Neuroblastoma: What It Is, Symptoms, Causes, and Treatment Options
Updated: 3 days ago
Medically reviewed by Dr. Baraa Alnahhal, MD · Last reviewed: October 17, 2026
Editorial note: This article is for general education. If your child has symptoms that worry you, talk to your child's healthcare professional.
Quick Answer
Neuroblastoma is a cancer of immature nerve cells that mostly affects children under age 5, usually starting in the adrenal glands [1] [3]. Symptoms depend on location and may include a belly lump or bone pain [1] [3]. It is diagnosed with urine tests, imaging and biopsy [2]. Treatment depends on age, stage and risk group, ranging from surgery alone to chemotherapy, transplant and immunotherapy [2].
What the research says
Eflornithine (DFMO) after immunotherapy was linked to better survival in high-risk disease. In a propensity score-matched comparison of 92 children given DFMO against 852 controls, DFMO was associated with improved event-free survival (HR 0.50; 95% CI 0.29–0.84) and overall survival (HR 0.38; 95% CI 0.19–0.76). It was not a randomized trial (Oesterheld et al., J Clin Oncol, 2024) [4]
High-dose chemotherapy with stem cell rescue improved event-free survival. A systematic review of 41 publications found event-free survival favored myeloablative therapy with autologous stem cell transplantation (HR 0.78; 95% CI 0.67–0.91), with a trend for overall survival (HR 0.86; 95% CI 0.73–1.00) (Żebrowska et al., Target Oncol, 2024) [5]
Long-term survivors can face lasting side effects. In a retrospective cohort of 65 children with high-risk neuroblastoma, 33 (50.8%) were long-term survivors; among the 25 survivors evaluated for late effects, 64% had hearing damage, 24% heart toxicity and 76% endocrine toxicity. The cohort was small and treated in the 1991–2002 era (Flaadt et al., Cancers, 2025) [6]
According to PubMed, these studies describe groups of patients, not individual outcomes; one is a small retrospective cohort and one is not randomized.
What Is Neuroblastoma?
Neuroblastoma is a cancer that starts in cells called neuroblasts. Neuroblasts are immature nerve cells, and they are found in several areas of the body [1].
Neuroblastoma most often starts in the neuroblasts in the adrenal glands, which sit on top of each kidney [1]. These small glands make hormones that control important functions in the body. Other types of tumors, such as pheochromocytomas, can also form in the adrenal glands. These are different from neuroblastomas but may cause similar symptoms [1].
The cancer can also develop in other parts of the body that contain neuroblasts, such as the spine, belly, chest, and neck [1]. If it spreads to other places in the body, it is called metastatic neuroblastoma [1].
Key fact | Detail |
Cell of origin | Immature nerve cells called neuroblasts |
Most common location | Adrenal glands on top of the kidneys |
Other locations | Spine, belly, chest, and neck |
Age group most affected | Children age 5 or younger |
Name for cancer that spreads | Metastatic neuroblastoma |
Neuroblastoma is different from other cancers that have similar names, such as glioblastoma and nephroblastoma (also called Wilms tumor). All of these names contain the term "blastoma," which means a cancer or tumor made up of blasts, cells that have not fully developed yet. Neuroblastoma starts in nerve cells that are in the blast stage [1].
Notably, some forms of neuroblastoma may go away on their own, while other forms need treatment [1] [3]. A child's healthcare team selects the treatments that are best for that child.
What are the symptoms of neuroblastoma?
Signs and symptoms of neuroblastoma vary depending on what part of the body is affected [1].
When the cancer is only in the place where it starts, called localized neuroblastoma, it does not always cause symptoms [1]. Symptoms are more likely to appear when the cancer grows into nearby areas (regional neuroblastoma) or spreads to distant parts of the body (metastatic neuroblastoma) [1].
Symptoms by location
Location of the tumor | Possible symptoms |
Adrenal glands and belly | Belly pain; a lump under the skin that typically is not tender when touched; diarrhea or constipation |
Chest and neck | Wheezing; difficulty breathing; eye changes such as drooping eyelids and pupils of different sizes |
Near the spine | Back pain; trouble urinating or passing a bowel movement; weakness in the legs |
Metastatic (spread) | Bone pain that may cause young children to limp; dark, bruise-like circles around the eyes ("raccoon eyes"); fever; fussiness; painless lumps of tissue under the skin; not growing or gaining weight as expected |
When to see a doctor
Contact your child's healthcare professional if your child has any symptoms that worry you. Mention any changes in your child's behavior, habits, or appearance [1].
