Neuroblastoma

Neuroblastoma is a cancer that starts in neuroblasts, which are immature nerve cells. It most often begins in the nerve tissue of an adrenal gland. There is one adrenal gland on top of each kidney. It can also start in nerve tissue along the spine, anywhere from the neck to the pelvis.

It is the most common solid tumor outside the brain in children, and the most common cancer in babies younger than 1 year. About 9 in 10 children are diagnosed before age 5. It rarely occurs in adults.

Neuroblastoma behaves very differently from one child to the next. In some babies, the tumor shrinks and goes away without treatment. In other children it spreads quickly. Care teams sort each child into a low-, intermediate- or high-risk group, and that group shapes the whole plan. About half of children with a new diagnosis are in the low- or intermediate-risk groups.

Neuroblastoma starts when changes in genes let immature nerve cells keep growing and dividing instead of maturing. In most children these changes happen by chance in the tumor cells only. They are not passed down from a parent. Studies have not clearly linked neuroblastoma to anything in the environment.

About 1 to 2 in every 100 children with neuroblastoma have a family history of it. In about 3 of every 4 of these families, the cause is an inherited change in a gene called ALK. Rarely, an inherited change in a gene called PHOX2B is the cause. Some other inherited conditions, such as Costello syndrome and Noonan syndrome, also raise the risk. Some children are born with certain ALK or PHOX2B changes. For them, a U.S. cancer research group (the American Association for Cancer Research) recommends regular ultrasound scans and urine tests until age 10.

Some changes found only in the tumor affect how serious it is rather than causing it. The best known is MYCN amplification, meaning extra copies of the MYCN gene. It is found in about 1 in 4 neuroblastomas and is linked with faster growth and spread.

Many signs come from the tumor pressing on nearby tissue or from cancer spreading to bone. A lump or swelling in the belly is the most common sign. Children may also have bone pain, dark circles or bruising around the eyes, bulging eyes, fever, tiredness or weight loss. Babies may have painless bluish bumps under the skin that look like blueberries.

By the time it is found, neuroblastoma has often spread. It most often spreads to lymph nodes, bones, bone marrow, liver and skin. A tumor near the spine can press on the spinal cord and cause weakness or paralysis, which needs treatment right away. A tumor in the neck can cause Horner syndrome: a droopy eyelid, a small pupil and less sweating on one side of the face.

Some tumors release hormones that can cause high blood pressure, a fast heartbeat, sweating or severe watery diarrhea. Rarely, the immune system attacks nerve tissue and causes opsoclonus-myoclonus syndrome, with quick, jerky eye and muscle movements. Children with this syndrome often have tumors with favorable features, but the nerve symptoms can be slow to improve.

A team that specializes in childhood cancer usually plans care. It is led by a pediatric oncologist and often includes surgeons, radiation doctors, nurses, social workers, child life specialists and psychologists. For high-risk neuroblastoma, treatment is long and intense. It includes chemotherapy, surgery, stem cell collection, the transplants, radiation and immunotherapy. Eflornithine can continue for up to 2 years after immunotherapy.

Immunotherapy has its own hard days. The FDA label for dinutuximab warns that it causes severe nerve pain. So children get strong pain medicine through a vein before, during and after each infusion. It can also cause serious, sometimes life-threatening reactions during the infusion.

After treatment, children have regular checkups with urine tests and MIBG scans. High-risk neuroblastoma can come back, sometimes more than 5 years later. Late effects can include hearing loss and changes in how teeth and bones grow. Some children later have high blood pressure or high cholesterol, or changes in mood, learning or memory. Second cancers can also occur. Some late effects can be treated or controlled.

No donor is involved in the usual neuroblastoma transplant. It is autologous, which means it uses the child’s own stem cells, collected during early treatment, frozen, and given back after high-dose chemotherapy. The returned cells rebuild the bone marrow after the high doses.

Donor (allogeneic) transplants have been studied, but they are not standard care. A review of 147 donor transplants for neuroblastoma, reported to the CIBMTR, an international transplant registry, found no advantage over own-cell transplants. European transplant experts (EBMT, 2025) list donor transplant for neuroblastoma as developmental, meaning more research is needed.

So a family facing neuroblastoma does not need to search for a matched donor. Joining a donor registry is a way to help other patients, such as children with leukemia, rather than a need for most children with neuroblastoma.