Neuroblastoma

July 23, 2025

This page was reviewed under our medical and editorial policy by Janet Yoon, M.D., clinical professor, medical director of the Pediatric Musculoskeletal Tumor Program, Department of Pediatrics, City of Hope^® Cancer Center Duarte.

Neuroblastoma is a type of cancer that develops in cells that form part of a child’s developing nervous system. Although it accounts for only 750 or so new cases each year, neuroblastoma is the most common cancer type diagnosed in infants under the age of 1. Almost all cases of neuroblastoma are diagnosed in children 5 and younger.

What Is Neuroblastoma?

Neuroblastoma occurs in a type of cell called a neuroblast, which is an early form of nerve cell. Neuroblasts play a critical role in the development of a child’s nervous system both before and after birth.

When a child develops neuroblastoma, it occurs in a part of the nervous system known as the sympathetic nervous system, which helps control some of the body’s autonomic functions. The sympathetic nervous system consists of:

• Nerve fibers on either side of the spinal cord
• Clusters of nerve cells located on these nerve fibers
• Nerve-like cells within the body’s adrenal glands, which are glands that help manage things like heart rate, blood pressure and blood sugar

Where Is Neuroblastoma Located?

Although neuroblastoma begins in the sympathetic nervous system, it may affect different parts of the body. Most often, neuroblastoma is found in tissue within the abdomen or in the adrenal glands. At a more advanced stage, neuroblastoma may spread to other areas of the body via the bloodstream. It most commonly spreads to:

• The bones or bone marrow
• The liver
• The skin

Neuroblastoma Symptoms

Because it primarily affects young children, it’s important for parents and loved ones to recognize the signs of neuroblastoma. Usually caused by a tumor pressing against other parts of a child’s body, some of the more common symptoms of neuroblastoma include:

• A swelling in the stomach area
• Lumps near the neck or in the torso
• Dark circles under the eyes or bulging eyes
• Blue-colored, painless lumps, especially in infants
• Trouble breathing, especially in infants
• Weakness or an inability to move certain parts of the body, especially in infants

What Causes Neuroblastoma?

In most cases, doctors do not know the exact cause of neuroblastoma. However, researchers are continuing to study how and why neuroblastoma develops.

Is Neuroblastoma Genetic?

Like all cancers, neuroblastoma is caused by genetic changes to the body’s cells. Most neuroblastomas are not hereditary, meaning they aren’t caused by gene mutations passed on from one or both parents to a child. However, there are some very rare cases in which a parent passes on a mutation in the ALK or PHOX2B genes, increasing the risk that a child may develop the condition.

Risk Factors

Some of the risk factors for neuroblastoma include:

• Being a child under the age of 10
• Certain rare genetic mutations passed on from a parent to a child, but this accounts for less than 2% of all neuroblastoma cases
• Having certain other forms of birth defect

Diagnosis and Testing

Neuroblastoma is usually diagnosed by doctors who specialize in ​childhood cancer. They may recommend a variety of tests to screen for and accurately diagnose neuroblastoma, including:

• Physical and neurological examinations to look for symptoms and signs of neuroblastoma
• A biopsy to remove a tissue sample to look for signs of cancer
• Bone marrow aspiration and biopsy, which can remove a sample of tissue from the body’s bone marrow
• Urine or blood tests
• Imaging tests, such as X-ray, magnetic resonance imaging, ultrasound or computed tomography, which may be able to detect tumors inside the body

Neuroblastoma Staging

When diagnosing neuroblastoma, cancer doctors “stage” the disease, which refers to the process of using tests to determine how much cancer is in someone’s body and how far it has spread. The first step in staging neuroblastoma is to assign it one of the following stages, based on a system called the International Neuroblastoma Risk Group Staging System.

L1: Neuroblastoma has not spread from its original location or affected a major part of the body.

L2: Neuroblastoma has not spread far, but has begun to impact another part of the body.

M: Neuroblastoma has spread further (metastasized) to a more distant part of the body.

MS: Neuroblastoma in a child under 18 months of age has spread to the bones, liver or skin.

