Leukemia - Chronic Myeloid - CML

ON THIS PAGE: You will find information about the estimated number of people who will be diagnosed with chronic myeloid leukemia (CML) each year. You will also read general information on surviving the disease. Remember, survival rates depend on several factors, and no 2 people with cancer are the same. Use the menu to see other pages.

Every person is different, with different factors influencing their risk of being diagnosed with this cancer and the chance of recovery after a diagnosis. It is important to talk with your doctor about any questions you have around the general statistics provided below and what they may mean for you individually. The original sources for these statistics are provided at the bottom of this page.

How many people are diagnosed with CML?

About 15% of all leukemia is CML. In 2023, an estimated 8,930 people (5,190 men and 3,740 women) in the United States will be diagnosed with CML. Most of these will be adults, with an average age of diagnosis at 64 years. Nearly 50% of cases occur in people over age 65. CML is rare in children.

It is estimated that 1,310 deaths (780 men and 530 women) from this disease will occur in the United States in 2023.

What is the survival rate for CML?

There are different types of statistics that can help doctors evaluate a person’s chance of recovery from CML. These are called survival statistics. A specific type of survival statistic is called the relative survival rate. It is often used to predict how having cancer may affect life expectancy. Relative survival rate looks at how likely people with CML are to survive for a certain amount of time after their initial diagnosis or start of treatment compared to the expected survival of similar people without this cancer.

Example: Here is an example to help explain what a relative survival rate means. Please note this is only an example and not specific to this type of cancer. Let’s assume that the 5-year relative survival rate for a specific type of cancer is 90%. “Percent” means how many out of 100. Imagine there are 1,000 people without cancer, and based on their age and other characteristics, you expect 900 of the 1,000 to be alive in 5 years. Also imagine there are another 1,000 people similar in age and other characteristics as the first 1,000, but they all have the specific type of cancer that has a 5-year survival rate of 90%. This means it is expected that 810 of the people with the specific cancer (90% of 900) will be alive in 5 years.

It is important to remember that statistics on the survival rates for people with CML are only an estimate. They cannot tell an individual person if cancer will or will not shorten their life. Instead, these statistics describe trends in groups of people previously diagnosed with the same disease, including specific stages of the disease.

The pace of CML treatment research is rapid, with several drug approvals in the past 2 decades. It is important to note that many of the new drugs for CML work very well, and survival rates are continually being measured based on these newer drugs. Due in large part to recent scientific advances in the area of targeted treatments like imatinib (Gleevec), which was approved in 2001, the 5-year relative survival rate for CML more than tripled from 22% for people diagnosed in the mid-1970s to 70% for those diagnosed between 2012 and 2018. In general, most people diagnosed with CML and treated with a targeted therapy currently are expected to live an average life span with the disease well controlled.

The survival rates for CML vary based on several factors. These include the phase of the disease and other biologic factors, a person’s age and general health, and how well the treatment plan works.

Experts measure relative survival rate statistics for CML every 5 years. This means the estimate may not reflect the results of advancements in how CML is diagnosed or treated from the last 5 years. Talk with your doctor if you have any questions about this information. Learn more about understanding statistics .

Statistics adapted from the American Cancer Society's (ACS) publication, Cancer Facts & Figures 2023, and the ACS website. Additional source was : Seigel R, et al.: Cancer Statistics 2023 . CA: A Cancer Journal for Clinicians . 2023 Jan; 73(1):17–48. doi/full/10.3322/caac.21763 . (All sources accessed January 2023.)

The next section in this guide is Medical Illustrations . It offers drawings of body parts often affected by CML. Use the menu to choose a different section to read in this guide.

ON THIS PAGE: You will find out more about the factors that increase the chance of developing CML. Use the menu to see other pages.

A risk factor is anything that increases a person’s chance of developing cancer. Although risk factors often influence the development of cancer, most do not directly cause cancer. Some people with several risk factors never develop cancer, while others with no known risk factors do develop cancer. Knowing your risk factors and talking about them with your doctor may help you make more informed lifestyle and health care choices.

The cause of CML is not known, though researchers now understand how the disease develops from genetic changes in the bone marrow cells. Environmental factors account for only a small number of CML diagnoses. Family history does not appear to play a role in the development of CML.

The following factors may raise a person’s risk of developing CML:

• Age. The average age of people diagnosed with CML is around 64. CML is uncommon in children and teens.

• Radiation exposure. Many people who were long-term survivors of the 1945 atomic bombings in Japan were diagnosed with CML. In addition, radiation therapy for a condition called ankylosing spondylitis has been linked to CML. However, there is no proven link between CML and radiation therapy or chemotherapy given for other types of cancer or other diseases.

• Gender. Men are somewhat more likely to develop CML than women.

The next section in this guide is Symptoms and Signs . It explains what body changes or medical problems CML can cause. Use the menu to choose a different section to read in this guide.

ON THIS PAGE: You will find a list of common tests, procedures, and scans that doctors use to find the cause of a medical problem. Use the menu to see other pages.

Doctors use many tests to find, or diagnose, leukemia. They also do tests to learn more about the extent of the disease. Doctors may also do tests to learn which treatments could work best.

For most types of cancer, a biopsy is the only sure way for the doctor to know if an area of the body has cancer. In a biopsy, the doctor takes a small sample of tissue for testing in a laboratory. If a biopsy is not possible, the doctor may suggest other tests that will help make a diagnosis.

This list describes options for diagnosing CML. Not all tests listed below will be used for every person. Your doctor may consider these factors when choosing a diagnostic test:

• The type of leukemia suspected

• Your signs and symptoms

• Your age and general health

• The results of earlier medical tests

The following tests may be used to diagnose or monitor CML:

• Blood tests. Most people are diagnosed with CML through a blood test called a complete blood count (CBC) before they have any symptoms. A CBC counts the number of different kinds of cells in the blood. A CBC is often done as part of a regular medical checkup. People with CML have high levels of white blood cells. However, white blood cell levels might also be caused by conditions that are not leukemia. When the CML is more advanced, there may also be low levels of red blood cells, a condition called anemia, and either high or low numbers of platelets.

• Bone marrow aspiration and biopsy . These 2 procedures are similar and often done at the same time to examine the bone marrow. Bone marrow has both a solid and a liquid part. A bone marrow aspiration uses a needle to remove a sample of the fluid containing bone marrow cells. A bone marrow biopsy is the removal of a small amount of solid tissue using a needle. A pathologist then analyzes the sample(s). A pathologist is a doctor who specializes in interpreting laboratory tests and evaluating cells, tissues, and organs to diagnose disease. A cytogenetic analysis (see below) may also be done on the bone marrow samples.

  A common site for a bone marrow aspiration and biopsy is the iliac crest of the pelvic bone (see the Medical Illustrations section), which is located in the lower back by the hip. A bone marrow biopsy can usually be done in your doctor's office, without a need to stay in the hospital. Before the procedure, the skin in that area is usually numbed with medication. Other types of anesthesia (medication to block awareness of pain) may also be used.

• Molecular testing. Your doctor may recommend testing the leukemia cells for specific genes, proteins, and other factors unique to the leukemia. Results of these tests can help determine your treatment options .

  Cytogenetics is a type of genetic testing that is used to analyze a cell’s chromosomes. It looks at the number, size, shape, and arrangement of the chromosomes. Occasionally, this test can be done on the peripheral or circulating blood when the CML is first diagnosed, but immature blood cells that are actively dividing need to be used. Because of this, a bone marrow sample (see above) is usually the best way to get a sample for testing.

  For most people with CML, the Philadelphia (Ph+) chromosome and the BCR-ABL fusion gene (described in the Introduction section) can be found through testing, which confirms the diagnosis. For a small number of patients, increased blood cell counts may suggest CML, but the Philadelphia chromosome cannot be found on the usual tests even though the BCR-ABL fusion gene is there. Treatment for these patients is the same and works as well as it does for patients with a detectable Philadelphia chromosome.

  Cytogenetic testing for CML is used to monitor how well treatment is working and if it is reducing the number of cells with the Philadelphia chromosome. The following tests are sometimes used with cytogenetic testing:

• Fluorescence in situ hybridization (FISH) is a test used to detect the BCR-ABL gene and to monitor the disease during treatment. This test does not require dividing cells and can be done using a blood sample or bone marrow cells. This test is a more sensitive way to find CML than the standard cytogenetic tests that identify the Philadelphia chromosome.

• Polymerase chain reaction (PCR) is a DNA test that can find the BCR-ABL fusion gene and other molecular abnormalities. PCR tests may also be used to monitor how well treatment is working. This test is quite sensitive and, depending on the technique used, can find 1 abnormal cell mixed in with approximately 1 million healthy cells. This test can be done using a blood sample or bone marrow cells.

After treatment begins, cytogenetic and/or molecular testing is repeated on another bone marrow sample to find out if there are fewer cells with the Philadelphia chromosome.

• Imaging tests. Doctors may use imaging tests to find out if the leukemia is affecting other parts of the body. For example, a computed tomography (CT or CAT) scan or ultrasound examination is sometimes used to look at and measure the size of the spleen in people with CML.

• A CT scan takes pictures of the inside of the body using x-rays taken from different angles. A computer combines these images into a detailed, 3-dimensional image that shows any abnormalities. Sometimes, a special dye called a contrast medium is given before the scan to provide better detail on the image. This dye can be injected into a patient’s vein or given as a liquid to swallow.

• An ultrasound uses high-frequency sound waves to create a picture of the inside of the body.

After diagnostic tests are done, your doctor will review all of the results with you. If the diagnosis is CML, these results also help the doctor describe the phase of the disease.

The next section in this guide is Phases . It explains the system doctors use to describe the extent of the disease. Use the menu to choose a different section to read in this guide.

ON THIS PAGE: You will learn about the different types of treatments doctors use for people with CML. Use the menu to see other pages.

This section explains the types of treatments that are the standard of care for CML. “Standard of care” means the best treatments known. When making treatment plan decisions, you are encouraged to consider clinical trials as an option. A clinical trial is a research study that tests a new approach to treatment. Doctors want to learn whether the new treatment is safe, effective, and possibly better than the standard treatment. Clinical trials can test a new drug, a new combination of standard treatments, or new doses of standard drugs or other treatments. Clinical trials are an option to consider for treatment and care for all stages of CML. Your doctor can help you consider all your treatment options. To learn more about clinical trials, see the About Clinical Trials and Latest Research sections.

Treatment overview

In cancer care, different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team . It is important that a hematologist or an oncologist experienced in blood cancers treats a person with CML. A hematologist is a doctor who specializes in treating blood disorders. An oncologist is a doctor who specializes in treating cancer. Cancer care teams include a variety of other health care professionals, such as physician assistants, oncology nurses, social workers, pharmacists, counselors, dietitians, financial counselors, and others.

Descriptions of the common types of treatments used for CML are listed below. This is followed by information on measuring treatment effectiveness and the common treatment recommendations outlined by the disease phase . Your care plan will also include options to help prevent symptoms and side effects, as well as treatment for any symptoms and side effects you do experience. These are both important parts of cancer care.

Treatment options and recommendations depend on several factors, including the phase of the disease, possible side effects, and the patient’s preferences and overall health. Treatments for CML have improved greatly in the last 16 years, completely changing how treatment is given and helping many patients live much longer.

Take time to learn about all of your treatment options and be sure to ask questions about things that are unclear. Talk with your doctor about the goals of each treatment and what you can expect while receiving the treatment. It is also important to talk with your health care team about the costs of treatment, as many of the drugs discussed below need to be continued throughout a person’s life. Learn more about making treatment decisions .

Therapies using medication

Systemic therapy is the use of medication to destroy cancer cells. This type of medication is given through the bloodstream to reach cancer cells throughout the body. Systemic therapies are generally prescribed by a medical oncologist or a hematologist.

Common ways to give systemic therapies include an intravenous (IV) tube placed into a vein using a needle or in a pill or capsule that is swallowed (orally).

The types of systemic therapies used for CML include:

• Targeted therapy

• Chemotherapy

• Immunotherapy

Each of these types of therapies are discussed below in more detail. A person may receive only 1 type of systemic therapy at a time or a combination of systemic therapies given at the same time.

The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions by using searchable drug databases.

Targeted therapy

Targeted therapy is a treatment that targets the cancer’s specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to healthy cells.

Not all cancers have the same targets. To find the most effective treatment, your doctor may run tests to identify the genes, proteins, and other factors involved in your leukemia. This helps doctors better match each patient with the most effective treatment whenever possible. In addition, research studies continue to find out more about specific molecular targets and new treatments directed at them. Learn more about the basics of targeted treatments .

For CML, the target is the unique protein called the BCR-ABL tyrosine kinase enzyme. Drugs that target the BCR-ABL tyrosine kinase enzyme are called tyrosine kinase inhibitors or TKIs. These types of drugs can stop the BCR-ABL enzyme from working, which causes the CML cells to die quickly.

It is important to note that men and women taking TKIs should avoid fathering a child or becoming pregnant while taking the drugs because of a risk to the developing child. To find the best treatment, patients should talk with their doctors about the risks and benefits of these drugs, including the possible side effects and how they can be managed. For example, these drugs can cause inflammation of the liver, which is a problem for people with hepatitis. So, patients should be tested for hepatitis before starting treatment with any of these drugs. In addition, some TKIs may interact with certain foods, vitamins, or supplements. Talk with your health care team about what foods, vitamins, or supplements you may need to avoid. If a patient experiences too many side effects, another TKI can be used instead.

There currently 5 TKIs available for CML:

• Imatinib (Gleevec). Imatinib was the first targeted therapy approved by the U.S Food and Drug Administration (FDA) for CML in 2001. It is taken as a pill once or twice a day. It works better than chemotherapy to treat CML and causes fewer side effects (see below). Nearly all patients with chronic phase CML have their blood counts return to healthy levels and their spleen shrink after receiving this drug. Most importantly, 80% to 90% of patients newly diagnosed with chronic phase CML who receive imatinib no longer have detectable levels of cells with the Philadelphia chromosome. Imatinib may also be used to treat other types of cancer, such as acute lymphoblastic leukemia (ALL) with the presence of the Philadelphia chromosome.
  
