Chemotherapy and Related Drugs for Neuroblastoma

Chemotherapy (chemo) is the use of anti-cancer drugs, which may be given into a vein or by mouth. 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 cannot be removed completely 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 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. If chemo is needed, the types of drugs and length of treatment depend on a child’s risk group. The main chemo drugs used for newly diagnosed neuroblastoma 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.

As researchers learn more about the genes that cause neuroblastoma, targeted therapies may be recommended as part of the treatment plan.

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 affect cells that grow fast. While cancer cells grow fast, other healthy cells in the body do too—for example blood-forming cells, hair cells, and cells that make up the lining of our gut. Damage caused by these drugs to fast-growing healthy cells results in 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 let your child’s doctor or nurse know if your child has side effects so they can be managed.

Long-term side effects of chemo

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 must be weighed against the importance of chemotherapy in treating neuroblastoma. For more on the possible long-term effects of treatment, see After Neuroblastoma Treatment.

Side effects of certain chemo drugs

Some chemo drugs can also have specific side effects. For example:

The doctors and nurses will watch closely for side effects. Do not hesitate to ask the cancer care team any questions about side effects.

For more information on the possible late or long-term side effects of chemo, including infertility and second cancers, see Late and Long-term Effects of Childhood Cancer Treatment.

Ornithine decarboxylase inhibitor

Eflornithine (also known as DFMO or Iwilfin) works in a different way than standard chemotherapy drugs. This drug blocks an enzyme inside cells known as ornithine decarboxylase (ODC), which is important for cell growth.

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

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

Side effects of DFMO

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: