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How We Treat Chronic Myelogenous Leukemia

  • Benjamin Ebert, MD, PhD, and Richard M. Stone, MD


    Benjamin Ebert, MD, PhD, Director of the Joint Leukemia Research Center at Dana-Farber/Brigham and Women's Cancer Center (DF/BWCC), and Richard M. Stone, MD, Director of DF/BWCC's Adult Leukemia Program

  • Our Unique Approach

    The Adult Leukemia Program is part of Dana-Farber/Brigham and Women's Cancer Center's Center for Hematologic Oncology, one of the world's largest and most respected treatment centers for patients with blood or bone marrow disorders.

    Your care will involve the best treatments currently available, including new therapies based on research in our laboratories and elsewhere in the field of leukemia treatment. You also have the full range of support services from a top-ranked hospital and leading cancer research center.

    Team Approach

    You receive world-class care from specialists with years of expertise in caring for patients with CML. These experts manage your diagnosis and treatment plan as a team in order to decide what approach will best treat your condition at each phase. Our specialists have extensive experience in standard and emerging therapies, and in monitoring the disease at a molecular level to ensure that your care plan offers the best possible long-term outcomes.

    Personalized Treatment

    As a highly specialized center, we focus on the distinct needs of people with leukemia. We provide a very personalized approach to your care.

    We view every patient as an individual, with unique needs and expectations. We take time to involve you and your family in each step of the treatment process. As you go through treatment, you will have access to a full range of support resources — from nutrition services to integrative therapies — that we provide to support you and your family.

    Older Adult Hematologic Malignancy Program

    Our Older Adult Hematologic Malignancy Program is a specialized DF/BWCC research and clinical service that addresses the specialized needs of blood cancer patients age 75 and older. Geriatricians work closely with oncologists to create individualized treatment plans for older patients that take into account other illnesses or concerns. These patients may participate in research protocols tailored to older adults, including clinical trials and tissue banking efforts.

    Offering Expertise through Consultations and Second Opinions

    • We believe there is great value in patients with suspected or diagnosed chronic myelogenous leukemia consulting with our team to verify or develop a plan of care.
    • We routinely evaluate specimens sent to us from outside centers. These specimens are evaluated by our entire team, including our expert hematopathologists.

    Reasons to consider a consultation or second opinion include:

    • To confirm your diagnosis.
    • If you have received a diagnosis elsewhere and want to be treated at Dana-Farber/Brigham and Women's Cancer Center.
    • To determine the optimal therapy and timing of treatment.
    • To learn more about your cancer from specialists who are world leaders in this disease, and who have treated hundreds of other patients like you.
    • To learn if you're eligible for a clinical trial.

    Phone: 617-632-6028 or 617-632-5138

    Online: Complete the Appointment Request Form.

    If you cannot travel to Boston in person, you can take advantage of our Online Second Opinion service.

    For Referring Physicians

    Because you, the referring physician, are an integral part of your patient's care team, we are committed to collaborating with you to provide the best care for your patient.

    If you are a physician and have a patient with diagnosed or suspected CML, we look forward to working with you. Learn how to refer a patient.

    Our Treatment Approach

    The treatment for CML has been revolutionized by the advent of the medication imatinib and the second- and third-generation tyrosine kinase inhibitors (TKIs) for CML. These are oral medications taken daily. They work to inhibit the function of the BCR-ABL gene. Many patients take these medications for the rest of their lives. In some instances, a patient may need treatment with a stem cell transplant.

    We look at several factors, including risk level, phase of the disease, diagnostic findings, and side effect profiles, to determine which tyrosine kinase inhibitor (TKI) to include as part of your therapy. Treatment has several goals. The first is the restoration of your blood counts to normal (hematologic response). More important, however, is to lower or eliminate the number of cells that have the Philadelphia chromosome. Patients will be tested and monitored using bone marrow biopsies and blood tests to determine cytogenetic and molecular response. Cytogenetic and molecular responses are achieved over several months and years, respectively.

    A distinguishing area of our expertise is evaluating how well you respond to therapy on a molecular level through sensitive testing.

    Treatment of CML may include the following:

    Targeted Therapy
    Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitors (TKIs) are targeted therapy drugs often used as treatment for newly diagnosed patients with chronic phase CML. TKIs, such as imatinib and others, can stop the BCR-ABL enzyme from working, which causes the CML cells to die.

    Most of the time, patients respond well to TKIs. Ultimately, the goal is to go into a cytogenetic remission, and then as deep a molecular remission as possible. This can take months or years to achieve. Patients are typically monitored 3 to 4 times a year after the first year to ensure that the disease is still responding to medication. Currently, we recommend that most patients remain on some form of TKI indefinitely, but some patients are able to come off and stay off their medications.

    Stem Cell Transplant
    Allogeneic stem cell transplant may be considered if the disease stops responding to TKI treatment. During an allogeneic transplant, diseased bone marrow cells are replaced with healthy ones from a donor. These new stem cells grow into (and restore) the body's blood cells. This treatment offers the only potential for a cure for CML. Dana-Farber/Brigham and Women's Cancer Center (DF/BWCC) has one of the largest and most experienced stem cell transplantation programs in the country.

    Chemotherapy
    Chemotherapy may be considered if targeted therapy and a stem cell transplant are not effective. Chemotherapy uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.

    Treating Resistant Disease
    In some cases, CML may become resistant to treatment. Fortunately, there are options available. You will have more testing, and we will discuss your treatment options. These may include targeted therapy used in a different combination or given at a different dose, or a stem cell transplant.

    New Directions in Treatment
    The Adult Leukemia Program operates a large and growing research program. We typically have clinical trials actively enrolling patients for the treatment of CML, including trials of newer agents for patients who have developed resistance.

    Patients have access to trials that:

    • Originate within Dana-Farber/Brigham and Women's Cancer Center and other Harvard-affiliated institutions
    • Ae sponsored by private industry
    • Are part of a collaborative effort, such as the Cancer and Leukemia Group B, of which our program is a member

    Your Care

    You will be assigned to one of our leukemia physicians as your primary oncologist overseeing all aspects of your care, both inpatient and outpatient.

    All outpatient therapy is provided at the Yawkey Center for Cancer Care at Dana-Farber Cancer Institute, one of the most advanced outpatient cancer centers in the country.

    If you need to be hospitalized during your care, or if you undergo stem cell transplantation, you will be admitted to Brigham and Women's Hospital (BWH) or the Dana-Farber Inpatient Hospital located within BWH. Your primary oncologist and nurse will closely monitor your care and will coordinate your care with the inpatient team and additional specialists, who will address any other symptoms you may be experiencing. This model ensures seamless care from the outpatient to the inpatient setting. Learn more about your stay.