Treatment of Neuroblastoma by Risk Group

Treatment for neuroblastoma is largely based on which risk group a child falls into.

Low risk

Children at low risk usually don’t need very intensive treatment to cure the neuroblastoma. In fact, some children might not need to be treated at all because some of these neuroblastomas will mature or go away on their own.

Many children can have surgery as their only treatment. Even if some neuroblastoma is left behind after surgery, the child can usually be watched carefully without further treatment because the remaining tumor will often mature or go away on its own.

If much of the tumor can’t be removed or if it has some unfavorable features, chemotherapy is typically given after surgery. A common chemo regimen is a combination of carboplatin, cyclophosphamide, doxorubicin, and etoposide. But other combinations may be used.

For those few children that have symptoms from a low-risk tumor that can’t safely be treated right away with surgery, a short course of chemotherapy might be given first. For example, if the tumor is pressing on the spinal cord or affecting breathing, chemotherapy may be used to shrink the tumor to control the symptoms. Radiation therapy might be needed if chemotherapy doesn’t shrink the tumor fast enough.

Infants with stage 4S disease and no symptoms can often be watched carefully with no treatment, because these cancers typically mature or go away on their own. If the tumor causes problems such as an enlarged liver, which can be life-threatening for very young infants, chemotherapy that is less intense may be used to shrink the tumor. Radiation therapy may be used if chemo does not shrink the liver right away.

Recent research has also shown that infants younger than 6 months with small adrenal tumors (which are assumed to be neuroblastomas) can often be watched closely without needing surgery or other treatments. Many of these tumors will mature or go away on their own, but if a tumor keeps growing or is causing symptoms, surgery can be done to remove it.

Intermediate risk

Surgery is an important part of treatment for children at intermediate risk, but it is rarely enough on its own. Children are typically given 4 to 8 cycles (about 12 to 24 weeks) of chemotherapy before or after surgery. The chemo drugs used usually include carboplatin, cyclophosphamide, doxorubicin, and etoposide. Radiation therapy may be used if chemotherapy is not effective.

If chemotherapy is used after surgery, a “second look surgery” may be done to see if there is any cancer remaining and, if there is, remove it if possible. This might be followed by radiation therapy, if needed.

Doctors are now looking at the possibility of not giving chemo after surgery in some infants whose tumors are not causing symptoms. These children might just be watched closely after surgery and would only get further treatment if the tumor begins growing or causing symptoms. This approach is now being studied.

High risk

Children at high risk require more aggressive treatment, which often includes chemotherapy, surgery, and radiation. Treatment is often divided into 3 phases.

Induction: The goal of this phase is to get the cancer into remission by destroying or removing as much of it as possible. Treatment usually starts with chemotherapy, using alternating regimens of several drugs (typically cisplatin, etoposide, vincristine, cyclophosphamide, doxorubicin, and topotecan) given at higher doses than what is used in other risk groups. Surgery is usually done after this to try to remove any tumors that are still visible.

Consolidation: This phase uses more intensive treatment to try to get rid of any cancer cells that remain in the body. High-dose chemotherapy is given (sometimes along with radiation therapy), followed by a stem cell transplant.

Maintenance: The goal of this phase of treatment is to try to lower the chance that the cancer will come back. The retinoid drug 13-cis-retinoic acid (isotretinoin) is often given for 6 months after other treatments are completed. Immunotherapy with the monoclonal antibody dinutuximab, along with immune-activating cytokines (GM-CSF and IL-2), is often given as well.

Recurrent neuroblastoma

If neuroblastoma comes back after initial treatment, it is known as a recurrence or relapse. Treatment of recurrent neuroblastoma depends on many factors, including the initial risk group, where the cancer recurs, and what treatments have been used.

For low- and intermediate-risk neuroblastomas that recur in the same area where they started, surgery with or without chemotherapy may be effective.

For high-risk cancers or those that recur in distant parts of the body, treatment is usually more intense, and may include a combination of chemotherapy, surgery, and radiation therapy (such as MIBG radiotherapy). Chemotherapy might include drugs that weren’t used during the initial treatment. Intensive treatment with high-dose chemotherapy/radiation therapy followed by a stem cell transplant might be another option. Because these cancers can be hard to treat, clinical trials of newer treatments, such as monoclonal antibodies or new anti-cancer drugs, might be another reasonable option. (See the section What’s New in Neuroblastoma Research and Treatment?)

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Last Medical Review: March 14, 2014 Last Revised: January 22, 2016

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