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Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Patient Information [NCI] - Treatment Option Overview


See Drugs Approved for Acute Myeloid Leukemia for more information.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. External radiation therapy may be used to treat childhood AML that has spread, or may spread, to the brain and spinal cord. When used this way, it is called central nervous system (CNS) sanctuary therapy or CNS prophylaxis.

Stem cell transplant

Stem cell transplant is a way of giving chemotherapy and replacing blood-forming cells that are abnormal or destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

Stem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Types of targeted therapy include the following:

  • Tyrosine kinase inhibitor therapy: Tyrosine kinase inhibitor (TKI) therapy blocks signals needed for tumors to grow. TKIs block the enzyme (tyrosine kinase) that causes stem cells to become more white blood cells (granulocytes or blasts) than the body needs. TKIs may be used with other anticancer drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).
  • Monoclonal antibody therapy: Monoclonal antibody therapy uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells.
    • Gemtuzumab is a type of monoclonal antibody used in the treatment of AML and a subtype of AML called acute promyelocytic leukemia (APL). Monoclonal antibodies may be used with chemotherapy as adjuvant therapy.
  • Proteasome inhibitor therapy: Proteasome inhibitors break down proteins in cancer cells and kill them.
    • Bortezomib is a proteasome inhibitor used to treat childhood APL.
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