Treatments for Chronic Myelogenous Leukemia

Medically Reviewed by Dany Paul Baby, MD on April 22, 2022

There are a lot of ways to treat chronic myelogenous leukemia (CML) that can help bring your disease under control. To find the best one for you, you'll work closely with a specialist called a hematologist-oncologist, a doctor with special training in blood diseases such as leukemia.

The goal is to destroy cells that contain the BCR-ABL gene, which leads to too many abnormal white blood cells.

The First Steps

Your doctor will decide on a treatment plan based on the stage of your disease. They'll likely start you off with a type of drug called a tyrosine kinase inhibitor (TKI). It blocks a protein called tyrosine kinase, which is made by the BCR-ABL gene and plays a role in the growth of abnormal blood cells.

Your doctor will likely prescribe a TKI such as:

Most people get a quick response from these drugs. Your doctor will likely know in 3 to 6 months if your treatment works.

You may go into "remission" while you take a TKI. That means that the abnormal gene is no longer in your cells. It doesn't mean that you're cured, but your CML is now under control.

Always tell your doctor about any new symptoms. Some side effects that you might have from a TKI are:

  • Nausea and vomiting
  • Diarrhea
  • Rash
  • Headache
  • Fatigue
  • Lower blood cell counts

Is Your Treatment Working?

Your doctor will set several goals to help them check if your treatment is doing its job. For instance, they'll look to see that you have:

  • Normal blood cell counts with no signs of abnormal white blood cells, called a complete hematologic response.
  • No blood or bone marrow cells that contain the "Philadelphia" chromosome, which creates the BCR-ABL gene. This is called a complete cytogenetic response.
  • No sign of BCR-ABL in your blood, also called a complete molecular response.

Regular Testing

While you take TKIs, you'll get regular blood tests, including:

  • Complete blood counts to check white blood cells, red blood cells, and platelets
  • Blood cell exams to check the percentage of abnormal blood cells
  • Cytogenetic analysis, which looks for the abnormal Philadelphia chromosome
  • Polymerase chain reaction (PCR) tests to check for the BCR-ABL gene

A typical schedule for testing may look something like this:

  • During the first 3 months, you'll likely have a complete round of blood tests every 2 weeks.
  • At 3 months, you may get a follow-up bone marrow study. After the third month, you'll have blood and bone marrow tests at least once every 6 months until you have a complete cytogenetic response.
  • Once you have a complete cytogenetic and molecular response, you'll get a PCR test every 3-6 months and a cytogenetic analysis once every year.

What if TKIs Don’t Work?

If your CML doesn't slow down after you are treated with two or more TKIs, your doctor may switch you to another drug such as omacetaxine mepesuccinate (Synribo). It helps stop the growth of cancer cells. You get it as an injection.

You've got some other options:

Immunotherapy. It helps your immune system, your body's defense against germs, destroy the cancer. One example is a drug called interferon, which you take as an injection every day.

Chemotherapy. It kills the abnormal cells in your body, but it doesn't work as well for CML as other types of leukemia. It's usually used if you're in the "blast" phase of the disease, a period when infections and bleeding are common and can be life-threatening.

Allogeneic stem cell transplant. It's the only potential cure. The procedure is usually done if you're young and don't have any medical issues besides CML. It replaces abnormal white blood cells with stem cells you get from a donor and lets your body make healthy blood cells. But there are serious risks, including a disease called GVHD (graft-versus-host disease). When this happens, the new stem cells attack your normal cells by mistake.

If other treatments don't work, an experimental drug may be an option. Researchers test new therapies in clinical trials, which could give you access to cutting-edge treatment not yet available to the general public. To find out more, talk to your doctor.

Show Sources


American Cancer Society: "Treatment of Chronic Myeloid Leukemia by Phase."

The Leukemia & Lymphoma Society: "Chronic Myelogenous Leukemia."

Goldman, L. and Ausiello, D., eds. Cecil Medicine, 23rd ed. Saunders Elsevier; 2007.

Baccarani, M. Annals of Oncology, May 4, 2009.

FDA website.

ARIAD Pharmaceuticals Inc.

© 2022 WebMD, LLC. All rights reserved. View privacy policy and trust info