  The risk of developing resistant CML once it completely responds to imatinib is very low. Patients with few numbers of cells with the Philadelphia chromosome remaining will stay in chronic phase longer by taking imatinib than they might have with previous treatments. It is too soon to know how long these responses will last or if patients will be cured with this medication alone. However, there are many patients who have been treated with imatinib since the first clinical trials in 1999 who still have no detectable cells with the Philadelphia chromosome.
  
  The side effects of imatinib are mild but can include stomach pain, which is very uncommon when imatinib is taken with food, changes in blood counts, fluid retention, swelling around the eyes, fatigue, diarrhea, and muscle cramps.
  
  
• Dasatinib (Sprycel). Dasatinib is approved by the FDA as an initial treatment for patients with newly-diagnosed chronic phase CML and when other drugs are not working. It is a pill that is usually taken once a day, or sometimes twice a day depending on the dose. The side effects include anemia, a low level of white blood cells called neutropenia, a low level of platelets called thrombocytopenia, and lung problems that include fluid around the lung and/or pulmonary hypertension. The doctor will monitor a patient’s blood counts frequently after starting dasatinib and may adjust the dose or stop giving the drug temporarily if the patient’s blood counts drop too low. Dasatinib may also cause bleeding, fluid retention, diarrhea, rash, headache, fatigue, and nausea. Dasatinib requires stomach acid in order to be absorbed so patients should not take any anti-acid medications.
  
  
• Nilotinib (Tasigna). Nilotinib is also approved by the FDA as an initial treatment for patients with newly-diagnosed chronic phase CML and when other drugs are not working. It is a capsule that patients take by mouth twice a day on an empty stomach. Common side effects include low blood counts, rash, headache, nausea, diarrhea, and itching. Other possible but uncommon serious side effects include high blood sugar levels, fluid build-up, and inflammation of the pancreas or liver. The most serious side effect of nilotinib includes possibly life-threatening heart and blood vessel problems that can lead to an irregular heartbeat, narrowing of the blood vessels, stroke, and possible sudden death. These side effects are very rare, but patients may need testing to check their heart health during treatment. There can be interactions with other medications that may increase these risks, so be sure to talk with your doctor about all medications you are taking.
  
  
• Bosutinib (Bosulif). In 2012, bosutinib was approved by the FDA to treat CML when 1 of the other TKIs was not effective or if a patient experienced too many side effects. The most common side effects include diarrhea, nausea and vomiting, low levels of blood cells, abdominal pain, fatigue, fever, allergic reactions, and liver problems.
  
  
• Ponatinib (Iclusig). Ponatinib was also approved by the FDA in 2012 for patients when 1 of the other TKIs was not effective or if a patient experienced too many side effects. Ponatinib also targets CML cells that have a particular mutation, known as T315I, which makes these cells resistant to other currently approved TKIs. The most common side effects include high blood pressure, rash, abdominal pain, fatigue, headache, dry skin, constipation, fever, joint pain, and nausea. Ponatinib may also cause heart problems, severe narrowing of blood vessels, blood clots, stroke, or liver problems.

Measuring treatment effectiveness of TKIs

Patients receiving a TKI should receive regular check-ups with the health care team to see how well the treatment is working. To start, these tests are generally done every 3 months during the first year of treatment. The response of CML includes:

• Complete hematologic response:

• Healthy levels of white blood cells and platelets

• No signs of abnormal blood cells, such as blasts, in the blood

• The spleen is a normal size and cannot be felt on a physical exam

• There are no CML symptoms

• Partial response:

• Blood counts are still abnormal

• There may still be some blasts in the blood

• The spleen may still be enlarged

• Symptoms and blood test results have improved since treatment began.

These responses can change over time, and there is a risk that the CML will worsen without more effective treatment. Sometimes this means continuing on the current TKI to see if the treatment helps further or it may mean changing to another TKI.

Other specific tests are used to find the number of cells that have the Philadelphia chromosome or contain the BCR-ABL fusion gene. When CML is diagnosed, the Philadelphia chromosome is found in almost all of a person’s bone marrow and blood cells. Once a person’s CML shows a complete hematologic response, the doctor then looks for a cytogenetic response with tests such as FISH (see Diagnosis ).

• A complete cytogenetic response means that there are no cells with the Philadelphia chromosome found on the routine cytogenetic tests.

• A partial cytogenetic response means that between 1% and 35% of the cells still have the Philadelphia chromosome.

• A minor cytogenetic response means that more than 35% of the cells still have the Philadelphia chromosome.

A molecular response can be determined when the PCR test is used to find the BCR-ABL fusion gene.

