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    Neuroblastoma is a cancer that arises in immature nerve cells and affects mostly infants and children. Learn about neuroblastoma and find information on how we support and care for children and teens with neuroblastoma before, during, and after treatment.

The Solid Tumor Center at Dana-Farber/Boston Children's Cancer and Blood Disorders Center treats children and teens with a variety of solid malignancies, including bone and soft tissue tumors, liver and kidney tumors, neuroblastomas, retinoblastomas and rare tumors. Our doctors provide unparalleled expertise in the diagnosis, treatment and management of these diseases.

Your child's care team will include pediatric oncologists, radiation oncologists, surgeons, pathologists, radiologists, and nurses with expertise in treating your child's specific type of cancer.

Our physicians are focused on family-centered care: From your first visit, you'll work with a team of professionals who are committed to supporting your family's needs. We consider you and your child integral parts of the care team. Our specialists will collaborate with you to customize a treatment plan that takes the needs of your child and your family into account.

As well as providing access to a range of innovative clinical trials through Dana-Farber/Boston Children's, we are New England's Phase I referral center for the Children's Oncology Group, which means we're able to offer clinical trials unavailable at other regional centers.

Your child will have access to long-term treatment and childhood cancer survivor support through Dana-Farber's David B. Perini, Jr. Quality of Life Clinic.

From diagnosis through treatment and survivorship, our team will be able to answer all of your questions about your child's care.

Find out more about our Solid Tumor Center, including the diseases we treat and our specialized programs for bone and soft tissue tumors, liver tumors, neuroblastoma, rare tumors, and retinoblastoma.

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What is neuroblastoma (NBL)?

Neuroblastoma is a cancerous tumor that begins in nerve tissue of infants and very young children. The abnormal cells are often found in the nerve tissue that is present in the unborn baby and later develop into a detectable tumor. Neuroblastoma is rare in children older than 10 years of age; however, it does occur occasionally in adults.

As you read further below, you will find general information about neuroblastoma. If you would like to view summary information about cancer first, see the cancer overview.

In neuroblastoma, the tumor usually begins in the tissues of the adrenal gland found in the abdomen, but may also begin in nerve tissue in the neck, chest, and/or pelvis. The adrenal glands are positioned on top of the kidneys. These glands secrete hormones and other important substances that are required for normal functions in the body, such as the nervous system.

Most children affected by neuroblastoma have been diagnosed before age 5. It is often present at birth, but not detected until the tumor begins to grow and compress the surrounding organs. In rare cases, neuroblastoma can be detected before birth by a fetal ultrasound.

In the United States, approximately 500 children are diagnosed with neuroblastoma each year. It is the most common tumor found in children younger than 1 year of age. For unknown reasons, it occurs slightly more often in males than in females.

Neuroblastoma cancer cells can spread (metastasize) quickly to other areas of the body (i.e., lymph nodes, liver, lungs, bones, central nervous system, and bone marrow). Approximately 70 percent of all children diagnosed with neuroblastoma will have some metastatic disease.

What is ganglioneuroblastoma?

About one-third of neuroblastomas may begin in the adrenal glands, but many others begin in the ganglia, clusters of nerve cells found throughout the body that are part of the nervous system. There is a benign tumor called ganglioneuroma, which is formed by mature ganglion and nerve cells that do not grow out-of-control.

Ganglioneuroblastoma, on the other hand, is a cancerous tumor that contains immature neuroblasts (nerve cells found in the embryo) which can grow and spread abnormally, as well as areas of tissue that have matured and are similar to ganglioneuroma. Treatment for ganglioneuroblastoma is similar to that of neuroblastoma. 

What causes neuroblastoma?

Most neuroblastoma cells have genetic abnormalities involving chromosome #1, where a deletion or rearrangement is found on the short arm of this chromosome. What causes this genetic alteration is generally unknown. This chromosome abnormality, in turn, causes a disruption in an oncogene called MYCN, even though this gene is not located directly on chromosome #1. The amplification of MYCN causes uncontrolled cell growth. A variety of other chromosome abnormalities may also be present in neuroblastoma.

It is estimated that as many as 20 percent of neuroblastoma cases result from an inherited mutation, followed by a second mutation occurring after birth, which together initiate uncontrolled cell growth. The remainder of the cases occurs from two acquired mutations after birth. Because the tumor occurs very early in childhood, it is doubtful that any environmental exposures the child has incurred could be linked to the development of the tumor.

