Chronic myeloid leukemia (CML) treatment

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It's important that your doctor is experienced in treating patients with chronic myeloid leukemia (CML) or has access to a CML specialist. Doctors who specialize in treating patients with CML are called “hematologist-oncologists.”

The information on this page covers treatment for adults with CML. Visit CML in Children and Young Adults to learn about signs and symptoms, diagnosis, and treatment information for children and young adults with CML.

Finding the best treatment approach

The treatment your doctor recommends is based on several factors, including the followin

The CML phase you're in
Your diagnostic test results
Prognostic factors
Other health issues

As you develop a treatment plan with your doctor, be sure to discuss certain important elements:

The results you can expect from treatment
The possibility of participating in a clinical trial, where you'll have access to advanced medical treatment that may be more beneficial to you than standard treatment
Potential side effects, including long-term and late effects

You may find it helpful to bring a loved one with you to your doctor's visits for support and to take notes and/or ask follow-up questions. It's a good idea to prepare questions you'd like to ask when you visit your doctor. You can also record your conversations with your doctor and listen more closely when you get home.

Other treatment considerations

If you're breastfeeding, pregnant, or thinking about getting pregnant, oral drug therapy puts your baby at risk. You and your doctor must discuss how treatment will adversely affect your baby or pregnancy. If you choose to stop treatment while pregnant, you risk a relapse.
If your cancer isn't controlled by treatment, you'll likely have to try a different treatment approach. See Refractory and Relapsed CML.

Types of CML treatment

Doctors use several types of approaches and treatment combinations for CML. Open each section below to learn more.

Some patients may have very high white blood cell (WBC) counts at the time of diagnosis. These elevated WBC counts can sometimes impair blood flow to the brain, lungs, eyes, and other sites, as well as cause damage to small blood vessels.

Hydroxyurea (Hydrea®) is sometimes given to lower very high WBC counts rapidly until a CML diagnosis is confirmed through blood and bone marrow tests. Hydroxyurea is taken as a capsule by mouth. Lowering those very high WBC counts can help reduce the size of the spleen. Once a diagnosis of CML is confirmed, doctors will usually start tyrosine kinase inhibitor (TKI) therapy and discontinue hydroxyurea.

Leukapheresis is a procedure that uses a machine similar to a dialysis machine to remove white blood cells from the circulating blood. Leukapheresis is used to lower WBC counts if chronic phase CML is diagnosed during the first months of pregnancy, when other treatments may be harmful to fetal development.

For information about the drugs listed on this page, visit our cancer drug listing.

For most people with CML, treatment begins with a targeted therapy called a tyrosine kinase inhibitor (TKI). These drugs are taken orally (by mouth). TKIs have changed CML from a potentially deadly cancer to one that can be controlled with medication.

Each TKI has risks and benefits. Patients should talk with their doctor to determine which TKI to use. Choosing a TKI is based on many factors including:

Patient age
Pre-existing medical conditions such as diabetes or high blood pressure
Phase of CML
Additional chromosomal abnormalities or gene mutations in the CML cells
Insurance coverage
Out-of-pocket costs

To date, there are six TKIs that are approved to treat CML. They are:

Asciminib (Scemblix®)
Bosutinib (Bosulif®)
Dasatinib (Sprycel®)
Imatinib mesylate (Gleevec®)
Nilotinib (Tasigna®, Danziten™)
Ponatinib (Iclusig®)

For some patients, their first TKI may not work because of drug resistance (when the cancer does not respond to treatment). For others, the side effects may be unbearable. In either case, a second TKI is tried. If both the first and second TKIs do not work, a third TKI can be offered to the patient.

Generic versions of TKIs have been available since 2016. A generic drug is a medication created to be the same as a brand-name drug that is already on the market. A generic medicine works in the same way and provides the same benefit as its brand-name version. In other words, you can take a generic medicine as an equal substitute for its brand-name counterpart.

The FDA has strict standards to ensure that generic drugs are the same as brand name drugs in the United States. Generic drugs typically cost less than brand-name versions. Talk to your doctor about whether a generic TKI is an option for you.

For information about the drugs listed on this page, visit our cancer drug listing.

Drug interactions

Certain drugs, herbal supplements, and even foods can affect the way TKIs work in the body. Corticosteroids, anti-seizure medication, antacids, and the herbal supplement St. John’s Wort can make some TKIs less effective. Certain antibiotics and antifungal medication, as well as grapefruit products, may increase the levels of TKIs in the blood to high, unsafe levels.

TKIs can have serious or even deadly interactions with other prescription medications, over-the-counter medications, supplements, and even certain foods. Patients should always provide their doctors with a list of any medications, herbal supplements, and vitamins that they are taking to be certain that it is safe to take the products with their TKIs. It is also important to ask the doctor about any foods that should be avoided.