• A major molecular response means that a very small number of cells (more than 1,000 times fewer than when diagnosed) with the BCR-ABL fusion gene are found in the bone marrow or peripheral blood.

• A complete molecular response is when no cells with the BCR-ABL fusion gene are found in the bone marrow or peripheral blood.

An important initial goal of treatment is to achieve a complete cytogenetic response. This may require doing another bone marrow biopsy if it is unclear whether the drug is working. Or, another bone marrow biopsy may be needed after 6 to 12 months of treatment to confirm a cytogenetic response. It is not clear whether any of these drugs can cure CML. The disease may come back if treatment is stopped. If treatment with 1 of these drugs is working, a patient no longer has evidence of cells with the Philadelphia chromosome in the bone marrow and has normal levels of blood cells. This is called a complete cytogenetic remission. It is currently recommended that patients take these drugs throughout their lives prevent the CML from coming back. Recent research suggests that some patients may be able to safely stop treatment after a deep and stable response.

Monitoring

More sensitive blood tests, such as PCR and occasionally FISH (see Diagnosis ), are usually done every 3 months on a blood sample after a person has a cytogenetic response in the bone marrow cells. Patients who have no cells with the Philadelphia chromosome on regular cytogenetic tests often need to have PCR testing to find a molecular response . Patients who have a rapid decrease in the number of cells with the Philadelphia chromosome by 3 months after starting treatment may have the best long-term outcomes.

The most sensitive test to look for remaining CML is called a quantitative reverse transcriptase PCR (Q-RT-PCR) test. This test is recommended every 3 months on a blood sample. Generally, this test can find 1 CML cell remaining among 1 million healthy blood cells. When this test is negative, it is very likely that the CML is nearly gone. On the other hand, if a person continues taking the medication as directed and the results of this test begin to rise, then the current treatment is no longer working. This means that it may be time to switch medications before the disease worsens.

Sometimes, a TKI stops working because the CML develops resistance to it. One reason resistance can occur is if patients do not take their medication regularly, as prescribed, so it is important for patients to take their medication as prescribed . Even if patients do take the medication correctly, CML may still become resistant to a TKI, which is why it is also important to receive regular monitoring with cytogenetic testing, FISH, or PCR to see how well the drug is continuing to work.

Both dasatinib and nilotinib have been shown to bring about a complete cytogenetic response sooner and in more people newly diagnosed with CML when compared with imatinib. However, imatinib has been used for longer. There is no difference in overall survival when using either imatinib or another TKI as initial treatment. Bosutinib and ponatinib are newer drugs but both have also produced complete cytogenetic responses in people with CML. Because of possible severe side effects, caution and careful monitoring is needed if ponatinib is recommended after other drugs have stopped working. However, ponatinib is the only TKI that works for patients whose CML cells have the T315I mutation. If the medication you start with stops working, the dose may be increased or a different TKI may be used instead.

CML being treated with a TKI over the long term may be called a chronic cancer. This type of extended treatment can bring unique challenges. Learn more about living with chronic cancer .

Chemotherapy

Chemotherapy is the use of drugs to destroy cancer cells, usually by ending the cancer cells’ ability to grow and divide.

A chemotherapy regimen, or schedule, usually consists of a specific number of cycles given over a set period of time. A patient may receive 1 drug at a time or a combination of different drugs given at the same time.

A drug called hydroxyurea (Droxia, Hydrea) is often given to lower the number of white blood cells until CML can be diagnosed with the tests described in the Diagnosis section. Given in capsule form, this drug works well to return blood cells to normal levels within a few days or weeks and reduce the size of the spleen, but it does not reduce the percentage of cells with the Philadelphia chromosome and does not prevent blast phase alone. Although hydroxyurea has few side effects, most patients newly diagnosed with chronic phase CML receive imatinib or another TKI (see above) as soon as possible. This means that they do not need hydroxyurea, or use it for only a short time. Side effects of chemotherapy depend on the specific drug and the dosage and usually become less severe over time.

In 2012, the drug omacetaxine mepesuccinate (Synribo) was approved by the FDA for patients with chronic or accelerated phase CML that is not responding to the TKIs described above. Omacetaxine is given by injection under the skin daily for 7 to 14 days. The most common side effects include thrombocytopenia, anemia, neutropenia, diarrhea, nausea, fatigue, weakness, skin irritation where the drug was given, fever, and infection.

Learn more about the basics of chemotherapy .

Immunotherapy

Immunotherapy, also called biologic therapy, is designed to boost the body's natural defenses to fight the cancer. It uses materials made either by the body or in a laboratory to improve, target, or restore immune system function. Interferon (Alferon, Infergen, Intron A, Roferon-A) is a type of immunotherapy. It can reduce the number of white blood cells and sometimes decrease the number of cells that have the Philadelphia chromosome.

Interferon is given daily or weekly by an injection under the skin. It sometimes causes flu-like side effects, such as fever, chills, fatigue, and loss of appetite. When given on an ongoing basis, it can also cause loss of energy and memory changes. Interferon was the primary treatment for chronic phase CML before imatinib became available. However, interferon is no longer recommended as the first treatment for CML because research has shown that TKIs work better to treat CML and cause fewer side effects. However, unlike TKIs, interferon is safe to use during pregnancy.

Talk with your doctor about possible side effects for the immunotherapy recommended for you. Learn more about the basics of immunotherapy .

Bone marrow transplantation/stem cell transplantation

A bone marrow transplant is a medical procedure in which bone marrow that contains leukemia is replaced by highly specialized cells, called hematopoietic stem cells, that develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the stem cells in the blood that are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment. They will also consider several other factors, such as the phase of CML, results of any previous treatment, and patient’s age and general health. Although a bone marrow transplant is the only treatment that can cure CML, it is used less often now. This is because bone marrow transplants have a lot of side effects, while TKIs are very effective for CML and have fewer side effects.

There are 2 types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO uses donated stem cells, while AUTO uses the patient’s own stem cells. In both types, the goal is to destroy cancer cells in the marrow, blood, and other parts of the body using chemotherapy and/or radiation therapy and then allow replacement blood stem cells to create healthy bone marrow. Only ALLO transplants are used to treat CML.

Side effects depend on the type of transplant, your general health, and other factors. Learn more about the basics of stem cell and bone marrow transplantation .

Care for symptoms and side effects

Leukemia and its treatment often cause side effects. In addition to treatments intended to slow, stop, or eliminate the disease, an important part of care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care is any treatment that focuses on reducing symptoms, improving quality of life, and supporting patients and their families. Any person, regardless of age or type of cancer, may receive palliative care. It works best when palliative care is started as early as needed in the cancer treatment process. People often receive treatment for the leukemia at the same time that they receive treatment to ease side effects. In fact, people who receive both at the same time often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. You may also receive palliative treatments similar to those meant to eliminate the leukemia, such as chemotherapy. Talk with your doctor about the goals of each treatment in your treatment plan.

Before treatment begins, talk with your health care team about the possible side effects of the specific treatment plan and palliative care options. During and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem so it can be addressed as quickly as possible. Learn more about palliative care .

Treatment options by phase

Chronic phase

The immediate goals of treatment are to reduce any symptoms of CML. The longer-term goals are to decrease or get rid of the cells with the Philadelphia chromosome to slow down or prevent the disease from moving to blast phase. Treatment will often first include a TKI (see Targeted therapy, above). An ALLO stem cell transplantation would be considered afterwards only if TKI treatment does not work.

Accelerated phase

The same drugs used for chronic phase CML may also be used for accelerated phase CML. Although treatment with a TKI can work well for accelerated phase CML, it is less likely to work as well as it does for chronic phase CML. Dasatinib or nilotinib are more effective in providing longer remissions, but many patients have the CML return within about 2 years. Therefore, an ALLO stem cell transplantation should be considered when possible. If an ALLO stem cell transplantation is not recommended or if a matched donor cannot be found, the treatment plan may include a different TKI or a clinical trial .

Blast phase

Treatment with a TKI only works well for a few months for patients in blast phase, but it can help to control the CML while a stem cell/bone marrow transplant is being arranged. If the transplant can be done while imatinib or dasatinib is working, then the long-term results are better. Stem cell/bone marrow transplantation in the blast phase is less successful than in chronic phase, but this approach has worked well for some patients. Many people with CML in blast phase receive imatinib or dasatinib plus chemotherapy similar to that used for patients with acute leukemia, such as acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) . The chance of remission from this approach is about 20% to 30%, although the leukemia comes back for most patients within weeks to a few months. Hydroxyurea (see Chemotherapy, above) is often given to patients because it can help control blood cell levels. If stem cell/bone marrow transplantation is not an option, your doctor may recommend a clinical trial .

Resistant CML

If the leukemia does not respond to treatment, it is a good idea to talk with doctors who have experience in treating resistant CML. Doctors can have different opinions about the best standard treatment plan. Clinical trials might also be an option. Learn more about getting a second opinion before starting treatment, so you are comfortable with your chosen treatment plan. This discussion may include clinical trials .

Your treatment plan may include a combination of targeted therapy, chemotherapy, and bone marrow/stem cell transplantation. Palliative care will also be important to help prevent and relieve symptoms and side effects.

For most people, a diagnosis of resistant leukemia can be very stressful. You and your family are encouraged to talk about how you feel with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, including through a support group .

