Leukemia--Chronic Myeloid (Myelogenous) Overview

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Treating Leukemia - Chronic Myeloid (CML) TOPICS

Targeted therapies for chronic myeloid leukemia

In the last 10 years, new drugs that are aimed at (target) specific parts of cancer cells have become the standard treatment option for many people with cancer. These drugs are often called targeted therapies. Some target a certain protein made by a cancer gene that is found in chronic myeloid leukemia (CML) cells. Drugs that do this are known as tyrosine kinase inhibitors (TKIs). Because these drugs are less likely to affect normal cells, their side effects are often not as bad as those seen with standard chemotherapy (chemo) drugs or with interferon (described below).

Still, these drugs have side effects, some of which are discussed below. It is also important to know that all TKIs can cause harm to the fetus if taken during pregnancy. They can also cause problems when you are taking other drugs. That’s why it is important to tell your doctor about any medicines you take, including over-the-counter medicines and supplements, and to check with your doctor before starting any new medicine to be sure it is safe.

Targeted therapies seem to work best on chronic myeloid leukemia (CML) that is still in the chronic phase, but they may also help people with more advanced disease.

All of these drugs are pills that are taken once a day.

Imatinib (Gleevec), Dasatinib (Sprycel), and Nilotinib (Tasigna): Imatinib was the first TKI and is often used as the first treatment for CML. Dasatinib and nilotinib were developed a little later, but may work even better than imatinib for some patients.

Nearly all patients respond to treatment with one of these drugs, and most of these responses seem to last for many years. But they don’t seem to cure CML (make it go away and stay away without more treatment). Because of this, doctors will start patients on one of these drugs, and give it to patients for as long as it seems to help. If one TKI stops working, doctors will often switch to another.

Bosutinib (Bosulif®): This is another TKI. So far, this drug does not seem to be much better than imatinib as the first treatment for CML, so it is mainly used in CML patients who have already been treated with another TKI.

Ponatinib (Iclusig®): This new TKI is used to treat patients with CML if their leukemia cells have a certain gene change that means the other TKIs don’t work. It can also be used if all of the other TKIs don’t work.

Side effects

Side effects of these drugs are usually mild, although more severe side effects can occur. There are often ways to treat these side effects if needed.

Common side effects are:

  • Diarrhea
  • Nausea
  • Muscle pain
  • Tiredness
  • Itchy skin rashes
  • Fluid build-up
  • Drop in the white blood cell and/or platelet count

Fluid build-up can cause swelling around the eyes, feet, or belly. In rare cases the fluid may collect in the lungs or around the heart, which can cause trouble breathing. If you are taking one of these drugs, tell your doctor right away if you notice sudden weight gain, trouble breathing, or fluid build-up anywhere in your body.

The different drugs can also have other side effects, some of which may be serious. Ask your doctor to expect.

More information about the targeted drugs used to treat CML can be found in our document Leukemia: Chronic Myeloid.


Last Medical Review: 08/13/2013
Last Revised: 02/10/2014