Overview
Treatment for CML depends on which stage of disease the patient is in: the chronic phase, accelerated phase, or blastic phase. Options include chemotherapy, immunotherapy, clinical trials and bone marrow transplantation.
Treatment by Phases
Chronic Phase
Gleevac is a drug that was licensed by the FDA (Food and Drug Administration) in June 2001. It may be recommended at the time of diagnosis. It works by decreasing the number of cells that contain the Ph chromosome. Gleevac is a pill that is taken by mouth. Because it is new and it is not yet clear what the long-term benefits may be, the role of bone marrow transplant has become less clear. For example, some doctors will recommend staying on Gleevac for a period of time, even if the patient has a bone marrow donor.
Interferon is also considered an important drug in this disease. It too, can decrease the number of cells with the Ph chromosome. However, interferon typically has more side effects. It is given by injection under the skin.
Bone marrow transplantation may be recommended in the chronic phase for patients who have an appropriate donor. This remains a controversial issue and you will need to discuss it with your doctor. For more information about stem cell or bone marrow transplantation, you may wish to order another of MSKCC's CD-ROM programs, Bone Marrow and Stem Cell Transplantation.
Accelerated Phase
This is the phase of CML when the leukemic cells begin to grow more rapidly and drug treatments fail. The goal of treatment is to reduce the white blood cell count.
Blast Phase
Treatment during this phase is aimed at getting the patient back into the chronic phase. Options for patients in the blast stage are a combination of hydroxyurea, 6-mercaptopurine, and prednisone; daunorubicin or doxorubicin and cytosine arabinoside; anagrelide (Agrylin®); and the interferons: interferon alpha 2a and interferon alpha 2b.
Drugs that might be used to treat CML are listed at the bottom of the page. Click on the drug(s) you will be receiving to get more information.
Treatment Decisions
The decision to attempt stem cell or bone marrow transplant will be guided by the patient's age and general health and the availability of a donor. Treatment with interferon often impacts quality-of-life and some patients may not tolerate it. However, interferon can prolong life in some patients. In the chronic phase, symptoms may be few and treatment with hydroxyurea improves these symptoms rather than extending survival.
Clinical Trials
Many leukemia patients are asked to participate in a clinical trial. A clinical trial is a research program that attempts to find better treatments. A Phase I clinical trial aims to find the safest way to give a new treatment. Patients are watched closely for side effects. Once the Phase I trial is completed, the new treatment may be evaluated in a Phase II clinical trial. At this stage the doctors are looking for anti-cancer activity, as well as side effects. After that, a Phase III clinical trial will compare the new treatment to a standard treatment, randomly assigning patients to either group. Patients may be offered the chance to take part in any of the three types of trials. Some people worry that they will receive only a placebo, such as an ineffective sugar pill, in a clinical trial, but that is very rarely done in cancer treatment clinical trials.
Patients are not required to take part in clinical trials. Those who choose to, however, may gain satisfaction from helping to evaluate new treatments, and may also have access to therapies that are not yet available to the general public. Patients may leave a clinical trial at any time, for any reason. Your doctor will explain why you may or may not be a candidate for a clinical trial.
For a listing of current clinical trials, see Clinical Trials.gov as well the clinical trials listing at this web site and other comprehensive or clinical NCI-designated cancer center websites.
During Treatment
Treatment for CML usually does not require hospitalization, unless there are complications. The drugs are taken by mouth at home and patients are followed as outpatients.
Activities
Patients are not generally limited in their activities during the chronic phase, but interferon immunotherapy may slow some down. The chronic phase often lasts for several years or more and patients may not have to make any changes in their lifestyle.