Neuroblastoma is more common in children born with fetal hydantoin syndrome, neurofibromatosis, and Beckwith-Wiedemann syndrome. The exact relationship between these conditions and the disease are not known.

The chance for neuroblastoma to be present in a future sibling of the patient is about 1 percent. If more than one child has neuroblastoma, the chance for reoccurrence increases. Research is being conducted to determine if maternal exposure to any toxic substances, environmental pollution, or radiation during pregnancy could have any link to the child developing neuroblastoma.

What are the symptoms of neuroblastoma?

The following are the most common symptoms of neuroblastoma. However, each child may experience symptoms differently. The symptoms of neuroblastoma vary greatly depending on size, location, and spread of the tumor. Symptoms may include:

  • an abdominal mass, either felt during an examination or seen as a swollen abdomen
  • tumors in the face or head that cause swelling and bruising of the area around the eyes and uncontrolled eye movement
  • compression of the kidney or bladder by the tumor that causes changes in urination
  • pain, limping, paralysis, or weakness caused by bone or bone marrow involvement, or spinal cord tumor growth
  • cough or shortness of breath caused by a tumor in the chest diarrhea caused by a substance produced by the tumor (vasoactive intestinal peptide, or VIP)
  • fever
  • high blood pressure and increased heart rate, depending on location of the tumor and the organs the tumor compresses
  • weight loss or poor appetite

The symptoms of neuroblastoma may resemble other conditions or medical problems. Always consult your child's physician for a diagnosis.

How is neuroblastoma diagnosed?

In addition to a complete medical and physical examination, diagnostic procedures for neuroblastoma may include the following:

  • blood tests - including a complete blood count

  • 7 multiple imaging studies - to evaluate primary tumor and determine extent/location of any metastases, including:
    • computerized tomography scan (also called a CT or CAT scan.) - a diagnostic imaging procedure that uses a combination of x-rays and computer technology to produce cross-sectional images (often called slices), both horizontally and vertically, of the body. A CT scan shows detailed images of any part of the body, including the bones, muscles, fat, and
    • magnetic resonance imaging (MRI) - a diagnostic procedure that uses a combination of large magnets, radiofrequencies, and a computer to produce detailed images of organs and structures within the body
    • x-ray - a diagnostic test that uses invisible electromagnetic energy beams to produce images of internal tissues, bones, and organs onto film
    • ultrasound (also called sonography) - a diagnostic imaging technique that uses high-frequency sound waves and a computer to create images of blood vessels, tissues, and organs. Ultrasounds are used to view internal organs as they function, and to assess blood flow through various vessels.
    • bone scans - pictures or x-rays taken of the bone after a dye has been injected that is absorbed by bone tissue. These are used to detect tumors and bone abnormalities. Other nuclear medicine scans may use an agent, MIBG, directly taken up by neuroblastoma.
  • bone marrow biopsy and/or aspiration is a procedure that involves taking a small amount of bone marrow fluid and tissue, usually from part of the hip bones, to further examine the number, size, and maturity of blood cells and/or abnormal cells

  • urine test - to measure for chemical products, VMA or HVA, made by the tumor
  • spinal tap/lumbar puncture - a procedure that involves inserting a special needle through the lower back into the spinal canal, the area around the spinal cord. The pressure in the spinal canal and brain can then be measured. A small amount of cerebral spinal fluid (CSF) can be removed and sent for testing to determine if there is an infection or other problems. CSF is the fluid that bathes your child's brain and spinal cord.
  • 7 biopsy - a sample taken of the primary tumor and/or metastatic lesions

Diagnosing neuroblastoma also involves staging and classifying the disease, which determines treatment options and prognosis. Staging is the process of determining whether cancer has spread and, if so, how far. There are various staging systems that can be used for neuroblastoma. Always consult your child's physician for information on staging. One method of staging neuroblastoma is the following:

  • stage 1 - involves a tumor that does not cross the midline of the body, appears to be completely removable, and has not spread to other areas of the body. The lymph nodes on the same side of the body as the tumor do not have cancer cells present.
  • stage 2A - involves a tumor that does not cross the midline of the body, but also does not appear to be completely removable. This stage of tumor has not spread to other areas of the body, and lymph nodes on the same side as the tumor do not have tumor cells present.
  • stage 2B - involves a tumor that may or may not be completely removable, has not spread to other areas of the body, but lymph nodes on the same side of the tumor have tumor cells present. Lymph nodes on the opposite side of the tumor must be negative for tumor cells in this stage of disease.

