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How We Treat Waldenström's Macroglobulinemia

  • Steven Treon, MD, PhD, director, Bing Center for Waldenström's Macroglobulinemia

    Our unique approach

    The Bing Center for Waldenström's Macroglobulinemia is part of Dana-Farber Brigham Cancer Center's Center for Hematologic Oncology, one of the world's largest and most respected treatment centers for patients with disorders of the blood or bone marrow. Hematological disorders can take many different forms, and your care team here includes experts with specialized knowledge in treating various manifestations of Waldenström's.

    Our Center includes specialists who care for more than 700 cases a year, and are experts in creating a carefully timed care plan that works for each patient, minimizing side effects and enhancing your quality of life.

    We offer the most advanced treatments, including clinical trials. Many of these therapies were developed by scientists in our own laboratories.

    Multidisciplinary team

    Our physicians, hematologists/oncologists, physician assistants, nurse practitioners, nurses, laboratory investigators, clinical care coordinators, clinical social workers, and nutritionists work closely together to decide what kind of treatment – if any is needed – will work best to combat your particular disease at each stage.

    Our Waldenström's Tumor Board meets weekly to discuss every case. These specialists review your care plan to reach a consensus recommendation, which we share with you. Your team works to ensure that your care plan offers the best possible outcomes, and that all your personal needs are met.

    Personalized treatment

    As a highly specialized Center within Dana-Farber Brigham Cancer Center's Center for Hematologic Oncology, we focus on the distinct needs of people with Waldenström's. We provide a very personalized approach to your care. If you are likely to have a specific side effect from a treatment that will interfere with your life or plans, we take care to tailor your therapy to keep your quality of life at the center of the treatment plan.

    A distinguishing area of our expertise is in the treatment of adults over 60 years, who often have different needs from younger adults. For example, they may have a higher risk for heart disease, diabetes, or kidney problems, which influence the choice of treatment options. To address the specific needs of these patients, we offer clinical trials specifically for adults age 60 and over.

    Our physician-scientists' investigations into the genetic underpinnings of Waldenström's are paving the way for treatment approaches based in personalized medicine. We have numerous Waldenström's-related clinical trials currently underway testing novel, targeted drugs.

    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 wide range of resources – from nutritional services to integrative therapies – that we provide to support you and your family.

    Offering Expertise through Second Opinions

    • We believe there is value in patients with suspected or diagnosed Waldenström's receiving a second opinion.
    • We routinely evaluate specimens sent to us from outside centers. These specimens are evaluated by our entire team, including our expert hematopathologists.

    Waldenström's can be difficult to diagnose and properly treat. Reasons to consider a second opinion include:

    • To confirm your diagnosis.
    • If you have received a diagnosis and want to be treated at Dana-Farber Brigham Cancer Center.
    • To determine the optimal therapy, and whether any is needed at this time.
    • 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: 877-442-DFCI or 877-442-3324
    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 Waldenström's, we look forward to working with you. Learn how to refer a patient.

    Treatments for Waldenström's

    Each patient's care combines the finest treatments currently available for Waldenström's with innovative new therapies based on research discovered through our own laboratories. For newly diagnosed patients and for patients whose disease has relapsed, your care team will carefully consider every approach, including clinical trials evaluating new therapies.

    Certain factors affect your treatment options:

    • Your age.
    • Your general health.
    • Your levels of Immunoglobulin M (IgM), the presence of low blood counts, the need for disease control, whether you have low-risk disease or high-risk disease, and the chance of associated health problems.
    • Your lifestyle and goals.

    Treatments can include:

    Watchful waiting

    We take a strategic and thoughtful approach to treatment that considers your quality of life and goals for treatment. If you are asymptomatic or have precancerous Smoldering Waldenstrom's Macroglobulinemia (SWM) or Monoclonal Gammopathy of Undetermined Significance (MGUS), you may not need treatment right away and can be monitored regularly for disease progression.

    The main reason to consider watchful waiting is to avoid the side effects of treatment. We make every effort to ensure that the potential side effects from your therapy impact your life, hobbies, work, and interests as little as possible. Certain treatments may also heighten the risk of contracting secondary leukemia in the future. We are careful to factor this into treatment plans, especially for younger patients.

    Treatment approaches

    When treatment is needed, we take one of two major approaches: a standard chemotherapy-based regimen or an investigational approach (non-chemotherapy based). Radiation and surgery are not often used.

    We consider active treatment based on an evaluation of your symptoms, the likely progression of your disease, and your preference. Many treatments for Waldenström's are effective. In 80-90 percent of patients, treatment lowers IgM levels, and neuropathy, and increases hemoglobin.

