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.”
Types of CML Treatment
Click the links below to read about types of treatment for CML.
- Lowering High White Blood Cell Counts (Leukapheresis)
- Tyrosine Kinase Inhibitor (TKI) Therapy
- Stem Cell Transplantation
- Clinical Trials
Each phase of CML requires different treatment:
For information about the drugs listed on this page, visit Drug Listings.
Tyrosine kinase inhibitor (TKI) therapy is standard treatment for chronic phase CML. TKIs are often successful at managing CML for long periods of time. Three TKIs are approved as primary treatment for chronic phase CML. They are
After the start of therapy, doctors will monitor patients to determine how well a patient is responding to treatment. A patient who is responding well will stay on his or her current drug therapy. If the patient is not meeting treatment milestones, the doctor will need to find out why. A gene mutation analysis should be done to check for mutations of the BCR-ABL1 gene. The doctor will also determine whether or not the patient has been adhering to the treatment plan. If the patient’s current treatment is not working, there are several options. They include
- Advising patients who have not been taking their TKIs as prescribed about the importance of adhering conscientiously to their medication regimen
- Increasing the dosage of the current drug (if possible)
- Switching to another TKI. For example, switching from imatinib to dasatinib, nilotinib, bosutinib or ponatinib
- Trying chemotherapy (such as omacetaxine, a chemotherapy option for patients with resistance or intolerance to two or more TKIs)
- Assessing whether an allogeneic stem cell transplantation is an option.
The goal in treating accelerated phase CML, just as with the chronic phase, is to eliminate all cells that contain the BCR-ABL1 gene, leading to a remission. If this is not possible, the goal is to return the disease to the chronic phase. Treatment at a specialized 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. This includes
If the CML progresses from chronic phase to accelerated phase during TKI therapy, a doctor may try to increase the dosage of the current TKI (if possible) or prescribe another TKI that the patient has not tried before. Other options include
- The TKI ponatinib for patients who have not responded to two or more TKIs and for patients who have the T315I mutation
- The chemotherapy drug omacetaxine (an option only for patients who have experienced resistance or intolerance to two or more TKIs)
- An allogeneic stem cell transplantation
- A clinical trial
Treatment at a specialized center with doctors who have expertise in treating CML patients is recommended for patients in the blast phase of the disease. Two important tests are needed before starting treatment for blast phase CML. The first test determines whether the blast phase involves myeloid or lymphoid blast cells. This test is needed because the type of blast cells is a factor in the treatment decision. The second test, a BCR-ABL1 kinase domain mutation analysis, checks for mutations in the part of the BCR-ABL1 gene that is targeted by TKIs. Different mutations can make the BCR-ABL1 protein either more or less resistant to certain TKIs.
Treatment options inlcude
- Treatment within a clinical trial
- TKI therapy, either with or without chemotherapy, and then proceed to an allogeneic stem cell transplantation.
Finding the Best Treatment Approach
The treatment your doctor recommends is based on several factors, including:
- The CML phase you're in
- Your diagnostic test results
- Prognositc factors
- Other health issues
As you develop a treatment plan with your doctor, be sure to discuss:
- 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 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.
To download lists of suggested questions to ask your healthcare providers, click here.
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.
- Communicating with Your Specialist
- Video clips answering FAQs about CML diagnosis and treatment
- Download or order The Leukemia & Lymphoma Society's free booklet, Chronic Myeloid Leukemia