There are no treatments that can cure WM, although in most cases the disease is indolent (slow growing) and can be effectively managed with appropriate therapies.

Overview of Treatments

There are many different treatment options available for WM patients, and they are increasing as researchers discover more about the biology and genetics of the disease.

Treatment may consist of just one drug (single-agent therapy), or two or more drugs (combination therapy).  Most studies seem to indicate that combination therapies are more effective, resulting in better and/or longer-lasting responses.

Treatment can usually be administered in an outpatient setting or at home.  It may be oral, by intramuscular or subcutaneous injection, or by intravenous therapy.  Some treatments require that certain medications be taken the day before or the day of treatment in order to minimize associated side effects.

Treatment cycles may take several weeks to months, depending on the course of therapy chosen. It is not unusual to have a round of therapy and then wait a week or a month before another round of treatment, although some of the newer oral therapies require daily dosing instead. Hematologist-oncologists follow established protocols for treatment but may make adjustments depending on side effects or response to therapy.

Most of the treatments in use today were originally approved for the related cancers of follicular lymphoma, chronic lymphocytic leukemia, and multiple myeloma.  Once additional Phase 1 and Phase 2 clinical trials established that these treatments had both an acceptable safety profile and were effective for WM patients as well, they were prescribed for “off label” use in WM.

The standard first-line treatment protocol (i.e., the typical first treatment for a WM patient) in Canada, across all provinces, is currently Bendamustine plus Rituximab.  But all WMers are unique and different.  Your oncologist may recommend other approaches, based on your individual symptoms and circumstances.  Below, we provide an overview of the breadth of treatments available today for WM.  Plus, there are a few words about Imbruvica (Ibrutinib).

More information can be obtained by visiting the various subsections in Education on our website.

Treatment options for Waldenstrom’s macroglobulinemia may include the following:

    If IgM proteins are found in your blood, but you don’t have any signs or symptoms, you may choose to wait before beginning treatment. Your doctor may recommend blood tests every few months to monitor your condition. You may go years without needing further treatment. This is called Watch and Wait.
    Such as transfusions or growth factors to boost red blood cells, white blood cells, and platelets;

    If you experience signs and symptoms related to having too many IgM proteins in your blood, your doctor may recommend plasma exchange (plasmapheresis) to remove the proteins and replace them with healthy blood plasma.
    is a drug treatment that kills quickly growing cells, such as the abnormal blood cells produced by Waldenstrom’s macroglobulinemia.

    Chemotherapy may be used alone or combined with other drug treatments as an initial treatment for people who experience signs and symptoms of Waldenstrom’s macroglobulinemia. High-dose chemotherapy may also be used to suppress your bone marrow production in preparation for a bone marrow transplant. Chemotherapy usually includes alkylating agents such as chlorambucil, cyclophosphamide, and bendamustine or with nucleoside analogs such as fludarabine and cladribine.

    Targeted therapy drugs kill cancer cells by focusing on the specific abnormalities present in the cancer cells that allow them to survive.  For WM, typical targeted therapy drugs include ibrutinib and everolimus, which target B-cell signalling pathways.  Targeted therapy drugs may be used alone or combined with other medications, such as chemotherapy or biological therapy.  They can be used, alone or in combination, as an initial treatment for Waldenstrom’s macroglobulinemia or in cases where the cancer returns despite treatment.

    Biological therapy drugs use your immune system to kill cancer cells. Biological therapy drugs can be used alone or in combination with other medications as an initial treatment or as a treatment for recurrent Waldenstrom’s macroglobulinemia.  The most common biological therapy drugs are monoclonal antibodies such as rituximab and ofatumumab.

    In certain highly selected situations, a bone marrow transplant, also known as a stem cell transplant, may be used to treat Waldenstrom’s macroglobulinemia. There are two types of bone marrow transplants. Autologous, where the patient’s own stem cells are removed before receiving high-dose chemotherapy or radiation treatment. (Some times the stem cells are treated before removal). After these treatments are done, the cleaned stems cells are given back to the patient. The second is the Allogeneic or Donor bone marrow transplant, where stem cells are removed from another person, called a donor. Most times, the donor must have the same or similar genetic makeup as the patient, so that he or she is a “match” to the recipient. During this procedure, a lighter dose of chemotherapy is used to wipe out your diseased bone marrow. Healthy donor blood stem cells are infused into your body where they can rebuild healthy bone marrow.

    Clinical trials give you a chance to try the latest in Waldenstrom’s macroglobulinemia treatment. Participation in a clinical trial can actually be the best or even the only option available – and should be seriously considered by the patient.

    Including prednisone and dexamethasone
    Including thalidomide and lenalidomide.
    Such as bortezomib and carfilzomib

A few words about Imbruvica (Ibrutinib)

Imbruvica (ibrutinib) was approved by the US Food and Drug Administration in 2015 as the first drug specifically designated for the treatment of WM.  It has since been approved by Health Canada and the European Medicines Agency for use in the European Union.

Imbruvica is a daily oral drug that targets the Bruton’s tyrosine kinase (BTK) pathway in B-cell growth and development.  While it is a very important step forward in treatment, it is not a cure for WM and not everyone responds to it.  Many of the older, established treatments are still appropriate for WM patients, and there are several new treatments still in the research pipeline that may achieve FDA and Canadian approval as well.

Note that approval by Health Canada does not mean that it is automatically funded in provincial drug plans.  It is typically not used as a first-line treatment in Canada.

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