Because symptoms vary by location and do not always appear early, an evaluation by a healthcare professional is the only way to find out what is causing a child's signs and symptoms [3].
What causes neuroblastoma to develop?
Neuroblastoma has no clearly known cause, though it begins when certain cells develop changes in their DNA [1]. The cancer starts in immature nerve cells called neuroblasts.
Neuroblastoma begins when neuroblasts develop changes in their DNA. A cell's DNA holds the instructions that tell the cell what to do [1].
Healthy cells | Cancer cells |
DNA gives instructions to grow and multiply at a set rate | DNA changes give different instructions |
Cells die at a set time | Cells keep living when healthy cells would die |
Controlled growth | Grow and multiply quickly, causing too many cells |
The cancer cells might form a mass called a tumor. The tumor can grow to invade and destroy healthy body tissue. Over time, cancer cells can break away and spread to other parts of the body. When cancer spreads, it is called metastatic cancer [1].
What are the risk factors for neuroblastoma?
Risk factors are traits or exposures that raise the chance of developing a condition. For neuroblastoma, the known risk factors are limited.
Risk factor | What it means |
Young age | Risk is higher in children; the cancer happens mostly in children age 5 and younger |
Genetic syndromes | Some inherited DNA changes can raise risk, including Turner syndrome, Costello syndrome, and familial neuroblastoma |
Healthcare professionals believe that only a small number of neuroblastomas are inherited [1].
There are no known ways to prevent neuroblastoma [1]. Unlike many adult cancers, there are no proven lifestyle steps, such as diet, exercise, or avoiding exposures, that lower a child's risk.
What complications can neuroblastoma cause?
Neuroblastoma can cause complications that go beyond the tumor itself. Understanding these helps families know what to watch for during and after treatment.
Complication | Description |
Spread of the cancer | Cells may spread to the lymph nodes, bone marrow, liver, skin, and bones (metastatic neuroblastoma) |
Pressure on the spinal cord | A growing tumor may press on the spinal cord, causing spinal cord compression, which may cause pain and paralysis |
Symptoms caused by cancer secretions | Neuroblastoma cells may secrete chemicals that irritate other tissues, causing paraneoplastic syndromes; symptoms may include rapid eye movements, difficulty with coordination, abdominal swelling, and diarrhea |
How Is Neuroblastoma Diagnosed?
A neuroblastoma diagnosis might start with a physical exam. Urine and blood tests can give clues about what might be causing a child's symptoms, and imaging tests can look for signs of a growth [2].
To be certain of the diagnosis, the healthcare team removes some of the growth for testing [2]. If that test finds neuroblastoma, the child may have additional tests to look for signs that the cancer has spread. These might include other imaging tests and a procedure to remove some bone marrow for testing [2].
Tests used in diagnosis
Test | What it involves |
Physical exam | Healthcare professional examines the child for signs of neuroblastoma and asks about symptoms and health history |
Urine and blood tests | Urine tests may look for high levels of chemicals made by neuroblastoma cells, such as catecholamines, which can be detected in urine as the body breaks them down |
Ultrasound | Uses sound waves to make pictures of the body; usually the first test for belly symptoms in children; gives clues about what test to do next |
CT scan | Uses X-rays and a computer to create cross-sectional slices; shows tumor size in the belly or chest and spread to lymph nodes, bones, liver, or skin; results come back quickly |
MRI | Uses a magnetic field and radio waves; particularly good at showing neuroblastoma in and around the spine |
Biopsy | Removes a tissue sample through the skin with a hollow needle, or during surgery; lab tests check for cancer and DNA changes that help shape the treatment plan |
Tests that check whether the cancer has spread
Test | What it involves |
MIBG scan | A radiotracer is placed into a vein, travels through the body, and attaches to neuroblastoma cells; a scanner detects the radiation and makes pictures showing where the cells are; can be repeated during and after treatment to see how well treatment is working |
PET scan | A radioactive tracer highlights fast-growing cells; not often used for neuroblastoma, but may be used if MIBG scans do not show it; MIBG scans do not work for about 1 out of every 10 neuroblastomas |
Bone marrow aspiration and biopsy | Needles collect fluid and small tissue samples, typically from the hip bone, to check if neuroblastoma has spread to the bone marrow |
How is neuroblastoma treated?
