response.
Additionally, doctors often prescribe cis-retinoic acid to patients with high-risk disease. This medication transforms neuroblastoma tumor cells into benign, mature nerve cells.
Your child’s doctor will work with their team to decide on the type of chemo and the length of treatment based on the variables above.
Stem cell transplant
Stem cell transplant involves removing stem cells from your child's bone marrow to freeze while they receive chemotherapy, then injecting those stem cells back into your child’s bloodstream. The stem cell transplant will help your child’s immune system rebuild after the harsh impact of chemotherapy. This type of therapy is only used for patients with high-risk neuroblastoma.
Immunotherapy
Immunotherapy uses the body’s own immune system to fight cancer cells and is generally only used in cases of high-risk neuroblastoma. This treatment option is typically used after chemotherapy and radiation. Your child’s doctor will inject antibodies through a vein in a process similar to chemotherapy.
Radiation
Radiation works by killing cancer cells using high levels of radiation targeted at the tumor and is typically only used in patients with high-risk neuroblastoma. This method is typically chosen after other treatment methods are completed to minimize the risk of recurrence.
Other therapies
Depending on the condition of your child’s cancer, doctors may look into other, less common treatments.
- Observation — Some children diagnosed within the first year of life with low-stage neuroblastoma may be treated with close observation alone. Your doctor will consider all aspects of your child's tumor to see if they qualify for observation only.
- Iodine 131-MIBG therapy — A less common treatment, MIBG therapy involves injecting radioactive iodine into the bloodstream to kill tumor cells alongside radiation treatment. This type of treatment is mainly used for children whose cancer keeps returning.
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Why Choose UPMC Children’s for Neuroblastoma Care?
At UPMC Children’s Hospital, every child diagnosed with neuroblastoma receives an individualized treatment plan and family-centered care.
- Our surgical team works closely with a multidisciplinary team including oncologists, bone marrow transplant physicians, and radiation oncologists to determine the best plan.
- Cutting-edge research and the latest technology ensure our patients' best possible outcomes.
Learn how to make an appointment with a surgeon at UPMC Children’s.
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By UPMC Editorial Staff. Last reviewed on 2025-11-24.