TKI adherence

It is important for patients to take their TKIs as prescribed by their doctor. Adherence to an oral therapy means that the following is true of a patient:

They take the correct dose of medication
They take the medication at the correct time
They never miss a dose
They never take an extra dose
They do not take a dose with foods, liquids, supplements, or other medications that are not allowed
They do take a dose following any additional instructions, such as taking the medication with a meal or glass of water

In most patients, TKIs can control CML. Patients should not skip doses to try to reduce the side effects of the medication and should tell their doctors about any side effects that they are experiencing. Doctors can provide supportive treatment to help patients manage these side effects.

Patients must take their medication as prescribed to get the best response. Poor adherence to the medication regimen is the main reason for inadequate response to the prescribed treatment. Patients should not stop taking their medication, nor should they take less than the amount prescribed, unless they are following their doctors’ instructions. Taking less than the amount prescribed can affect how well the medication works and may result in unfavorable treatment outcomes.

TKI resistance

Drug resistance is the term used when a disease is not responding to treatment. Drug resistance occurs when a patient is taking a TKI but the number of leukemia cells in their blood increases.

Sometimes resistance to a TKI can be overcome by increasing the dose of the TKI or by switching to another TKI. Before changing treatment, the doctor should determine if the patient is taking the TKI as prescribed and find out if the patient is taking other drugs that may interact with the TKI. For some, a TKI stops working if there is a new mutation in the CML cells. The doctor should check for new mutations in the BCR::ABL1 gene.

Treatment-free remission

Treatment-free remission is achieved when patients can safely stop taking their TKI medications and still continue to have a deep molecular response. Patients in the chronic phase of CML who have had a good and deep molecular response for at least two years are considered candidates for stopping TKI therapy under careful medical supervision.

Talk to your doctor to see whether trying treatment-free remission may be a potential option in your case. It is important to consult with an experienced CML doctor before attempting treatment-free remission.

Immunotherapy is a drug therapy that stimulates the immune system. Interferon, a type of immunotherapy, is a substance made naturally by the immune system, but it can also be made in a laboratory. Interferon reduces the growth and division of cancer cells.

Before the introduction of TKIs, interferon was considered the first-line treatment for patients who were not candidates for an allogeneic stem cell transplant. Today, interferon therapy is rarely used as a treatment for CML because TKIs are more effective and have fewer side effects than interferon. But interferon may be an option for patients who cannot tolerate the side effects of TKI therapy or who are pregnant.

For certain patients with CML, allogeneic stem cell transplantation—the infusion of donor stem cells into a patient—is their best treatment option. However, this type of transplant can cause serious or even life-threatening complications and side effects. In addition, it is often not a good option for older patients or for patients who have other health problems.

The decision to pursue allogeneic transplantation has become more complicated because many patients have very good responses to TKIs. It is true that stem cell transplantation has been proven to be curative for some CML patients, but today, treatment with TKIs may control the disease for very long periods and preserve quality of life without the serious side effects of transplantation.

Doctors consider many important factors when deciding if an allogeneic transplant is the preferred choice of treatment for a patient. These factors include the patient’s age, general health, phase of CML, history of poor response to other treatments, and the availability of a well-matched donor. Results of transplants using stem cells from matched sibling donors are very similar to those of transplants that use stem cells from matched unrelated donors. Stem cell transplantation is considered for patients who have resistance to at least two types of TKIs, for patients whose CML is in accelerated or blast phase, and for patients who are intolerant to all TKIs.

The most important prognostic factor for post-transplant survival is the patient’s phase of CML. Approximately 80 percent of patients with chronic phase CML will be disease-free for five years after transplant. In patients with accelerated phase CML, approximately 40 percent to 50 percent are disease-free after five years, and only 10 percent to 20 percent of blast phase patients are alive and disease-free after five years.

Graft-versus-host disease (GVHD)

If you undergo allogeneic stem cell transplantation, you're at high risk of developing graft-versus-host disease (GVHD). GVHD develops when the donor's immune cells mistakenly attack the patient's normal cells. GVHD can be mild, moderate, or severe—even life threatening. The older you are, the higher your risk for GVHD.

Learn more about stem cell transplantation. 

Taking part in a clinical trial may be the best option for some CML patients. Clinical trials are designed to be accurate and very safe. There are clinical trials for newly diagnosed patients, for patients with advanced disease, and for patients who are either intolerant to or resistant to their current medications. The Leukemia & Lymphoma Society continues to invest funds in CML research.

Learn more about clinical trials.

Types of treatment for CML by phase

The phase of CML one is in helps determine the best treatment options. Open each section below to learn more.

The goal of treatment for chronic phase CML is remission.