    Treatments are delivered in various ways: IV, subcutaneously or with oral medication.

    For many patients, treatments last about two years, during which time patients are in remission. Some patients may continue with oral medication indefinitely. We use our deep experience and clinical judgment to put together the right treatment plan for you.

    While there is no cure, patients can live for decades with this disease.

    Chemotherapy-based regimens

    Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells by either eliminating the cells or stopping them from dividing.

    Cyclophosphamide-based chemotherapy is often used in combination with rituximab, and, when used together, they are highly effective. It goes inside the nucleus of malignant cells, causing mutations in its genes.

    Newer agents, such as proteasome inhibitor-based therapy, go inside the cell but not the nucleus, blocking the pathway in the cytoplasm of the cell so the cell can't recycle IgM and dies. Some studies show a side effect of neuropathy to this therapy, but it can be given subcutaneously in some cases, to lessen this risk.

    Bendamustine chemotherapy has been used since the 1940s and is a good option for patients with an enlarged liver or spleen. Overall, the response rate is 80 percent, though there is some chance of secondary leukemia.

    In 2015, Waldenström's became the fourth cancer for which ibrutinib has been approved by the U.S. Food and Drug Administration (FDA).

    • Ibrutinib is an oral agent that works by stifling a protein called BTK in malignant B cells. Normally, BTK enables B cells to reproduce, survive, and carry out two critical functions: adhere to neighboring cells and travel in and out of lymph tissue.
    • By blocking BTK, ibrutinib prevents cancerous B cells from carrying out the activities they need to survive.
    • Steven P. Treon, MD, PhD, director of Dana-Farber's Bing Center for Waldenström's Macroglobulinemia, led the clinical trial that supplied the data on which the FDA's decision was based.
    • Recent results from a clinical trial show that ibrutinib continued to control the cancer, with 95 percent of patients surviving at two years.

    Investigational approaches

    Our Center is committed to both world-class care and innovative research. This uniquely positions us to develop and deploy promising new treatments.

    The goals of all our clinical trials include increasing the number of patients entering into remissions – particularly complete remissions – as well as increasing the duration of these remissions.

    Clinical trials provide a range of treatment options, including studies aimed at improving the outcomes of patients undergoing biological therapies using agents, such as rituxan and bortezomib, which are designed to kill Waldenström's tumor cells directly. We also conduct trials evaluating supportive therapies like statins and phosphodiesterase inhibitors to better understand the mechanics of this disease.

    Clinical trials listed here are currently available at our clinic, as well as a number of our participating institutions.

    Some of our clinical trials are open through the Waldenström's Macroglobulinemia Clinical Trials Group, which is composed of more than 20 major cancer centers around the world.

    Relapsed disease

    At some point in time, nearly all patients with Waldenström's experience the progression of their disease. Fortunately, there are a large and expanding number of effective treatment options available, and you will have the opportunity to speak with physician about which regimen is most appropriate for you.

    Treatment decisions will take into account factors such as the type of treatment you have received in the past, the success of prior treatments for your condition, side effects that you have experienced, and your preferences for the type of treatment you'd like to receive.

    Once treatment starts for relapsed disease, you will be monitored closely for evidence that the treatment is working. Adjustments are made to enhance its effectiveness and how well you tolerate therapy. Under certain circumstances, therapy could be stopped and you may be observed without treatment.

    Breakthrough Research Informs Breakthrough Treatments

    Our scientists are working to understand the genetic basis and pathogenesis of Waldenström's, and are developing therapies. By establishing a family registry with more than 1,500 patients and their family members, our program has gained important insights into the genetic basis of the disease.

    These revelations have helped establish the importance of targeting certain cells and their tumor cell interactions, and have resulted in several clinical trials leveraging these findings.

    In recent years, Steven P. Treon, MD, PhD, and colleagues found that Waldenström's is driven by genetic mutations known as MYD88 and CXCR4, both of which were discovered by Treon in our Bing Center for Waldenström's Macroglobulinemia. Over 90 percent of patients with Waldenström's have the MYD88 mutation, which is a target for ibrutinib, a BTK inhibitor.

    These breakthrough findings show how whole genome sequencing can lead to the discovery of cancer mutations that can be specifically targeted by new therapies.

    New directions in treatment

    Some of the new agents being tested in our clinical trials over the next few years include oral proteasome inhibitors (ixazomib); BTK inhibitors (acalabrutinib, BCB-311); PI3K-delta (idelalisib); BCL2 antagonism (venetoclax); Anti-CD38 therapy (daratumumab); Anti-CXCR4 therapy (ulocuplumab); TLR inhibitor (IMO8400); IRAK1/4 inhibitor; MYD88 inhibitor.

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