  • stage 3 - involves a tumor that crosses the midline of the body, is not completely removable, and lymph nodes are positive for tumor cells. This stage also includes a tumor that does not cross the midline, but the lymph nodes on the opposite side also contain tumor cells.
  • stage 4 - involves a tumor that has metastasized (spread) to distant lymph nodes, bone marrow, liver, skin, and/or other organs (except as defined in stage 4S).
  • stage 4S - involves a tumor in a child less than 1 year old that has metastasized (spread) to liver, skin, and/or bone marrow (includes minimal bone marrow involvement; more extensive bone marrow involvement should be classified as stage 4).
What are the treatments for neuroblastoma?

Specific treatment for neuroblastoma will be determined by your child's physician based on:

  • your child's age, overall health, and medical history
  • extent of the disease, which will include the expression of the oncogene N-myc
  • your child's tolerance for specific medications, procedures, or therapies
  • how your child's doctor expects the disease may progress
  • your opinion or preference

Treatment of this tumor requires extremely close coordination among the pediatric oncologists, radiotherapists and surgeons. This tumor, more than any other, has a wide spectrum of behavior. Tumors arising in children under one year of age often have very slow progression and are readily treated while those occurring in children over one year of age are often more aggressive and more difficult to cure. These tumors may be quite extensive at the time of presentation and those in the abdomen often surround the blood vessels to the kidney as well as the blood vessels to the liver, spleen and small intestine.

Recent developments in the treatment of neuroblastoma have included identification of several genetic findings in the tumors that can predict either a favorable or unfavorable prognosis. These tests have provided valuable information indicating which tumors need to be treated aggressively and which tumors require little treatment beyond surgical removal.

In small tumors, surgery is generally performed initially to remove the tumor and provide information regarding the extension or spread of the tumor. This information determines the stage of neuroblastoma and is used in conjunction with genetic information about the tumor to define which additional therapy will be used. Small tumors in young children are often cured with surgery alone.

Large tumors occurring in children over one year of age often require an initial biopsy followed by chemotherapy and, sometimes, radiation therapy. Initial treatment with chemotherapy provides several advantages. First, it often produces a dramatic decrease in the size of the tumor. Second, it generally decreases the friability or consistency of the tumor that can result in rupture of the tumor or significant bleeding during the original operative resection. Third, separation of the tumor from the critical blood vessels it surrounds can be accomplished best in tumors treated with chemotherapy.

Children who have spread of the tumor to their bone marrow or bones, who are over one year of age, and have genetic findings suggesting that they have an aggressive tumor, may receive stem cell transplants after initial chemotherapy and surgical removal of the tumor to enhance their chances of cure. In rare instances a lymphoma may weaken one or more bones. In addition to radiation, a special plate or rod may be necessary to prevent or treat a fracture of the involved bone.

Radiation therapy is used in areas where the tumor has been incompletely removed, in tumor with expected aggressive behavior, and in conjunction with stem cell transplantation.

A summary of these treatments follows: 

  • surgery - biopsy and surgical removal of tumors and cancerous lymph nodes, performed by a surgeon
  • chemotherapy - a drug treatment that works by interfering with the cancer cell's ability to grow or reproduce. Different groups of drugs work in different ways to fight cancer cells and shrink tumors. Chemotherapy may be used alone for some types of cancer or in conjunction with other therapy such as radiation or surgery. Often, a combination of chemotherapy drugs is used to fight a specific cancer. Certain chemotherapy drugs may be given in a specific order depending on the type of cancer it is being used to treat. While chemotherapy can be quite effective in treating certain cancers, the agents do not differentiate normal healthy cells from cancer cells. Because of this, there can be many adverse side effects during treatment. Being able to anticipate these side effects can help the care team, parents, and child prepare, and, in some cases, prevent these symptoms from occurring, if possible. Chemotherapy is systemic treatment, meaning it is introduced to the bloodstream and travels throughout the body to kill cancer cells. Chemotherapy can be given:
    • as a pill to swallow
    • as an injection into the muscle or fat tissue
    • intravenously (directly to the bloodstream; also called IV)
    • intrathecally - chemotherapy given directly into the spinal column with a needle
  • radiation therapy - using high-energy rays (radiation) from a specialized machine to damage or kill cancer cells and shrink tumors
  • stem cell transplant - a treatment involving stem cells, a specific type of cell from which all blood cells develop. Stem cells develop into red blood cells to carry oxygen, white blood cells to fight disease and infection, and platelets to aid in blood clotting. Transplantation of normal stem cells from another person is used to help restore normal blood production in patients whose own ability to make any or all of these blood cells has been compromised by cancer, intensive cancer treatment, or other types of damage or abnormality. The use of cells from another individual is called allogeneic transplantation. Stem cells collected form patients themselves prior to intensive treatment can also be used to supplement the recovery of the patient's own cells after particularly aggressive course of chemotherapy or radiation therapy. The use of a patient's own cells is referred to as autologous transplantation. Stem cell transplantation and the treatment needed to manage its effects are complex. Your physician will give you more detailed information on what to expect.
  • supportive care - any type of treatment to prevent and treat infections, side effects of treatments, and complications, and to keep your child comfortable during treatment
  • continuous follow-up care - a schedule of follow-up care determined by your child's physician and other members of your care team to monitor ongoing response to treatment and possible late effects of treatment

