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Chemotherapy for Childhood Leukemia

Chemotherapy (chemo) is the main treatment for the most common childhood leukemias. Chemo is the use of drugs to kill cancer cells. Chemo can be given in a vein (IV), in a muscle, in the cerebrospinal fluid (CSF) during a lumbar puncture (called intrathecal chemo), or taken by mouth.

Chemotherapy for childhood leukemia by type

The type of chemotherapy used for treatment depends on the type of childhood leukemia.

ALL is treated with combinations of several chemo drugs. Chemo is given in phases or cycles. Rotating these drugs throughout treatment and giving them together can help reduce the chances that the cancer cells become resistant. Some periods during treatment are more intense and may cause more side effects and require time in the hospital. Other periods during treatment allow the body to recover.

ALL treatment starts with chemo to kill the leukemia cells such that cancer cells are no longer seen on tests of the blood and bone marrow. This is called putting the leukemia in remission. After that, chemotherapy is continued for a longer period (usually 2-3 years total) to prevent the cancer from coming back.

Some of the chemo drugs used to treat ALL include:

  • Anthracycline drugs (daunorubicin, doxorubicin)
  • Asparaginase (PEG-L-asparaginase [pegaspargase], calaspargase [CAL-PEG], Erwinia asparaginase)
  • Cyclophosphamide
  • Cytarabine
  • Mercaptopurine (6MP)
  • Methotrexate
  • Nelarabine (for T-cell ALL)
  • Steroids (prednisone, dexamethasone)
  • Thioguanine (6TG)
  • Vincristine

AML is treated with combinations of several chemo drugs. Chemo is given in phases or cycles. Each cycle includes a period of giving chemo, followed usually by a period of rest, which allows the body to recover from the chemo.

AML is treated with chemo over a shorter period compared to ALL, usually less than a year. Chemo for AML is more intense and may require more time in the hospital during treatment.

Some of the chemo drugs used to treat AML include:

  • Anthracycline drugs (daunorubicin, doxorubicin, mitoxantrone)
  • Cytarabine
  • Etoposide

Chemotherapy is not typically used in the treatment of CML. Chemotherapy may be used for CML if a stem cell transplant is recommended. See High-dose Chemotherapy and Stem Cell Transplant for Childhood Leukemia.

JMML is rare. The best chemo drugs for JMML are not known. Some of the chemo drugs that have been shown to work against JMML include:

  • Azacitidine
  • Cytarabine
  • Decitabine
  • Etoposide
  • Fludarabine
  • Mercaptopurine
  • Thioguanine

None of these chemo drugs have been found to work well in JMML without a stem cell transplant.

Possible side effects of chemo

Chemo drugs affect cells that multiply fast. Cancer cells multiply quickly, but 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 depend on the type and doses of the drugs, and the length of time they are given.

Common side effects

These side effects can include:

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

Possible side effects from low blood cell counts

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, called leukopenia)
  • Easy bruising or bleeding (from low platelets, called thrombocytopenia)
  • Fatigue (from low red blood cells, called anemia)

The problems with blood cell counts are often caused by the leukemia itself at first. They might get worse during the first part of treatment because of the chemo, but they will likely improve as the leukemia cells are killed off and the normal cells in the bone marrow have space to recover.

Possible side effects from specific chemo drugs

Some chemo drugs have other specific side effects. For example:

  • Steroids can have many side effects. These include high blood pressure, high blood sugar, changes in mood, changes in appetite, or trouble sleeping.
  • Vincristine can damage nerves, which can lead to numbness, tingling, or weakness in hands or feet (known as peripheral neuropathy).
  • Asparaginase can increase the risk of blood clots.

Some chemo drugs can also have long-term side effects, such as effects on growth and development, effects on fertility later in life, or an increased risk of getting a second cancer (often AML). Intrathecal (IT) chemo, given directly into the cerebrospinal fluid (CSF) around the brain and spinal cord, can have its own side effects. Intrathecal chemo may cause trouble thinking or learning in some children. It may even cause seizures.

For more on this, see After Treatment for Childhood Leukemia.

Managing side effects

Most side effects tend to go away once treatment is finished. There are often ways to reduce these side effects. For instance, 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.

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.

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Developed by the American Cancer Society medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).

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Last Revised: July 22, 2025

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