How CML is Treated (Treatment)

Different treatment options are available for patients with CML. Bone marrow transplantation, drug therapy, radiation therapy, biologic therapy, or a combination of these approaches are used. Additionally, if the spleen is swollen, it may be removed.

The age of the patient, the phase of CML the patient is in, and other factors are considered in coming up with the best treatment plan for a patient.

Stem cell transplantation (SCT). SCT is the only treatment for CML known to bring about a cure. For this reason doctors will consider this treatment first for a newly diagnosed patient. But because SCT is a risky procedure that puts a great deal of strain on the body, not all patients can tolerate this treatment. This method is most successful when used in patients in the chronic phase of CML and in younger patients.

The goal of SCT is to kill the patient's damaged bone marrow (the stem cells are in the bone marrow) and replace it with healthy bone marrow. If the transplanted cells take hold, they grow and produce healthy blood cells.

The first step in stem cell transplantation involves destroying the patient's existing bone marrow with high doses of one or more types of anticancer drugs. Sometimes radiation therapy is also used. Both healthy and unhealthy bone marrow cells are killed in this step.

Most often the patient's bone marrow is replaced with healthy tissue from another person (the donor). This is called an allogeneic transplant. The patient receives the donor marrow through a needle into a vein, much like a blood transfusion. The donor marrow must closely match the tissue of the patient to allow the patient's body to accept the transplant. The most suitable donor is often a brother, sister, or another family member of the patient who has the same tissue type-known as HLA type-as the patient. If there is not a family member with the same tissue type, the doctor may search for a donor whose tissue type matches the patient. There are computerized lists of volunteer donors set up for this purpose. However, it can be difficult to find a donor whose tissue is a close enough match to use successfully.

Another approach is to use bone marrow taken from the patient. This is called an autologous transplant. This approach involves taking bone marrow from the patient and treating it with anticancer drugs with or without radiation to destroy the cancer cells. The patient then receives high-dose chemotherapy to kill the remaining bone marrow. After this step, the saved and treated bone marrow is injected through a vein back into the patient.

Interferon alpha. Called a biological therapy because it is a substance that the body makes to fight disease, interferon-alpha is given by injection. It can help slow the growth of the leukemia cells and prolong life in about two-thirds of patients. Other patients either do not respond or cannot tolerate the drug's side effects, which include flu-like symptoms such as fever, chills and fatigue. Interferon-alpha may be given alone or in combination with the drug cytarabine (ara-C). Interferon-alpha can produce long periods where the patient is free of the signs and symptoms of CML, but it does not bring about a lasting cure as SCT can.

Chemotherapy. Hydroxyurea and busulfan are two oral chemotherapy drugs that are used when patients cannot receive SCT or interferon-alpha or while they are waiting for the SCT. These drugs are usually better tolerated than interferon-alpha and can lessen the symptoms of CML. As with interferon-alpha, they do not provide a lasting cure.

Tyrosine Kinase Inhibitors (TKI's), molecularly-targeted agents to block the bcr-abl oncogenic pathway of CML, have revolutionized treatment of this disease. Most newly diagnosed patients are now considered appropriate candidates for TKI therapy which offers excellent rates of disease control for at least 5 years.

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