Return blood cell counts to normal levels
Eliminate all cells that have the BCR:ABL1 gene

Tyrosine kinase inhibitor (TKI) therapy is standard treatment for chronic phase CML. TKIs are often successful at managing CML for long periods of time.

After you start TKI therapy, your doctor will check to see if your treatment is working. If you are not responding well, the doctor will need to find out why. If you are taking your TKI as prescribed, and your treatment is not working, a BCR::ABL1 gene mutation testing should be done to see if there are new mutations in the BCR::ABL1 gene.

If your first-line treatment does not work or stops working, your doctor may increase the dose of your current TKI or switch to another TKI.

Just as with the chronic phase, the goal in treating accelerated phase CML is to eliminate all cells that contain the BCR::ABL1 gene, leading to remission. If this is not possible, the goal is to return the disease to the chronic phase. Treatment at a specialized cancer center, with doctors who have expertise in treating CML patients, is recommended for patients in the accelerated phase of the disease.

In accelerated phase CML, the cancer cells often acquire new genetic mutations that may make treatments less effective. Patients should undergo BCR::ABL1 gene mutation analysis before starting treatment to determine which treatment option is best for them.

Treatment options for accelerated phase CML depend on the patient’s previous treatments. If CML is diagnosed in the accelerated phase and the patient has not yet tried a TKI, one treatment option is to begin TKI therapy using one of the following:

Dasatinib
Nilotinib
Bosutinib
Ponatinib
Other possible options include:
An allogeneic stem cell transplant
A clinical trial

For information about the drugs listed on this page, visit our cancer drug listing.

Patients with blast phase CML have more leukemia cells in the blood and bone marrow and more severe symptoms. Treatment at a specialized center with doctors who have expertise in CML is recommended for patients with blast phase CML. Patients’ cancer cells will need to be tested for different mutations.

Treatment options include:

TKI therapy, either with or without chemotherapy, followed by an allogeneic stem cell transplantation
A clinical trial

Get free clinical trial support! Visit our Clinical Trial Support Center (CTSC).

Connect with registered nurses with expertise in blood cancers who can personally assist you or your caregiver through each step of the clinical trial process.

Side effects

CML treatment can sometimes produce side effects. Side effects are problems that occur when treatments affect healthy cells or organs in the body. Most patients can manage their side effects without stopping therapy.

Before you start treatment, speak with your doctor about potential side effects. Drugs and other therapies can prevent or manage many side effects. Many treatment side effects go away or become less noticeable over time.

Side effects of tyrosine kinase inhibitor (TKI) therapy

Each TKI has its own possible side effects. In some cases, they can be serious. Your doctor will monitor you for side effects. Tell your doctor if you notice any change that could be a side effect of your TKI. Most side effects can be handled without the need to stop the drug; however, changing to another TKI may be an option to decrease side effects and improve quality of life.

Here are some questions to begin a discussion with your doctor.

What changes can safely be made with my current TKI to reduce my side effects?
Can I change the way I take my TKI to help manage the side effects? (Examples include dissolving the pill in juice, avoiding taking it before lying flat in bed at night, splitting up the dose, etc.)
What are the long-term side effects of my current TKI?
When should I think about changing my TKI based on side effects and how my treatment is affecting my daily activities?
Are there any tools or tips to help me track my side effects and the impact on my quality of life?
If I switch my TKI but find the new one less tolerable, can I go back on my current medication?

At your regular check-ups, your doctor should ask if you are experiencing any side effects from your TKI. Some common side effects of TKIs include:

Feeling very tired
Headache
Rash
Muscle cramps
Diarrhea
Nausea and vomiting
Low blood cell counts
Muscle and joint pain

Cardiac (Heart) effects

Patients treated with certain TKIs have sometimes, although rarely, developed cardiac issues. Most of the patients with these conditions have other health problems and risk factors, including older age and a medical history of heart disease. Your doctor should give you a list of medications to avoid and will monitor you for these conditions as needed. Cardiac effects can include:

Severe congestive heart failure (a weakness of the heart that leads to a buildup of fluid in the lungs and surrounding body tissues)
Left ventricular dysfunction (difficulty emptying blood from the left lower chamber of the heart)

A possible side effect of nilotinib that needs to be regularly monitored is a heart rhythm condition called QT prolongation. Patients should avoid other medications that are known to cause QT prolongation.

Other rare side effects

Dasatinib may increase the risk of a serious condition called pulmonary arterial hypertension (PAH). This side effect appears to be rare. Nilotinib may be associated with an increased risk of vascular events (disease relating to blood vessels).

Learn more about CML treatment side effects and how to manage them.

Treatment outcomes

Since the introduction of the drug Gleevec® and similar tyrosine kinase inhibitors, the number of patients with CML who enter remission and stay in remission for years has increased dramatically over the past decade.

