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Neuroblastoma

Neuroblastoma is a cancerous tumor that comes from nerve tissue of very young children.  It is the most common type of tumor in young children outside of brain tumors.  Around 800 children in the United States are affected by neuroblastoma each year.  Successful treatment of neuroblastoma requires a multidisciplinary team of pediatric oncologists, surgeons, radiologists, neurosurgeons and other specialists.  At Ochsner for Children, our Pediatric Hematologists and Oncologists lead a multidisciplinary team to provide state of the art care to children and young adults diagnosed with neuroblastoma.  We are very active in research and conduct clinical trials designed to increase cure rates, decrease treatment-related side effects and improve care for long-term survivors.

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What is neuroblastoma?

Neuroblastoma is a cancer that occurs almost exclusively in children.  Though the name can be confusing, neuroblastoma is not a brain tumor.  It is the most common solid tumor in children under the age of 2 years, and the most common non-brain solid tumor in children.  Neuroblastoma develops from neuroectoderm cells found in the adrenal gland located just above the kidneys or in parts of the nervous system called sympathetic ganglia which are located in the neck, chest and abdomen. 

What are the symptoms of neuroblastoma?

Neuroblastoma is usually discovered when a parent or doctor finds a tumor or “lump” in a child.  Children with Symptoms of neuroblastoma include:

  • Hard, painless mass in the abdomen or neck
  • Irritability, decreased appetite or fatigue
  • Abdominal pain
  • Weakness in the legs
  • Fever
  • Paleness

How common is neuroblastoma?

Neuroblastoma is the most common tumor in infants less than one year, accounting for around 50% of cancer in this age group.  It almost always occurs before age 5.  There are ~800 new cases of neuroblastoma diagnosed in the US each year, and neuroblastoma accounts for ~10 percent of all childhood cancers.

How is neuroblastoma diagnosed?

Neuroblastoma can be diagnosed either by obtaining a biopsy of the tumor or by obtaining a urine sample, as most neuroblastoma makes substances called catecholamines that  may be found in the urine.  Whenever possible, biopsy is preferred because it allows for more detailed studies of the tumor.  The biopsy sample is examined under a microscope and determined to be either favorable or unfavorable histology.  Important molecular studies will also be performed that will help determine the risk category of the cancer.  Your child will also have blood tests, bone marrow biopsies and scans like CT’s and MRI’s, and a special type of scan used for neuroblastoma called a metiodobenzylguanidine (MIBG) scan.  For this test, a radioactive chemical that binds to neuroblastoma is given IV and images are obtained over time which can show where the neuroblastoma has spread.

How is neuroblastoma staged?

The size, number and location of the tumors is combined with the pathology results from the tumor and bone marrow biopsies and the molecular studies and a stage and risk category is assigned. 

  • Stage 1: The cancer is still in the area where it started. It is on one side of the body (right or left). All visible tumor has been removed completely by surgery (although looking at the tumor’s edges under the microscope after surgery may show some cancer cells). Lymph nodes outside the tumor are free of cancer (although nodes enclosed within the tumor may contain neuroblastoma cells).
  • Stage 2: The cancer is still in the area where it started and on one side of the body.  Nearby lymph nodes outside the tumor contain neuroblastoma cells, but the cancer has not spread to lymph nodes on the other side of the body or elsewhere.
  • Stage 3: The cancer has not spread to distant parts of the body, but one of the following is true:
    • The cancer cannot be removed completely by surgery and it has crossed the midline (defined as the spine) to the other side of the body.
    • The cancer is still in the area where it started and is on one side of the body. It has spread to lymph nodes that are relatively nearby but on the other side of the body.
    • The cancer is in the middle of the body and is growing toward both sides (either directly or by spreading to nearby lymph nodes) and cannot be removed completely by surgery. - need to indent one more level when available in WYSIWYG
  • Stage 4: The cancer has spread to distant sites such as distant lymph nodes, bone, liver, skin, bone marrow, or other organs (but the child does not meet the criteria for stage 4S).
  • Stage 4S (also called “special” neuroblastoma): The child is younger than 1 year old. The cancer is on one side of the body.  The neuroblastoma has spread to the liver, skin, and/or the bone marrow. However, no more than 10% of marrow cells are cancerous, and imaging tests such as an MIBG scan do not show that the cancer has spread to the bones.
  • Recurrent: While not formally part of the staging system, this term is used to describe cancer that has come back (recurred) after it has been treated. The cancer might come back in the area where it first started or in another part of the body.

Your child’s physician, in conjunction with a multidisciplinary team of specialists, will determine a specific course of treatment for your child’s neuroblastoma based on the tumor risk group and other factors. The types of treatment that are used in neuroblastoma may include:

  • Surgery:  To remove the tumor either at diagnosis or after using chemotherapy or radiation to make the tumor smaller.
  • Chemotherapy:  To decrease the size of the tumor, kill tumor that has spread and prevent further tumor spread.
  • Radiation: To kill neuroblastoma cells left behind after chemotherapy and/or surgery. It is also sometimes used for patients who need immediate treatment of a tumor that is pressing on an important nerve or the spinal cord.
  • Stem cell transplant: High-risk patients may require stem cell transplant as part of their treatment.  This involves replacing blood-forming cells in the bone marrow that have been killed by chemo and/or radiation therapy.  Often a child’s own bone marrow is collected and then given back to the patient at a alter date.  This is known as an autologous stem cell transplant
  • Biologic therapy: Newer targeted therapies, known as immune therapies, are often utilized for the treatment of neuroblastoma.  Immune therapies include drugs known as monoclonal antibodies which target neuroblastoma cells, as well as special vitamins such as retinoic acid (isotretinoin) which help the body eliminate neuroblastoma.

These different types of treatments are used alone or in combination depending on the neuroblastoma risk group. In general, recommended treatment for the three neuroblastoma risk groups is:

  • Low-risk neuroblastoma:  Patients with low-risk neuroblastoma usually require only surgery and/or observation. Occasionally, a patient with a low-risk tumor will need chemotherapy (or radiation treatment) to shrink a tumor before it is removed
  • Intermediate-risk neuroblastoma:  Patients with intermediate-risk disease are treated with chemotherapy to shrink the tumor before the tumor can be surgically removed.
  • High-risk neuroblastoma: High-risk neuroblastoma is an aggressive disease that is treated with a multimodality therapy including chemotherapy, surgery, radiation, stem cell transplant and biologic therapy.

What clinical trials does Ochsner Hospital for Children offer for neuroblastoma treatment?

As part of the Children’s Oncology Group, Ochsner Hospital for Children is an active participant in children’s cancer research.  We currently have multiple clinical trials available to treat children and young adults with neuroblastoma.