Chronic Myeloid Leukemia (CML) Treatment

There are several different treatment options for chronic myeloid leukemia (also called chronic myelogenous leukemia or CML). The type of treatments that are recommended are based on several factors, including the phase of CML, as well as the individuals' age and general health. The phases of CML are:

  • Chronic phase is when most people are diagnosed and is characterized by the blood and bone marrow containing less than 10% blasts (immature white blood cells).
  • Accelerated phase is generally characterized by an increase in blasts (more than 10% but less than 30%), a high basophil (specific white blood cell) count in the blood (at least 20%), new genetic changes in the leukemia cells, changes in the counts of white blood cells (high), and/or a decrease in the amount of platelets.
  • Blast phase, also called blast crisis, occurs when the bone marrow or blood has more than 20% blasts, and the blast cells typically have spread beyond the bone marrow to other organs or tissues in the body. At this phase, CML tends to behave more like an aggressive leukemia.1

Types of treatment for chronic myeloid leukemia

There are several different types of treatment that may be used for CML, including:

  • Targeted therapy
  • Chemotherapy
  • Stem cell transplant
  • Immunotherapy
  • Surgery
  • Donor lymphocyte infusion2

Targeted therapy

Targeted therapies are cancer treatments that block or slow the spread of cancer by interfering with specific areas of cancerous cells that are involved in the cancer cell's growth, or by focusing on particular characteristics that are unique to cancer cells. While chemotherapy drugs are often cytotoxic, meaning they kill cancer cells, targeted therapy is typically cytostatic, meaning it blocks the growth of cancer cells. Targeted therapies that may be used to treat CML are known as tyrosine kinase inhibitors (TKIs). TKIs target a specific protein that is found on CML cancer cells. TKIs are the standard treatment for CML in the chronic phase, although they may also be used to treat other phases of CML.2,3


Chemotherapy is the use of drugs to stop cancer cells. Chemotherapy medications may be used in combination, and chemotherapy drugs may be taken by mouth or injected into a vein or muscle. Chemotherapy works by targeting fast-growing cells, such as cancer cells. However, there are other fast-growing cells in the body that can also be affected, such as those in the gastrointestinal tract and hair.2,4

Stem cell transplants

Stem cell transplants are used in combination with high doses of chemotherapy. The high dose of chemotherapy destroys the cancer cells and also damages healthy blood cells. The transplant of stem cells, immature cells that can become new blood cells, is given to restore the bone marrow. The stem cells may be gathered from the patient prior to chemotherapy (called an autologous transplant), or they may be given by a donor (called an allogeneic transplant). Allogeneic stem cell transplants are generally more common in CML treatment, and they are the only known cure for CML. Not everyone is a candidate for stem cell transplants, however, as the high doses of chemotherapy can be very taxing on a person's body and may not be tolerated by older patients or those with other health problems.2,5


Immunotherapy, also called biologic therapy, helps boost the patient's own immune system to fight the cancer. For CML, the most common type of biologic therapy is interferon-alpha.2,6


In some cases, a person with CML may have surgery to remove an enlarged spleen. This surgery is called a splenectomy. While this surgery does not cure CML, it can relieve some of the symptoms, such as when an enlarged spleen presses on other organs like the stomach.2,7

Donor lymphocyte infusion

Donor lymphocyte infusions may be used in patients with CML following a stem cell transplant. Lymphocytes, one of the white blood cells that play a key role in the immune system, are gathered from the donor who provided stem cells. These donated lymphocytes are given to the patient, with the goal that the lymphocytes will attack and kill any remaining cancer cells.2

Clinical trials

Clinical trials are a type of research where new treatments are studied. Clinical trials are an important part of the scientific process to find and prove the safety and effectiveness of new treatments, and they offer patients a chance to receive the latest treatments and be closely monitored by healthcare professionals. Clinical trials can be found by talking to a doctor or through the website Patients can discuss treatment options with their doctor to determine if they might be eligible to participate in a clinical trial. 2,8

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Written by: Emily Downward | Last reviewed: October 2020