ConditionCancer
Neuroblastoma
Neuroblastoma is a cancer that develops from immature nerve cells (neuroblasts) of the sympathetic nervous system, most often in an adrenal gland above the kidney, but also along the spine in the belly, chest, neck or pelvis. It mainly affects babies and young children, with most diagnosed before age 5. Signs include a swollen belly or lump, bone pain, bruising around the eyes and fever. Outlook varies widely: low-risk tumors may shrink on their own, while high-risk disease needs intensive treatment.
Also called: Neuroblastoma tumor, neuroblastic tumor, childhood adrenal cancer
At a glance
- What it is
- A cancer of immature nerve cells, most often starting in the adrenal gland.
- Who gets it
- Mostly children under 5; the most common cancer in infants.
- Signs
- Belly swelling or lump, bone pain, limping, bruising around the eyes, fever, weight loss.
- Diagnosis
- Urine catecholamine tests, imaging, MIBG scan, biopsy and bone marrow tests.
- Risk groups
- Low, intermediate and high, based on age, stage and tumor biology such as MYCN.
- Treatment
- Observation, surgery, chemotherapy, radiation, stem cell transplant and immunotherapy.
When to get help
Call 911 or go to the ER
- A child with sudden leg weakness, inability to walk, or loss of bladder or bowel control
- Difficulty breathing or swallowing, or swelling of the face and neck
- A child who is very pale, floppy, bleeding or hard to wake
See a doctor within a day or two
- A swollen, firm belly or a lump in a young child
- Bruising around the eyes ("raccoon eyes") or eyes that bulge, without an injury
- Bone pain, limping or refusal to walk with fever or pallor
- Rapid, jerky eye movements and jerking muscles with unsteadiness (opsoclonus-myoclonus)
Book a routine appointment
- Ongoing unexplained fever, poor appetite or weight loss in a young child
- A droopy eyelid with a small pupil on one side (Horner syndrome)
- Persistent diarrhea or high blood pressure without a clear cause in a young child
What neuroblastoma is
Before birth, nerve cells called neuroblasts develop into the sympathetic nervous system, which controls automatic functions such as heart rate and blood pressure, and the adrenal glands. Usually neuroblasts mature into nerve or adrenal cells. In neuroblastoma, some stay immature and keep dividing.
About 650 to 800 children are diagnosed with neuroblastoma in the US each year. The average age at diagnosis is about 1 to 2 years. It is very rare after age 10.
Where it starts
- adrenal gland: about half of cases (see adrenal tumors)
- abdomen, along the spine
- chest: often found on an X-ray for another reason
- neck
- pelvis
Neuroblastoma often has spread by the time it is diagnosed, most commonly to bone, bone marrow, lymph nodes, liver and skin.
Symptoms
Symptoms depend on where the tumor is and whether it has spread:
| Location or spread | Possible signs |
|---|---|
| Abdomen | Swollen belly, a firm lump, belly pain, constipation, poor appetite |
| Chest | Cough, breathing difficulty, swallowing problems |
| Neck | Lump, droopy eyelid and small pupil (Horner syndrome) |
| Spine | Weakness, inability to walk, bladder or bowel problems (spinal cord compression) |
| Bone and bone marrow | Bone pain, limping, irritability, pale skin, bruising, infections |
| Around the eyes | Bruising ("raccoon eyes"), bulging eyes |
| Skin (in infants) | Bluish-purple lumps ("blueberry muffin" spots) |
| Liver (in infants) | Rapidly swelling belly |
General symptoms include fever, tiredness, weight loss and irritability. Rarely, the tumor releases substances that cause severe watery diarrhea or high blood pressure, or triggers an immune syndrome called opsoclonus-myoclonus (dancing eyes and jerky movements).
Causes and risk factors
The cause of most neuroblastomas is unknown. They are not caused by anything parents did. About 1 to 2 percent are inherited, usually through changes in the ALK or PHOX2B genes, and these children may have tumors at a younger age or in more than one place.
Diagnosis
- Urine tests: homovanillic acid (HVA) and vanillylmandelic acid (VMA), breakdown products of catecholamines, are raised in most cases
- Imaging: ultrasound, CT or MRI of the tumor
- MIBG scan: a special nuclear scan that detects neuroblastoma cells anywhere in the body
- Biopsy: confirms the diagnosis and tests tumor biology, including MYCN gene amplification, chromosome changes and DNA content
- Bone marrow aspirate and biopsy: checks for spread
Stages and risk groups
Doctors use the International Neuroblastoma Risk Group (INRG) staging system:
- L1: localized tumor without image-defined risk factors
- L2: localized tumor with risk factors that make surgery harder
- M: distant spread
- MS: spread limited to skin, liver and bone marrow in children under 18 months, which often regresses
Children are then placed into low, intermediate or high-risk groups based on stage, age, MYCN status, tumor histology and other genetic features.
Treatment
| Risk group | Typical treatment |
|---|---|
| Low risk | Observation (some tumors shrink on their own, especially in infants) or surgery alone |
| Intermediate risk | Surgery and moderate chemotherapy |
| High risk | Intensive chemotherapy, surgery, high-dose chemotherapy with stem cell transplant, radiation, immunotherapy (anti-GD2 antibody dinutuximab) and maintenance therapy |
Newer approaches include MIBG therapy, ALK inhibitors for tumors with ALK changes, and DFMO (eflornithine) to reduce relapse risk after high-risk treatment. Many children are treated within clinical trials.
Outlook
- Low-risk: 5-year survival is above 95 percent
- Intermediate-risk: about 90 to 95 percent
- High-risk: around 50 to 60 percent with modern treatment
Survivors, especially after high-risk treatment, need long-term follow-up for effects on hearing, growth, heart, kidneys, fertility and learning, and for second cancers.
Frequently asked questions
What are the first signs of neuroblastoma?
Common first signs include a swollen belly or lump, belly pain, bone pain or limping, bruising around the eyes, fever, tiredness and weight loss. Many signs are vague and similar to common childhood illnesses, so persistent symptoms should be checked.
What is the survival rate for neuroblastoma?
It depends on the risk group. Children with low-risk or intermediate-risk neuroblastoma have 5-year survival rates above 90 percent. High-risk neuroblastoma has a 5-year survival of around 50 to 60 percent with modern treatment.
Can neuroblastoma go away on its own?
Yes, in some infants. Certain low-risk tumors, especially stage MS in babies under 18 months, can shrink and disappear without treatment, or mature into benign tumors. Doctors monitor these closely.
Is neuroblastoma hereditary?
Rarely. About 1 to 2 percent of cases are inherited, usually through ALK or PHOX2B gene changes. Families with more than one affected child or very young children with multiple tumors may be offered genetic testing.
Sources
This guide was written from current patient and clinical guidance, including:
- National Cancer Institute: Neuroblastoma Treatment (PDQ) Patient Version
- American Cancer Society: Neuroblastoma
- Children's Oncology Group: Neuroblastoma
- St. Jude Children's Research Hospital: Neuroblastoma
- International Neuroblastoma Risk Group (INRG) classification system