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Targeted therapy
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary. Independent clinical review is pending.
Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following:
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Tyrosine kinase inhibitor therapy: This treatment blocks the enzyme, tyrosine kinase, that causes stem cells to become more white blood cells (blasts) than the body needs. Tyrosine kinase inhibitors may be used with chemotherapy drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).
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Tyrosine kinase inhibitor therapy: This treatment blocks the enzyme, tyrosine kinase, that causes stem cells to become more white blood cells (blasts) than the body needs. Tyrosine kinase inhibitors may be used with chemotherapy drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).
Imatinib, dasatinib, and nilotinib are used to treat childhood chronic myelogenous leukemia.
Tyrosine kinase inhibitor therapy: This treatment blocks the enzyme, tyrosine kinase, that causes stem cells to become more white blood cells (blasts) than the body needs. Tyrosine kinase inhibitors may be used with chemotherapy drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).
Sorafenib, midostaurin, and gilteritinib, which target a mutated (changed) form of a gene called FLT3, are being studied to treat childhood AML.
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Tyrosine kinase inhibitor therapy: This treatment blocks the enzyme, tyrosine kinase, that causes stem cells to become more white blood cells (blasts) than the body needs. Tyrosine kinase inhibitors may be used with chemotherapy drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).
Trametinib is being studied in children with recurrent or refractory juvenile myelomonocytic leukemia.
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Monoclonal antibodies: Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then 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.
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Monoclonal antibodies: Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then 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 ozogamicin is a type of monoclonal antibody that is attached to a chemotherapy drug. It is used in the treatment of AML.
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See Drugs Approved for Leukemia for more information.
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Preserved source evidence · Independent clinical review pending · Not medical advice
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