Treatments for neuroblastoma include surgery, radiation therapy, and medicines such as chemotherapy, among others. Healthcare teams consider several things when creating a treatment plan, including the child's age, the stage of the cancer, and the risk group [2].
Risk group | Typical treatment approach |
Low risk | Surgery to remove the cancer may be the only treatment needed |
Intermediate risk | A combination of chemotherapy medicines before surgery to improve the chances that the entire cancer can be removed |
High risk | High-dose chemotherapy before surgery and bone marrow transplant, followed by radiation and sometimes immunotherapy |
Surgery
During surgery for neuroblastoma, surgeons use cutting tools to remove the cancer cells. In children with low-risk neuroblastoma, surgery may be the only treatment needed [2].
Whether the cancer can be removed completely depends on its location and size. Cancers that are attached to nearby vital organs may be too risky to remove [2].
In intermediate-risk and high-risk neuroblastoma, treatment usually starts with chemotherapy to shrink the cancer. Surgeons may then try to remove as much of the cancer as possible [2].
Chemotherapy
Chemotherapy treats cancer with strong medicines. Most chemotherapy medicines are given through a vein, though some come in pill form [2].
Children with intermediate-risk neuroblastoma often receive a combination of chemotherapy medicines before surgery. This improves the chances that the entire cancer can be removed [2].
Children with high-risk neuroblastoma often receive high doses of chemotherapy to shrink the cancer and to kill any cancer cells that have spread to other parts of the body. Chemotherapy is often used before surgery and before bone marrow transplant [2].
Bone marrow transplant
A bone marrow transplant (also called a bone marrow stem cell transplant) involves putting healthy bone marrow stem cells into the body. These cells replace cells hurt by chemotherapy and other treatments [2].
A bone marrow transplant might be an option for children with high-risk neuroblastoma. For neuroblastoma, it uses the child's own blood stem cells, which is called an autologous stem cell transplant [2].
Step | What happens |
1. Collection | A procedure filters and collects blood stem cells from the child's blood; cells are stored for later use |
2. High-dose chemotherapy | The child receives high doses of chemotherapy to kill any remaining cancer cells |
3. Transplant | The blood stem cells are put back into the child's body |
4. Recovery | The transplanted cells form new, healthy blood cells |
Radiation therapy and proton therapy
Radiation therapy treats cancer with powerful energy beams, which can come from X-rays, protons, or other sources. Children with high-risk neuroblastoma may receive radiation therapy after chemotherapy and surgery. The radiation can help lower the risk that the cancer will come back [2].
Proton therapy is a type of radiation therapy that uses energy from protons. Proton beams can be more carefully targeted to the cancer cells, so proton therapy may be less likely to hurt healthy tissue near the cancer [2]. This is a newer form of radiation therapy and is not available at all medical centers [2]. Like other forms of radiation, it is typically used after chemotherapy and surgery [2].
Immunotherapy
Immunotherapy is a treatment with medicine that helps the body's immune system kill cancer cells. Cancer cells survive by hiding from the immune system, and immunotherapy helps immune cells find and kill them [2].
Immunotherapy medicines for neuroblastoma target a substance called glycolipid GD2 on the surface of neuroblastoma cells. Medicines that work this way are called anti-GD2 immunotherapies. The medicine is given through a vein, travels through the body, and attaches to the cancer cells, causing immune system cells to attack the cancer [2].
Immunotherapy is sometimes used for high-risk neuroblastoma, typically after other treatments. The medicines are usually combined with other medicines, such as chemotherapy and isotretinoin [2] [3].
Treatment to prevent relapse
Children with high-risk neuroblastoma sometimes receive extra treatment to reduce the risk that the cancer will start growing again. It is generally used after other treatments have controlled the cancer [2].
Eflornithine (Iwilfin) comes as a pill taken twice a day and can be taken for as long as two years [2] [3]. This medicine, also called difluoromethylornithine (DFMO), reduces the risk of relapse, which is when the cancer starts to get worse after a period of getting better [2] [4].
Treatment for recurrence or relapse
Other treatments might be needed if the cancer comes back (recurrence or relapse) or does not respond to the usual treatments (refractory cancer) [2].
