Leukemia--Chronic Myeloid (Myelogenous) Overview

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Treating Leukemia - Chronic Myeloid (CML) TOPICS

Targeted therapies for chronic myeloid leukemia

The main treatment for chronic myeloid leukemia (CML) is drugs that target an abnormal protein (called a tyrosine kinase) in the leukemia cells. These drugs are called tyrosine kinase inhibitors (TKIs). Because these drugs target an abnormal protein in a cancer cell, they are also known as targeted therapy drugs.

It’s 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 or certain foods, like grapefruit or pomegranate. 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’s safe.

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

The TKIs used to treat CML are:

  • Imatinib (Gleevec)
  • Dasatinib (Sprycel)
  • Nilotinib (Tasigna)
  • Bosutinib (Bosulif®)
  • Ponatinib (Iclusig®): this is the only TKI that helps patients with CML if their leukemia cells have a certain gene change that means the other TKIs don’t work. It has more serious side effects than the other TKIs.

Bosutinib and ponatinib aren’t used as the first treatment for CML.

Nearly all patients respond to treatment with one of these drugs, and most of these responses 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.

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 detailed guide, Leukemia: Chronic Myeloid.


Last Medical Review: 02/03/2015
Last Revised: 02/03/2015