Neuroblastoma Risk Groups

Doctors may also use a system that designates patients as part of certain risk groups when staging neuroblastoma. Based on factors including how extensive a child’s cancer is, how old the child is and the results of any diagnostic tests, they may be classified as:

• Low risk
• Intermediate risk
• High risk

In some cases, the first of these groups is divided into “very low risk” and “low risk” patients, depending on which staging system is being used. In general, lower risk cases of neuroblastoma may have a greater chance of being cured or treated effectively with less aggressive treatments and therapies, while higher risk cases may require more assertive therapies.

Neuroblastoma Treatment

A range of treatment options are available for neuroblastoma. Doctors may recommend one type of treatment or a combination, depending on a variety of factors, including a child’s age, the stage of the cancer and other health issues.

Surgery

Surgery is a common treatment option for neuroblastoma, especially for smaller tumors. In some cases, surgeons may be able to remove all of the cancer from a patient’s body. Surgery is often combined with other treatment options, such as radiation therapy or chemotherapy. These other therapies may be used to shrink a tumor before surgery or to kill any remaining cancer cells after surgery.

Chemotherapy

​Chemotherapy is another common treatment for neuroblastoma. Chemotherapy refers to a range of medications that are usually given intravenously (into a vein) and are designed to kill cancer cells in the body. It may be used alongside surgery, radiation therapy or other treatments for neuroblastoma.

High-Dose Chemotherapy and Stem Cell Transplantation

For high-risk cases of neuroblastoma, doctors may recommend an intensive course of chemotherapy followed by a ​​pediatric stem cell transplant, which replaces any cells in the body that get damaged by the chemotherapy. In most cases, the stem cells are collected from patients after their second chemotherapy cycle. These cells are then used during the stem cell transplant to replenish healthy cells.

Radiation Therapy

Radiation therapy, which uses strong radiation beams to destroy cancer cells, is a less common treatment for neuroblastoma. This is primarily because its side effects may be more severe for children. However, it is sometimes used for high-risk patients.

Retinoid Therapy

Retinoid therapy is the use of a type of retinoid (a chemical produced in the body) called isotretinoin to help neuroblasts mature into healthy cells rather than cancer cells. It is most frequently recommended for patients who have undergone high-dose chemotherapy and stem cell transplant and it can help prevent neuroblastoma from coming back.

Targeted Therapy

Targeted therapy is a term that refers to certain kinds of medications that are designed to target cancer cells without damaging healthy cells. Doctors may recommend targeted therapy for neuroblastoma, depending on a patient’s unique diagnosis.

Iodine 131-MIBG Therapy

Iodine 131-MIBG therapy is a treatment that uses a form of radioactive iodine. This iodine is injected into the body, where it targets and kills neuroblastoma cells.

Who Treats Neuroblastoma?

Children with neuroblastoma are cared for by a multidisciplinary team of pediatric cancer experts and clinical providers, including:

• Pediatric oncologists with expertise in treating types of childhood cancers.
• Surgical oncologists, who are experts in complex surgical techniques for removing tumors from the body.
• Neurologists, who specialize in treating diseases of the nervous system.
• Radiation oncologists, who have expertise in innovative radiation therapy techniques.
• Supportive care staff specializing in providing physical and emotional support to patients and their loved ones.
• Trained oncology nurses, who care for patients during their hospital treatments and stays.

Neuroblastoma Survival Rate

The event-free five-year survival rates for neuroblastoma depend mostly on whether a patient is considered low, intermediate or high risk when the child is diagnosed with the condition, as follows.

For low-risk neuroblastoma patients: The five-year event-free survival rate is greater than 85% to 95%.

For intermediate-risk neuroblastoma patients: The five-year event-free survival rate is 50% to 75%.

For high-risk neuroblastoma patients: The five-year event-free survival rate is below 50%.

Survival rates depend on a wide variety of factors. It’s important for patients and their families to talk to the care team to get a neuroblastoma prognosis tailored to the patient’s specific stage, risk category, overall health and other factors.