It is important for patients to continue taking their medication to get the best response. Unless they are following their doctor’s instructions, stopping medication or taking less than the amount prescribed can impact how well the medication works and may result in a loss of response and an unfavorable treatment outcome.

For patients with CML, adhering to treatment is associated with the probability of achieving and improving long-term outcomes, including achieving a major molecular response and improved survival. Good adherence means that a patient follows all of their doctor’s recommendations, including the following:

Taking the appropriate number of pills as ordered at the right time, on the right day, every day
Reporting any side effects so that supportive treatment can be administered
Avoiding foods, liquids, or other medications that are not allowed

Patients should let their treatment team know if there are any problems (including side effects) that make it difficult for them to take the medication as prescribed. Generally, these difficulties can be either managed or completely overcome.

Learn more about oral treatment adherence in our free fact sheet, Oral Treatment Adherence Facts. (PDF)

Measuring treatment response

Your doctor needs to monitor your response to drug therapy for CML with blood and bone marrow tests. This is a critically important part of treatment for people with CML.

Your test results help your doctor decide whether to do the following:

Increase your drug dose to try for a better response
Decrease or stop the drug briefly because of side effects
Change to a different drug or drug combination to better control the CML
Change to a different drug or drug combination to manage side effects

Your doctor measures your response using general treatment response guidelines for your first year of CML drug therapy. They regularly measure the number of red cells, white cells, platelets, and CML cells in your blood. Your doctor compares these numbers with the results of the lab test done at the start of your treatment.

Types of responses

After patients begin treatment, their doctors will periodically order blood and bone marrow tests to determine whether they are responding to treatment. A treatment response is an improvement related to the patient’s treatment. Monitoring treatment response is one of the key strategies for managing CML. In general, the greater the response to drug therapy, the longer the disease will be controlled.

There are three types of responses: hematologic, cytogenetic, and molecular.

Hematologic

A complete hematologic response means that the numbers of white blood cells, red blood cells and platelets are normal or near normal.

The test: A complete blood count (CBC) is done to measure the numbers of white blood cells, red blood cells and platelets, and the levels of hemoglobin (a protein in red blood cells that carries oxygen) and hematocrit (the amount of red blood cells in the total blood volume).

Cytogenetic

A complete cytogenetic response means that there are no cells with the Ph chromosome detected in the bone marrow.

The test: A qPCR test measures the number of cells that have the BCR::ABL1 cancer gene in the blood. Either cytogenetic analysis or a FISH test is used if a reliable qPCR test is not available.

Molecular

A molecular response is a decrease in the number of blood cells with the BCR::ABL1 cancer gene. A patient’s molecular response is important in predicting outcomes and in determining future treatments.

In an early molecular response, no more than 10 percent of blood cells (10 out of every 100 cells) have the BCR::ABL1 cancer gene.
In a major molecular response (MMR), no more than 0.1 percent (1 out of 1,000 cells) have the BCR::ABL1 cancer gene.
In a deep molecular response (DMR), no more than 0.01 percent (1 out of 10,000 cells) have the BCR::ABL1 cancer gene or no more than .0032 percent (1 out of every 32,000 cells) have the BCR::ABL1 gene.

The test: A qPCR test measures the amount of BCR::ABL1 that can be detected in the blood. If possible, the same laboratory should be used for qPCR testing each time. This is because the results may vary from lab to lab.C

Fertility, pregnancy, and TKIs

Many CML patients of childbearing age are living with stable remissions and are considering having children while taking TKIs. For males taking TKIs, there are generally no concerns associated with having children.

However, for female patients who want to become pregnant, the issues are more complex. TKIs may result in abnormalities to the fetus or miscarriages. At present, no data suggest that any TKI can be taken safely during pregnancy. If treatment is needed during pregnancy, interferon is safe to use throughout pregnancy.

Doctors continue to study how CML treatment affects pregnancy. For more information, speak with your doctor so that you understand the:

Need to stop treatment during preconception (before you become pregnant) and pregnancy
Risk of relapse, if therapy is stopped
Risk for fetal effects from TKI drugs (probably greatest during the first trimester)
Need for females on TKI therapy to refrain from breastfeeding their babies

Treatment-free remission is now an emerging treatment goal for many patients with CML who have achieved a deep, stable response to treatment. Female patients who are interested in having children should discuss all their options with their treatment team, including the possibility of stopping TKI therapy.

If you are on TKI therapy, it is important to talk to your doctor if you are thinking about having a child.

Learn more about the impact CML treatment has on fertility and reproductive health.

Follow-up care

Find more information about follow-up care, including what to expect, long-term and late effects of treatment, survivorship clinics, and other resources, such as The National Comprehensive Cancer Network (NCCN) treatment guidelines.

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