Neuroblastoma Treatment and Diagnosis in Children
The Cancer Center at The Children's Hospital of Philadelphia has a multidisciplinary team of highly experienced professionals who will provide expert diagnosis and treatment of your child's neuroblastoma. In addition, Children's Hospital physician researchers are actively involved in developing new therapies to treat neuroblastoma. For children whose cancer is relapsed or refractory, our Refractory Neuroblastoma Program is comprised of international leaders in neuroblastoma treatment who are able to offer families a large portfolio of clinical trial options and unrivaled support services.
What is neuroblastoma?
Neuroblastoma is a tumor of nerve tissue that develops in infants and children and can occur in many areas of the body. It develops from the tissues that form the sympathetic nervous system (which controls body functions, such as heart rate and blood pressure, digestion and levels of certain hormones). It most commonly begins in the abdomen in the tissues of the adrenal gland, but it may also occur in other areas. It can spread to the lymph nodes, liver, bones and bone marrow. Children's Hospital doctors have discovered many of the reasons why neuroblastomas occur. Genetic testing for children is available at CHOP, under the direction of Drs. Yael Mosse and John Maris, where there appears to be a family history.
Who is diagnosed with neuroblastoma?
Neuroblastoma is typically diagnosed in babies or young children. The average age for neuroblastoma is about 18 months of age but it is occasionally seen in teenagers or even young adults. It is found slightly more often in boys than in girls. In the United States, about 800 new cases of neuroblastoma are diagnosed each year.
What are the signs and symptoms of neuroblastoma?
Signs and symptoms will vary depending on the site of the primary tumor or the extent that it has spread (metastasized). The most common primary site is the abdomen. Other primary sites include the chest, neck and pelvis. Neuroblastoma can spread to other areas of the body. If your child has a primary tumor in the abdomen, you may notice bellyaches, weight loss or a bigger abdomen. Some children only complain of general aches and pains.
How is neuroblastoma diagnosed?
Your child will need to have a series of tests to help us learn the extent of the disease. Some of the neuroblastoma tests include:
- Urine VMA/HVA test. This test will determine the amount of a substance called catecholamines in the urine; the tumor secretes and produces catecholamines.
- CT scan (computerized tomography) or MRI scan (magnetic resonance imaging). These scans are done to get pictures of the head, chest, abdomen and/or pelvis to measure the size of the tumor and to look for the spread of the disease.
- Bone marrow aspirate and biopsy. Bone marrow is removed from the bone (usually the hip bone) either by aspiration (suctioning a small amount through a hollow needle) or by biopsy (cutting out a small piece of bone marrow). This test is generally done under anesthesia.
- 123I-MIBG scan. A small amount of radioactive iodine (123I), linked to the very specific neuroblastoma chemical MIBG, is injected and an area of the body is viewed through pictures taken 24 hours later.
- Surgical biopsy. This test allows us to collect a small sample of the tumor. It is done in the operating room under general anesthesia. After the surgery, a large panel of laboratory and genetic tests are done on the tumor sample to determine how aggressive it is. These tests include, but are not limited to, International Neuroblastoma Criteria grade, MYCN amplification status, DNA index, whole genome SNP array, ALK mutation and amplification status, and 1p and 11p deletion status. We do not expect you to know what these tests mean, but your doctors use this information to determine the best treatment for your child.
How is newly diagnosed neuroblastoma treated?
We start by determining whether the disease risk is low, intermediate or high.
- Low-risk disease. If your child is under a year of age at diagnosis, or has small, easily removed tumors, the child may only need a simple surgical procedure or careful follow-up.
- Intermediate-risk disease. If your child has a larger tumor that has not spread, or is a baby with disease that has spread (but does not have the MYCN abnormality mentioned above), we will probably treat the child with surgery and four to eight months of chemotherapy. Often, patients with intermediate-risk disease do not need radiation treatments.
- High-risk disease. About half of neuroblastoma patients have high-risk disease, either because the tumor has spread to other parts of the body or because test results show high-risk features, such as MYCN amplification. If your child has high-risk neuroblastoma, treatment includes five months of chemotherapy, surgery, radiation therapy, high dose chemotherapy followed by his or her own stem cell rescue, and immunotherapy combined with biological therapy. These very intense treatments have improved the cure rate of this complex disease and Children's Hospital has led the efforts to develop these modern treatment approaches.
Newly diagnosed patients are generally treated according to Children's Oncology Group protocols, many of which were developed by CHOP doctors. However, every child will receive individualized treatment planning and other treatments may be recommended. Drs. John Maris, Yael Mosse and Edward Attiyeh provide comprehensive oversight for all newly diagnosed neuroblastoma patients. Dr. Stephan Grupp regularly focuses on children with advanced disease, and oversees all transplant and stem cell collection aspects of high-risk neuroblastoma patient care.
Surgery for Children with Neuroblastoma
The majority of patients with neuroblastoma will eventually require surgery. These operations tend to be quite difficult because of the nature of these tumors, which often wrap themselves around delicate structures in the body. At CHOP, surgical oncologists aim to remove as much of the tumor as possible, while preserving all normal organ functioning.
How is relapsed or refractory neuroblastoma treated?
Unfortunately, high-risk neuroblastoma still has a very high rate of non-responsiveness, or recurring/relapsing during or after treatment. Learn more about the innovative ways we treat kids whose disease is relapsed or refractory, through the Refractory Neuroblastoma Program at The Children's Hospital of Philadelphia.
Neuroblastoma late effects and survivorship
Some children treated for cancer develop complications years later. Our Cancer Survivorship Program provides information about the potential long-term effects of the specific neuroblastoma treatment your child received, including ways of monitoring and treating these effects.
Neuroblastoma research news
View the latest news in neuroblastoma research.
Read the Philadelphia Inquirer e-reprint article about Dr. Maris and Dr. Mosse's neuroblastoma translational research.
Reviewed by: John M. Maris, MD, The Children's Hospital of Philadelphia
Date: November 2009
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