Remission and the chance of having the CML return

It is not yet proven whether imatinib, dasatinib, or nilotinib, or the newer drugs bosutinib, ponatinib, or omacetaxine can cure CML. A remission is when leukemia cannot be detected in the body by cytogenetic testing and there are no symptoms. This may also be called having “no evidence of disease” or NED.

A remission may be temporary or permanent. This uncertainty causes many people to worry that the leukemia will come back. While many remissions are permanent, it is important to talk with your doctor about the possibility of the disease returning. Understanding your risk of having the disease come back and the treatment options may help you feel more prepared if the leukemia does return. Learn more about coping with the fear of the CML returning .

If the leukemia does return despite the original treatment, a new cycle of testing will begin again to learn as much as possible about the disease. After this testing is done, you and your doctor will talk about the treatment options. Often the treatment plan will include the treatments described above such as targeted therapy, chemotherapy, and immunotherapy, but they may be used in a different combination or given at a different dose. Your doctor may suggest clinical trials that are studying new ways to treat this type of leukemia. Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.

People with leukemia that has come back after remission often experience emotions such as disbelief or fear. You are encouraged to talk with the health care team about these feelings and ask about support services to help you cope. Learn more about dealing with CML that comes back .

If treatment does not work

Recovery from leukemia is not always possible. If the leukemia cannot be cured or controlled, the disease may be called advanced or terminal.

This diagnosis is stressful, and for many people, advanced leukemia is difficult to discuss. However, it is important to have open and honest conversations with your health care team to express your feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable and that pain and other side effects are well-managed is extremely important.

People who have advanced disease and who are expected to live less than 6 months may want to consider hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to talk with the health care team about hospice care options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Learn more about advanced cancer care planning .

After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss .

The next section in this guide is About Clinical Trials . It offers more information about research studies that are focused on finding better ways to care for people with cancer. Use the menu to choose a different section to read in this guide.

ON THIS PAGE: You will read about the scientific research being done now to learn more about CML and how to treat it. Use the menu to see other pages.

Doctors are working to learn more about CML, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Most cancer centers are actively involved in clinical trials aimed at increasing the number of people who are cured of CML. Always talk with your doctor about the best diagnostic and treatment options for you.

• Improving current treatments. Research focused on increasing the effectiveness of CML treatments are listed below:

• Combining imatinib with other drugs

• Determining whether people with CML can safely stop taking TKIs after a molecular remission

• Creating vaccines against BCR-ABL

• Developing newer methods of bone marrow/stem cell transplantation aimed at decreasing the side effects

• Evaluating other new TKIs for CML that does not respond to imatinib

• Looking for unexpected long-term side effects

• Treatment to target remaining CML cells. Several laboratory studies are focused on possible treatments that may help destroy the few remaining CML cells in most patients who have received TKIs so they can stop medical treatment.

• Palliative care . Clinical trials are underway to find better ways of reducing symptoms and side effects of current CML treatments to improve patients’ comfort and quality of life.

Looking for More About the Latest Research?

If you would like additional information about the latest areas of research regarding CML, explore these related items that take you outside of this guide:

• To find clinical trials specific to your diagnosis, talk with your doctor or search online clinical trial databases now .
  
  
• Visit the website of Conquer Cancer, the ASCO Foundation , to find out how to help support cancer research. Please note that this link takes you to a separate ASCO website.

The next section in this guide is Coping with Treatment . It offers some guidance in how to cope with the physical, emotional, and social changes that CML and its treatment can bring. Use the menu to choose a different section to read in this guide.

ON THIS PAGE : You will read about why follow-up care is important. Use the menu to see other pages.

For many people with CML, targeted therapy is an ongoing cancer treatment. Any decision to stop taking these drugs should be made by a patient and doctor together, based on how well the drug continues to work and the side effects.

Even if after stopping treatment or a successful stem cell/bone marrow transplant, care for people diagnosed with CML does not end. Your health care team will continue to check to make sure the leukemia has not returned, manage any side effects, and monitor your overall health. This is called follow-up care.

Your follow-up care may include regular physical examinations, medical tests, or both. Doctors want to keep track of your recovery in the months and years ahead. Learn more about the importance of follow-up care .

Watching for resistant CML

One goal of follow-up care is to check for CML that has come back or worsened. CML comes back or worsens because small areas of leukemia cells may remain undetected in the body. Over time, these cells may increase in number until they show up on test results or cause signs or symptoms. During follow-up care, a doctor familiar with your medical history can give you personalized information about your risk of developing resistant CML. Your doctor will ask specific questions about your health. Some people may have blood tests or imaging tests done as part of regular follow-up care, but testing recommendations depend on several factors including the type and phase of leukemia originally diagnosed and the types of treatment given.

The anticipation before having a follow-up test or waiting for test results can add stress to you or a family member. This is sometimes called “scan-xiety.” Learn more about how to cope with this type of stress .

