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Chemotherapy and Related Drugs for Neuroblastoma

Chemotherapy (chemo) is the use of anti-cancer drugs, which are usually given into a vein. The drugs enter the bloodstream and travel throughout the body to reach and destroy cancer cells. This makes chemo useful for treating neuroblastoma, especially if it can’t all be removed with surgery.

When might chemotherapy be used?

Whether a child with neuroblastoma will get chemotherapy depends on their risk group, which is based on the stage (extent) and location of the cancer, the child’s age, and other factors. 

Some children with neuroblastoma are treated with chemo either before surgery (neoadjuvant chemotherapy) or after surgery (adjuvant chemotherapy).

In other cases, especially when the cancer has spread too far to be removed completely by surgery, chemo is the main treatment. 

Which chemo drugs are used for neuroblastoma?

Chemo for neuroblastoma usually includes a combination of drugs. The main chemo drugs used include:

  • Cyclophosphamide
  • Cisplatin or carboplatin
  • Vincristine
  • Doxorubicin (Adriamycin)
  • Etoposide
  • Topotecan
  • Melphalan (sometimes used during stem cell transplant)
  • Busulfan (sometimes used during stem cell transplant)
  • Thiotepa (sometimes used during stem cell transplant)

The most common combination of drugs includes cisplatin (or carboplatin), cyclophosphamide, doxorubicin, vincristine, and etoposide, but others may be used.

For children in the high-risk group, other drugs might be added as well, and some drugs might be given at higher doses. This may be followed by a stem cell transplant.

Doctors give chemo in cycles. Treatment is given for a few days in a row, followed by time off to allow the body time to recover. The cycles are typically repeated every 3 or 4 weeks. The total length of treatment depends on which risk group the child is in – higher risk groups usually require longer treatment.

Possible side effects of chemotherapy

Chemo drugs can affect other cells in the body that are dividing quickly, which can lead to side effects. The side effects of chemo depend on the type and dose of drugs given and the length of time they are taken.

General side effects of chemo

Side effects common to many chemo drugs include:

  • Hair loss
  • Mouth sores
  • Loss of appetite
  • Nausea and vomiting
  • Diarrhea or constipation

Chemo can damage the bone marrow, where new blood cells are made. This can lead to low blood cell counts, which can result in:

  • Increased chance of infections (from having too few white blood cells)
  • Easy bruising or bleeding (from having too few blood platelets)
  • Fatigue (from having too few red blood cells)

Most of these side effects tend to go away after treatment is finished. There are often ways to lessen these side effects. For example, drugs can be given to help prevent or reduce nausea and vomiting. Be sure to ask your child’s doctor or nurse about medicines to help reduce side effects, and let them know if your child has side effects so they can be managed.

Side effects of certain chemo drugs

Along with the effects listed above, some drugs can have specific side effects. For example:

Cyclophosphamide can damage the bladder, which can cause blood in the urine. The risk of this can be lowered by giving the drugs with plenty of fluids and with a drug called mesna, which helps protect the bladder. This drug can also damage the ovaries or testicles, which could affect fertility (the ability to have children).

Doxorubicin can damage the heart. Doctors try to reduce this risk as much as possible by limiting the doses of doxorubicin and by checking the heart with an echocardiogram (an ultrasound of the heart) during treatment. This drug can also cause skin damage if it should leak out of the vein while it is being given.

Cisplatin and carboplatin can affect the kidneys. Giving plenty of fluids can help reduce this risk. These drugs can also affect hearing. Your child’s doctor may order hearing tests (audiograms) during or after treatment.

Vincristine and etoposide can cause nerve damage (neuropathy). Some patients may have tingling, numbness, weakness, or pain, particularly in the hands and feet.

Chemotherapy can also have some longer-term side effects. For example, some drugs can increase the risk of later developing another type of cancer (such as leukemia). While this is a serious risk, it is not common, and the small increase in risk has to be weighed against the importance of chemotherapy in treating neuroblastoma. For more on the possible long-term effects of treatment, see Late and Long-term Effects of Neuroblastoma and Its Treatment.

Ornithine decarboxylase inhibitor

Eflornithine (Iwilfin) works in a different way than standard chemotherapy drugs. This drug affects an enzyme inside cells known as ornithine decarboxylase (ODC), which is important for the function of the MYCN oncogene. High-risk neuroblastoma cells often have too many copies of this gene, which helps them grow.

Eflornithine can be used in people with high-risk neuroblastoma to lower the risk of it coming back, if the cancer has already shrunk in response to previous treatment that included an anti-GD2 antibody.

This drug is taken as pills, typically twice a day for up to 2 years.

Side effects of eflornithine

Common side effects of this drug include: 

  • Infections, including ear, sinus, skin, respiratory, and urinary tract infections (UTIs)
  • Red and swollen eyes (pink eye) 
  • Diarrhea
  • Vomiting 
  • Cough
  • Stuffy, runny, or itchy nose, or sneezing
  • Fever 

More serious side effects can include:

Low blood cell counts: This drug can damage the bone marrow, where new blood cells are made. This can lead to low blood cell counts, which can result in:

  • Increased chance of infections (from having too few white blood cells)
  • Easy bruising or bleeding (from having too few blood platelets)
  • Feeling tired (from having too few red blood cells)

Tests will be done to check blood counts before and during treatment.

Liver problems: This drug can damage the liver, which might result in jaundice (yellowing of the eyes and skin), easy bruising or bleeding, nausea, and other problems. Blood tests will be done to check liver function before and during treatment.

Hearing loss: This drug might cause hearing loss, which can sometimes be serious. For people getting this drug, hearing will be checked before and during treatment.

More information about chemotherapy

For more general information about how chemotherapy is used to treat cancer, see Chemotherapy.

To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as editors and translators with extensive experience in medical writing.

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National Cancer Institute. Neuroblastoma Treatment (PDQ). 2020. Accessed at on April 9, 2021.

Park JR, Hogarty MD, Bagatell R, et al. Chapter 23: Neuroblastoma. In: Blaney SM, Adamson PC, Helman LJ, eds. Pizzo and Poplack’s Principles and Practice of Pediatric Oncology. 8th ed. Philadelphia Pa: Lippincott Williams & Wilkins; 2021.

Shohet JM, Lowas SR, Nuchtern JG. Treatment and prognosis of neuroblastoma. UpToDate. 2021. Accessed at on April 9, 2021.

Last Revised: December 19, 2023

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