Your child's neuroblastoma treatment will depend on whether their disease is low-risk, intermediate-risk or high-risk. Your child's doctor can explain your child's risk level.
Treatments by Risk Level
Here are the most common treatments based on risk level. All of them are offered through Seattle Children's.
Most children with low-risk neuroblastoma have surgery to remove their primary tumor. Some have chemotherapy or radiation, too.
Some children with a certain form of low-risk disease have tumors that go away on their own. For children with this form, doctors may suggest "watchful waiting." This refers to the practice of closely tracking the child's cancer, without treatment, to see whether it changes.
Children at this risk level most often have surgery and chemotherapy. Some have radiation, too.
Treatment for high-risk neuroblastoma most often involves some mix of surgery, chemotherapy, radiation, hematopoietic stem cell transplant, a vitamin-like drug that slows formation of new cancer cells and a therapy that triggers the immune system to kill the neuroblastoma cells.
Neuroblastoma Treatment Options
Children with neuroblastoma usually have surgery. The first surgery your child has may be a biopsy. This means doctors remove a small sample of tumor cells to check for cancer. Doctors will remove as much of the tumor as is safely possible. This depends on where the cancer is and how high risk it is. Sometimes, the tumor is removed during the same surgery as the biopsy.
In other children, the tumor has spread to other places in the body or is too large to remove safely during the biopsy. In these cases, after the biopsy and diagnosis, the child receives chemotherapy to help control the tumor. Later, the child has surgery to remove as much of the tumor as possible. Studies have shown excellent results in low-risk and intermediate-risk neuroblastoma even if the whole tumor is not removed.
Doctors sometimes use chemotherapy or radiation before or after surgery. When used before surgery, these treatments may help shrink a tumor so that it’s easier to remove.
Even when doctors can remove the whole tumor, they may suspect or know that cancer cells are still in the child’s body. They often use chemotherapy, radiation or both to kill any cancer cells that are left.
Chemotherapy means giving medicines that go throughout your child’s body to kill cancer cells.
Your child’s doctors may suggest chemotherapy as the main treatment. Or they may suggest using it before, during or after other treatments. These medicines spread around the body through the bloodstream. They can help kill cancer cells that are in the blood and bone marrow. The drugs also help kill cancer cells that may have spread to other parts of the body.
The exact mix of medicines and how long they are given depend on the type of neuroblastoma your child has. Researchers are studying new mixes of medicines to find which work best against each type of the disease.
Our neuroblastoma patients receive chemotherapy at our hospital’s main campus in Seattle. Most often, children get treated while they stay in the hospital (as inpatients). But sometimes they are treated in a clinic (as outpatients). It depends on the type of medicines they need.
Your child’s doctors may suggest using radiation therapy too.
For low-risk or intermediate-risk disease, doctors may use radiation when a child has tumors that cause severe problems and treatment with medicine isn’t helping fast enough. Problems include tumors that block breathing, blood flow or organ function.
All children with high-risk neuroblastoma receive radiation to the first tumor. Sometimes they also get radiation to places where the tumor has spread.
Most children with neuroblastoma get external radiation. This means a machine sends a dose of radiation through the outer structures, such as the skin and the skull, into deeper parts of the body. Radiation uses strong X-rays to kill cancer cells and shrink tumors.
Radiation can cause unpleasant side effects. Patients may have nausea, sore throat or mild skin burning. Sometimes radiation causes long-term problems, such as infertility or second cancers. Radiation also can affect the way a child develops.
Researchers are looking for ways to give it in smaller doses or to smaller areas. They also study treatments that might replace radiation.
Our patients receive radiation through our partner, UW Medicine.
Children with high-risk neuroblastoma that has come back after treatment or that does not respond well to their first treatment may be able to have a newer type of radiation therapy. It is called I-131-MIBG therapy. This is a way to get radiation inside your child’s body and into their cancer cells via the bloodstream.
Learn more about Seattle Children’s Radiation Therapy Service.
Hematopoietic cell transplant, or stem cell transplant
Children with high-risk neuroblastoma may have high doses of chemotherapy medicines combined with a hematopoietic cell, or stem cell, transplant.
The medicines are designed to kill cancer cells that remain in the child’s body. Such high doses damage the bone marrow so it cannot make new stem cells that would become blood cells.
To rescue a child from the damaging effects of high-dose chemotherapy, doctors give the child bone marrow or stem cells through a vein. This is called an infusion.
Doctors remove the stem cells from the child before chemotherapy. The cells are frozen and stored until they can be returned to the child's body. This is called an autologous stem cell transplant.
Our patients who have these cell transplants get care through Seattle Children’s Hospital and our partner, Fred Hutch. Learn more about the Pediatric Blood and Marrow Transplant Program.
After transplant, children receive more treatment for 6 months. The aim is to kill any cancer cells that may regrow and cause the disease to come back. It includes:
- Isotretinoin. This vitamin-like drug slows the cancer’s ability to make more cancer cells. It is taken by mouth.
- A monoclonal antibody (ch14.18). It targets a substance (GD2) on neuroblastoma cells. The antibody then triggers the immune system to kill the neuroblastoma cells. “Monoclonal” means making many copies of the same antibody in the lab. Then doctors infuse the antibodies into your child’s body.
- Cytokines (GM-CSF). This is a type of messenger that helps make more immune system cells. These can attack and kill cancer cells.
- Interleukin-2 (IL-2). This boosts your child’s immune cells.
New Treatments for Neuroblastoma
Researchers are conducting studies to find better treatments for children with neuroblastoma. They are looking for the best mixes of medicines and for ways to use radiation and transplants with better results. They are also looking for new types of cancer treatments.
The cancer doctors at Seattle Children’s are active members of the Children’s Oncology Group (COG). COG is an international organization of childhood cancer specialists who conduct studies on many forms of childhood cancer.
Dr. Julie R. Park is chair of the COG neuroblastoma committee. The committee’s goal is to understand how the disease works and find new ways to treat it. Another goal is to reduce later effects of the disease and treatments.
Clinical trials mean more treatment options
Our doctors also take part in the NANT (New Approaches to Neuroblastoma Therapy) group. NANT works to find new treatments for children whose neuroblastoma has not fully responded to their first treatment or that has grown back.
Many of our patients with neuroblastoma take part in clinical trials. These research studies give children the chance to get the very latest treatment options being studied. These options are not offered at all treatment centers.
Among the options available at Seattle Children’s is a phase 1 clinical trial testing T-cell therapy in children and adolescents with neuroblastoma that has come back after treatment (recurrent) or that does not respond well to other treatment (refractory). This trial is known as Engineered Neuroblastoma Cellular Immunotherapy (ENCIT)-01. This therapy reprograms the body’s infection-fighting T cells to find and destroy cancer cells.
Children with high-risk recurrent or refractory neuroblastoma may be able to have a newer type of radiation therapy. It is called I-131-MIBG. This is a way to get radiation inside your child’s body and into their cancer cells via the bloodstream. Learn more.
Your child’s doctor will talk with you in detail about any new treatment that might be a match for your child. Then you can decide whether you want to try this option.
Read more about research at Seattle Children’s and about follow-up after treatment ends.
Contact the Cancer and Blood Disorders Center at 206-987-2106 for a referral, a second opinion or more information.