Childhood Leukemia

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Treating Leukemia in Children TOPICS

Targeted therapy for childhood leukemia

In recent years, new drugs that target specific parts of cancer cells have been developed. These targeted drugs work differently from standard chemotherapy drugs. They sometimes work when chemo drugs don’t, and they often have different (and less severe) side effects. Some of these drugs may be useful in certain cases of childhood leukemia.

For instance, drugs such as imatinib (Gleevec) and dasatinib (Sprycel) specifically attack cells that have the Philadelphia chromosome (a shortened chromosome 22 that results from a translocation with chromosome 9).

Nearly all children with chronic myeloid leukemia (CML) have this abnormal chromosome in their leukemia cells. These drugs are very effective at controlling the leukemia for long periods of time in most of these children, although it’s not yet clear if the drugs can help cure CML.

A small number of children with acute lymphocytic leukemia (ALL) also have the Philadelphia chromosome in their leukemia cells. Studies have shown that the outcome is improved when these drugs are combined with chemotherapy drugs.

These drugs are taken daily as pills. Possible side effects include diarrhea, nausea, muscle pain, fatigue, and skin rashes. These are generally mild. A common side effect is swelling around the eyes or in the hands or feet. Some studies suggest this fluid buildup may be caused by the drugs’ effects on the heart. Other possible side effects include lower red blood cell and platelet counts at the start of treatment. These drugs might also slow a child’s growth, especially if used before puberty.

Other targeted drugs are now being tested in clinical trials as well.

For more general information on targeted drugs, see our document Targeted Therapy. If you’d like more information on a drug used in your child’s treatment or a specific drug mentioned in this section, see our Guide to Cancer Drugs, or call us with the names of the medicines your child is taking.


Last Medical Review: 10/24/2013
Last Revised: 02/03/2014