Treatment options will vary greatly, depending on your child's individual situation. Your child's physician and other members of your care team will discuss these options with you in-depth.

What is the long-term outlook for patients with neuroblastoma?

Prognosis greatly depends on:

  • the extent of the disease
  • the tumor's characteristics when examined under a microscope, and for N-myc oncogene expression
  • a presence or absence of metastasis
  • the tumor's response to therapy
  • the age and overall health of your child
  • your child's tolerance of specific medications, procedures, or therapies
  • new developments in treatment
  • the size and location of the tumor

As with any cancer, prognosis and long-term survival can vary greatly from individual to individual. Prompt medical attention and aggressive therapy are important for the best prognosis. Continuous follow-up care is essential for a child diagnosed with neuroblastoma. Side effects of radiation and chemotherapy, as well as recurrence of the disease, can occur in survivors of neuroblastoma.

What is the latest research on neuroblastoma?

Boston Children's Hospital and Dana-Farber Cancer Institute are conducting numerous research studies that will help clinicians better understand and treat neuroblastoma. Researchers are constantly studying how to use high-dose therapy in combination with stem cell transplantation.

In addition, the Dana-Farber/Boston Children's Transplant Program is one of only eight institutions around the country that are investigating the use of umbilical cord transplantation. Also under study is a novel method for preventing graft versus host disease, a serious complication that occurs when transplanted cells do not recognize the tissues and organs of the recipient's body and react against the recipient's tissue. The result of this treatment approach, if it continues to be as successful, will be that the degree of match between donor and the recipient will not need to be particularly close, greatly increasing the pool of potential donors for each patient. This could also eliminate the need for long-term drug therapy traditionally needed to treat graft versus host disease.

Another promising area of research is occurring in the area of autologous transplantation, developing treatments that allow the use a patient's own stem cells to overcome neuroblastoma.

Other types of treatment currently being studied include:

  • angiogenesis inhibitors - substances that may be able to prevent the growth of tumors by blocking the formation of new blood vessels that feed the tumors

  • biological therapies - a wide range of substances that may be able to involve the body's own immune system to fight cancer or lessen harmful side effects of some treatments



Osteosarcoma occurs predominantly in adolescents and young adults.

Review of data from the Surveillance, Epidemiology and End Results program of the NCI resulted in an estimate of 4.4 per million new cases of osteosarcoma each year in people aged 0 to 24 years. The U.S. Census Bureau estimates that there will be 110 million people in this age range in 2010, resulting in an incidence of roughly 450 cases per year in children and young adults less than 25 years old.
  Osteosarcoma accounts for approximately 5% of childhood tumors. In children and adolescents, more than 50% of these tumors arise from the bones around the knee.

Osteosarcoma can rarely be observed in soft tissue or visceral organs. There appears to be no difference in presenting symptoms, tumor location, and outcome for younger patients (<10 years) compared with adolescents.

Two trials conducted in the 1980s were designed to address the natural history of surgically treated localized, resectable osteosarcoma of the extremity. The outcome of patients in these trials who were treated with surgical removal of the primary tumor recapitulated the historical experience before 1970; more than half of these patients developed metastases within 6 months of diagnosis, and overall, approximately 90% developed recurrent disease within 2 years of diagnosis.

Overall survival for patients treated with surgery alone was statistically inferior. The natural history of osteosarcoma has not changed over time, and fewer than 20% of patients with localized resectable primary tumors treated with surgery alone can be expected to survive free of relapse.


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