Most neuroblastoma relapses and recurrences happen in the first two years after treatment [2]. Rarely, the cancer can come back many years later [2].
Treatment usually starts with medicines that may have helped before, such as chemotherapy and immunotherapy. If these are no longer helping, other options might include:
Option | Description |
Radiopharmaceutical therapy | A medicine containing a radioactive substance travels through the body, attaches to target cells, and releases radiation; one example is I-131 MIBG (MIBG therapy); others, such as lutetium Lu-177 dotatate, are being studied |
Targeted therapy | Medicines that attack specific chemicals in cancer cells; blocking these chemicals can cause cancer cells to die; being studied in clinical trials |
CAR-T cell therapy | Cells are taken from the body and changed so they can fight cancer; most often used for blood cancers such as lymphoma; being studied for neuroblastoma in clinical trials |
Clinical trials are also available for children with neuroblastoma, testing new treatments, interventions, and tests [2].
How can families cope with neuroblastoma?
When a child is diagnosed with cancer, it is common for parents to feel a range of emotions; some describe shock, disbelief, guilt, and anger. At the same time, you may need to make decisions about your child's treatment, which can feel overwhelming [2].
Coping strategy | How it helps |
Gather all the information you need | Learn enough about neuroblastoma to feel comfortable making care decisions; keep a list of questions for the next appointment; ask the team to recommend good information sources |
Organize a support network | Friends and family can offer emotional and practical support, accompany your child to visits, or help with other children and the home |
Take advantage of resources for children with cancer | Clinic social workers can connect families to support groups, summer camps, temporary housing, and other support |
Maintain your usual routines as much as possible | Keep regular nap times and mealtimes, allow time for play, and bring comfort items from home during hospital stays |
Some hospitals have recreation therapists or child life workers who can give more-specific ways to help your child cope through treatment [2].
How should you prepare for an appointment about neuroblastoma?
Make an appointment with your family healthcare professional if your child has any symptoms that worry you. Because appointments can be brief, it helps to be prepared [2].
Preparation step | Why it matters |
Ask about restrictions before the appointment | Some tests require advance preparation |
Write down all symptoms | Helps the team see the full picture |
Note key personal information | Stresses and life changes can relate to symptoms |
List medicines, vitamins, and supplements | Avoids interactions and duplications |
Bring a family member or friend | A second person remembers what you might miss |
Prepare a prioritized question list | Makes the most of limited appointment time |
Questions to ask your child's healthcare team
What may be causing my child's symptoms or condition?
What kinds of tests does my child need?
What is the best course of action, and what are the alternatives?
If my child has other health conditions, how can they best be managed together?
Are there any restrictions my child needs to follow?
Should my child see a specialist?
Where can I find more information?
The healthcare team is likely to ask questions such as when your child began experiencing symptoms, how the symptoms have changed over time, and what seems to improve or worsen them [2].
Conclusion
Neuroblastoma is a childhood cancer of immature nerve cells that mostly affects children age 5 or younger [1]. It most often begins in the adrenal glands but can develop in the chest, belly, neck, or near the spine [1], and symptoms depend heavily on the tumor's location. Diagnosis relies on a combination of urine and blood tests, imaging scans, and biopsy, while treatment is matched to the child's age, cancer stage, and risk group, ranging from surgery alone for low-risk cases to combinations of chemotherapy, bone marrow transplant, radiation, and immunotherapy for high-risk disease [2].
If your child shows symptoms that worry you, such as a belly lump, bone pain, bruise-like circles around the eyes, or unexplained fever, talk to your child's healthcare professional right away. Early evaluation is the first step toward answers and care.
Talk to your child's healthcare professional about any symptoms that worry you, and ask whether a referral to a pediatric specialist is right for your family.
Frequently Asked Questions
1. What is neuroblastoma?
Neuroblastoma is a cancer that starts in immature nerve cells called neuroblasts. It most often begins in the adrenal glands on top of the kidneys and mostly affects children age 5 or younger. It can also develop in the chest, belly, neck, or near the spine.
2. What are the first signs of neuroblastoma?
Early signs depend on the tumor's location. In the belly, they can include belly pain and a painless lump under the skin. In the chest or neck, they may include wheezing, breathing difficulty, or drooping eyelids. Near the spine, they can cause back pain and leg weakness.
