Chemotherapy for Neuroblastoma

Chemotherapy (chemo) uses 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 that has spread to the lymph nodes, bone marrow, liver, lungs, or other organs.

Whether a child with neuroblastoma will get chemotherapy depends on their risk group.  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, chemotherapy is the main treatment. 

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

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

The most common combination of drugs includes carboplatin (or cisplatin) , cyclophosphamide, doxorubicin, and etoposide, but others may be used. For children in the high-risk group, more combinations are used, and some drugs are given at higher doses, which may be followed by a stem cell transplant (described further on).

Doctors give chemo in cycles, which consist of treatment on 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 attack cells that are dividing quickly, which is why they work against cancer cells. But other cells in the body, such as those in the bone marrow (where new blood cells are made), the lining of the mouth and intestines, and the hair follicles, also divide quickly. These cells are also likely to be affected by chemo, 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 can include:

  • Hair loss
  • Mouth sores
  • Loss of appetite
  • Nausea and vomiting
  • Diarrhea or constipation
  • 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 him or her know if your child has side effects so they can be managed.

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. These drugs can also damage the ovaries or testicles, which could affect fertility (the ability to have children).

Doxorubicin can cause heart damage. 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 can damage nerves. 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.

For more information on chemotherapy in general, see Chemotherapy.

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 journalists, editors, and translators with extensive experience in medical writing.

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National Cancer Institute. Physician Data Query (PDQ). Neuroblastoma Treatment. 2017. Accessed at on October 12, 2017.

Pinto NR, Applebaum MA, Volchenboum SL, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol. 2015: 30;3008-3017.

Speleman F, Park JR, Henderson TO.  Neuroblastoma: a tough nut to crack. Am Soc Clin Oncol Educ Book. 2016; 35:e548-557.  Accessed at on October 15, 2017.

Last Revised: March 19, 2018

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