Neuroblastoma is a cancer that grows from cells in the nervous system. It forms when healthy nervous system cells do not develop as they should. Instead, cancer cells called "neuroblasts" become stuck in an early stage of development. They do not become healthy, normal cells. The cancer cells begin to grow, and this causes a tumor to form.
Patients with neuroblastoma may have only one tumor. Or neuroblastoma cells may spread to other parts of the body. Neuroblastoma is usually found in children younger than 5. It is the most common solid tumor found outside the brain in children.
Neuroblastoma often starts in the abdomen, either in the adrenal gland or in other nerve cells. It can also form in the neck, chest, or pelvis.
Symptoms depend on the location of the tumor (or tumors if the cancer has spread). The symptoms may include a lump, pain, loss of appetite, tiredness, and irritability.
- Some babies with neuroblastoma do not need treatment. The doctor will follow the tumor's progress closely, and sometimes it will go away on its own.
- Some children with neuroblastoma need surgery only.
- But about half of neuroblastoma patients have high-risk disease. They need chemotherapy, surgery, radiation therapy, and immunotherapy.
About 700 children are diagnosed with neuroblastoma in the U.S. each year.
Symptoms of neuroblastoma
Signs and symptoms depend on where the cancer is located. They may include:
- Lump or mass in the neck, chest, or abdomen
- Bulging eyes or dark circles around the eyes
- Stomach pain
- Irritability
- Less appetite
- Constipation
- Feeling tired
- Leg weakness
Other symptoms may include:
- Diarrhea
- Change in eye movement
- High blood pressure
- Headache
- Cough
- Trouble breathing
- Fever
- Bruising
- Horner syndrome
Neuroblastoma has often spread to other parts of the body before it is diagnosed.
What is Horner syndrome?
Some neuroblastoma patients develop Horner syndrome, or nerve damage around the eye. Symptoms may include:
- A drooping eyelid
- Small pupil
- Loss of the ability to sweat on one side of the face
Learn more about Horner syndrome
Risk factors and causes of neuroblastoma
Neuroblastoma is most common in young children. It happens slightly more often in males than females.
A small number of patients (1–2%) have hereditary neuroblastoma. This can be passed down in families. It is often caused by a change in the ALK or PHOX2B genes. But other gene mutations have also been linked to hereditary neuroblastoma.
Learn more about hereditary neuroblastoma
Diagnosis of neuroblastoma
Doctors use several types of tests when diagnosing neuroblastoma. Tests may include:
- A physical exam and health history
- Blood tests to check blood counts, kidney and liver function
- Urine tests to look for vanillylmandelic acid (VMA) and homovanillic acid (HVA). VMA and HVA are produced by the breakdown of catecholamines, which are hormones produced by neuroblasts. Children with neuroblastoma often have high levels of VMA and HVA. These substances may help monitor response to treatment.
- A neurological exam to measure brain and nerve function including memory, vision, hearing, muscle strength, balance, coordination, and reflexes
- Imaging tests to learn about the size and location of the tumor(s)
Doctors will study the tumor for features that are important to diagnose and treat neuroblastoma. Some tumors are aggressive and require intense therapy. Doctors can predict how a tumor might respond to treatment by how the cells look and whether there are certain gene changes in the tumor.
Neuroblastoma is staged using the International Neuroblastoma Risk Group Staging System (INRGSS). Staging is based on these factors:
- Tumor location
- Effect on nearby organs
- Spread of disease
Neuroblastoma risk groups
Doctors use risk groups to classify neuroblastoma and plan treatments. There are 3 risk groups:
- Low risk
- Intermediate risk
- High risk
High-risk neuroblastoma means that the cancer is hard to remove and more likely to come back. Children with high-risk neuroblastoma need intense therapy.
Risk groups are based on factors that include:
- Patient age
- Stage of disease
- Tumor characteristics
Treatment of neuroblastoma
Treatment depends on the assigned risk group. Treatment options may include:
Very young, low-risk patients may be followed with observation instead of active treatment. Sometimes neuroblastoma goes away on its own (regression). But this is rare. Patients are watched closely for tumor progression.
Patients with neuroblastoma may be offered treatment as part of a clinical trial. Neuroblastoma treatments are planned based on risk groups:
Prognosis for neuroblastoma
Neuroblastoma survival depends on several factors:
- Child’s age at diagnosis
- Risk group
- The genetics of the tumor
- Whether the cancer has spread
- How the tumor responds to treatment
- Whether the cancer is recurrent (has come back)
Your child's doctor is the best source of information on your child's specific case.
Support for neuroblastoma patients
Monitoring for relapse
Patients need follow-up care to watch for recurrence after treatment ends. The medical team will suggest specific tests and their schedule.
In patients who do not have high-risk disease, the chance of relapse is 5–15%. In high-risk patients, the risk of relapse is about 50%. Relapse is most common in the first 2 years after treatment. Relapse is rare when there is no sign of cancer 5 years after completing treatment.
Health after cancer
Survivors treated with chemotherapy or radiation should be monitored for long-term and late effects of therapy. Problems due to treatment could include hearing loss, heart problems, thyroid problems, decreased growth, decreased bone density, and kidney damage.
About 25% of survivors have serious chronic health conditions 25 years after diagnosis, according to the Childhood Cancer Survivor Study.
Regular medical checkups by a primary health care provider are important to watch for health problems that can develop years after therapy.
Your child's care team should give you a survivorship care plan after treatment has ended. This report will include needed tests and tips for a healthful lifestyle.
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Reviewed: November 2023