3. What age is neuroblastoma most common?
Neuroblastoma mostly affects children age 5 or younger. Risk is highest in children, and most cases occur in this young age group.
4. Is neuroblastoma curable?
Outcomes depend on the child's age, the cancer stage, and the risk group. Some forms of neuroblastoma may go away on their own, and treatments such as surgery, chemotherapy, radiation, bone marrow transplant, and immunotherapy are available. A child's healthcare team determines the best course of action.
5. What causes neuroblastoma?
The exact cause is not clear. It starts when neuroblasts develop changes in their DNA, causing the cells to grow and multiply quickly and to live longer than healthy cells would.
6. Can neuroblastoma be prevented?
There are no known ways to prevent neuroblastoma. Some cases are linked to inherited genetic syndromes, such as Turner syndrome, Costello syndrome, and familial neuroblastoma, but only a small number of neuroblastomas are thought to be inherited.
7. What tests diagnose neuroblastoma?
Diagnosis may include a physical exam, urine and blood tests (looking for chemicals such as catecholamines), ultrasound, CT scan, MRI, and biopsy. MIBG scans, PET scans, and bone marrow aspiration and biopsy check whether the cancer has spread.
8. What is a MIBG scan?
A MIBG scan uses a radioactive tracer placed into a vein that attaches to neuroblastoma cells. A scanner detects the radiation and makes pictures showing where the cells are in the body. It is used to check for spread and to monitor how well treatment is working.
9. What is the treatment for high-risk neuroblastoma?
High-risk neuroblastoma is usually treated with high-dose chemotherapy before surgery and bone marrow transplant, followed by radiation therapy and sometimes immunotherapy. Extra medicine such as eflornithine may be used to lower the risk of relapse.
10. Does neuroblastoma come back after treatment?
Cancer that comes back is called recurrence or relapse. Most neuroblastoma relapses and recurrences happen in the first two years after treatment, though rarely the cancer can return many years later. Further treatments may include chemotherapy, immunotherapy, or radiopharmaceutical therapy.
11. What is immunotherapy for neuroblastoma?
Immunotherapy uses medicines that help the body's immune system find and kill cancer cells. For neuroblastoma, anti-GD2 immunotherapies target glycolipid GD2 on the surface of neuroblastoma cells and are usually combined with other medicines such as chemotherapy and isotretinoin.
Related Reading
Catecholamines Test: How to Prepare and What High Levels Mean explains the stress hormone test used to look for this tumor.
MIBG Scan: How It Finds Neuroendocrine Tumors, Preparation and Results explains the nuclear scan used to find and stage this type of tumor.
References
Mayo Clinic Staff. Neuroblastoma - Symptoms and causes. Mayo Clinic. Sept. 30, 2025. https://www.mayoclinic.org/diseases-conditions/neuroblastoma/symptoms-causes/syc-20351017
Mayo Clinic Staff. Neuroblastoma - Diagnosis and treatment. Mayo Clinic. Sept. 30, 2025. https://www.mayoclinic.org/diseases-conditions/neuroblastoma/diagnosis-treatment/drc-20351022
National Cancer Institute. Neuroblastoma Treatment (PDQ®)–Patient Version. National Cancer Institute. Updated October 23, 2024. https://www.cancer.gov/types/neuroblastoma/patient/neuroblastoma-treatment-pdq
Oesterheld J, Ferguson W, Kraveka JM, et al. Eflornithine as postimmunotherapy maintenance in high-risk neuroblastoma: externally controlled, propensity score-matched survival outcome comparisons. J Clin Oncol. 2024;42(1):90-102. PMID: 37883734. https://doi.org/10.1200/JCO.22.02875
Żebrowska U, Balwierz W, Wechowski J, et al. Survival benefit of myeloablative therapy with autologous stem cell transplantation in high-risk neuroblastoma: a systematic literature review. Target Oncol. 2024;19(2):143-159. PMID: 38401028. https://doi.org/10.1007/s11523-024-01033-4
Flaadt T, Rehm J, Simon T, et al. Long-term outcomes and quality of life of high-risk neuroblastoma patients treated with a multimodal treatment including anti-GD2 immunotherapy: a retrospective cohort study. Cancers (Basel). 2025;17(1):149. PMID: 39796776. https://doi.org/10.3390/cancers17010149
Medical disclaimer
This article is for informational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your child's physician or other qualified health provider with any questions about a medical condition.
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