Managing long-term and late side effects

Most people expect to experience side effects when receiving treatment. However, it is often surprising to survivors that some side effects may linger beyond the treatment period. These are called long-term side effects. Other side effects called late effects may develop months or even years afterwards. Long-term and late effects can include both physical and emotional changes.

Talk with your doctor about your risk of developing such side effects based on the type of leukemia, your individual treatment plan, and your overall health. If you had a treatment known to cause specific late effects, you may have certain physical examinations, scans, or blood tests to help find and manage them.

Keeping personal health records

You and your doctor should work together to develop a personalized follow-up care plan. Be sure to discuss any concerns you have about your future physical or emotional health. ASCO offers forms to help keep track of the treatment you received and develop a survivorship care plan .

This is also a good time to decide who will lead your follow-up care. Some survivors continue to see their hematologist or oncologist, while others transition back to the care of their family doctor or another health care professional. This decision depends on several factors, including the type and phase of leukemia, side effects, health insurance rules, and your personal preferences.

If a doctor who was not directly involved in your care for CML will lead your follow-up care, be sure to share your treatment summary and survivorship care plan forms with him or her and with all future health care providers. Details about your treatment are very valuable to the health care professionals who will care for you throughout your lifetime. It may also be important to have these records available for those who care for you in case of an emergency.

The next section in this guide is Survivorship . It describes how to cope with challenges in everyday life after a CML diagnosis. Use the menu to choose a different section to read in this guide.

ON THIS PAGE: You will find some questions to ask your doctor or other members of the health care team, to help you better understand your diagnosis, treatment plan, and overall care. Use the menu to see other pages.

Talking often with the health care team is important to make informed decisions about your health care. These suggested questions are a starting point to help you learn more about your care and treatment. You are also encouraged to ask additional questions that are important to you. You may want to print this list and bring it to your next appointment, or download Cancer.Net’s free mobile app for a digital list and other interactive tools to help manage your care.

Questions to ask after getting a diagnosis

• What is my diagnosis?

• Can you explain my pathology report (laboratory test results) to me?

• What phase is the CML in?

• Can you recommend a leukemia specialist?

Questions to ask about choosing a treatment and managing side effects

• Where is the best place for me to receive treatment?

• What are my options for treatment?

• What clinical trials are available for me? Where are they located, and how do I find out more about them?

• What treatment plan do you recommend? Why?

• What are the pros and cons of each option?

• What is the goal of each treatment? Is it to eliminate the leukemia, help me feel better, or both?

• Do I need to start treatment right away?

• Who will be part of my health care team, and what does each member do?

• Who will be leading my overall treatment?

• What kinds of symptoms should I call my health care team about?

• What is my chance of remission and long-term benefit, including cure?

• What are the possible side effects of this treatment, both in the short term and long term?

• If I experience too many side effects, can I switch to a different treatment?

• How will this treatment affect my daily life? Will I be able to work, exercise, and perform my usual activities?

• Could this treatment affect my sex life? If so, how and for how long?

• What type of costs are associated with each treatment option? If I am worried about managing the costs of care, who can help me?

• What support services are available to me? To my family?

• Whom should I call with questions or problems?

• How will the results of my treatment be monitored?

Questions to ask about having targeted therapy

• What type of treatment is recommended?

• What is the goal of this treatment?

• How long will it take to give this treatment? When can you tell if it is working?

• What side effects can I expect during treatment?

• What are the possible long-term effects of having this treatment?

• What can be done to relieve the side effects?

• Will the medicine for CML interfere with any of my other medications?

• Are there foods I should avoid while taking this TKI?

• Can I stop TKI treatment to start a family? What if my partner and I conceive while I am taking a TKI?

• What are the risks of trying to conceive a child while taking a TKI?

Questions to ask about having chemotherapy and/or a bone marrow/stem cell transplant

• What type of treatment is recommended?

• What is the goal of this treatment?

• How long will it take to give this treatment?

• What side effects can I expect during treatment?

• What are the possible long-term effects of having this treatment?

• What can be done to relieve the side effects?

• Could this treatment affect my ability to become pregnant or have children? If so, should I talk with a fertility specialist before the transplantation process begins?

• Will the medicine for CML interfere with any of my other medications?

• Am I a candidate for an ALLO stem cell transplantation, and when should it be considered?

Questions to ask about planning follow-up care

• What is the risk of the CML returning or worsening? Are there signs and symptoms I should watch for?

• What is the chance that the leukemia will come back?

• What follow-up tests will I need, and how often will I need them?

• How do I get a treatment summary and survivorship care plan to keep in my personal records?

• Who will be leading my follow-up care?

• What survivorship support services are available to me? To my family?

The next section in this guide is Additional Resources . It offers some more resources on this website beyond this guide that may be helpful to you. Use the menu to choose a different section to read in this guide.