Waldenstrom macroglobulinemia (WM) is a rare type of cancer that affects the immune system. There are numerous treatments that may help, such as chemotherapy, immunotherapy, targeted drug therapy, and more.

Waldenstrom macroglobulinemia (WM) is a cancer that starts in cells known as lymphocytes, which are part of the immune system.

It is a type of non-Hodgkin lymphoma that some people call lymphoplasmacytic lymphoma or simply Waldenstrom’s.

This article looks at the treatments doctors may recommend for WM. It also discusses the effectiveness of WM treatments, how to choose the right one, and more.

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If a person with WM is asymptomatic, which means they do not have any symptoms, doctors may recommend closely observing the condition and following up with the individual every 1–2 months.

During those appointments, the doctor may order tests to assess the person’s blood and antibodies.

Biological therapy helps the body’s immune system fight the cancer.

Doctors may use the anti-CD20 monoclonal antibody drug rituximab alone or in combination with chemotherapy. Research indicates that monoclonal antibody-based therapy plays an important role in treating WM. Monoclonal antibodies are artificial versions of the proteins that the body uses to fight infections.

The American Cancer Society (ACS) advises that, while doctors mostly use immunomodulating drugs such as thalidomide and pomalidomide for multiple myeloma, they may also help treat WM.

Because of concerns about congenital disabilities if a person takes these drugs during pregnancy, people can only obtain them through special programs that the drug companies run. Side effects can include severe blood clots, neuropathy, and constipation.

Interferon is a lab-made drug consisting of hormone-like proteins known as cytokines. The ACS explains that some studies suggest interferon can help tumors shrink, and doctors mostly use it for people who don’t respond to other treatments.

Doctors may recommend chemotherapy in cycles for WM. Each cycle typically lasts for several weeks.

A doctor may recommend oral medications, or they may inject anticancer drugs into a vein, muscle, or under the skin. A person with WM may receive chemotherapy drugs in combination with targeted or immunotherapy medications.

Types of chemotherapy medications for WM include:

  • alkylating agents such as bendamustine (Treanda) and cyclophosphamide (Cytoxan)
  • corticosteroids such as dexamethasone (Decadron) and prednisone
  • purine analogs such as cladribine (2-CdA, Leustatin) and fludarabine (Fludara)

The side effects of chemotherapy may include nausea and vomiting, hair loss, and fatigue. Long-term side effects include the possibility of developing leukemia at a later stage.

Learn more about chemotherapy.

Targeted therapy drugs treat the cancerous changes that happen inside cells. They may target proteins or enzymes that help the cancer to grow.

Sometimes these drugs work when chemotherapy is unsuccessful. The drugs include:

  • bruton tyrosine kinase (BTK) inhibitors such as zanubrutinib (Brukinsa) and Ibrutinib (Imbruvica)
  • proteasome inhibitors such as carfilzomib (Kyprolis) and bortezomib (Velcade)
  • mTOR inhibitors such as everolimus (Afinitor)

Side effects may include muscle and bone pain, low blood counts, or serious infections.

Radiation therapy involves high-energy rays that kill cancer cells. Doctors rarely use this treatment for WM, but it may be helpful to shrink enlarged lymph nodes or spleen if they cause symptoms.

Although painless, the procedure may cause side effects such as sunburn-like skin problems, low blood counts, and fatigue.

Plasma exchange, or plasmapheresis, is a procedure in which a machine separates the liquid part of the blood (plasma) that contains abnormal cells, discards it, and then mixes it with blood cells from a donor. A doctor then returns the blood cells to a person with WM through an intravenous line.

This type of treatment can help to thin blood so that it flows more easily through the vessels. This may be useful if a person has a large amount of immunoglobulin M (IgM) that causes their blood to become too thick.

The procedure can take 2–3 hours to remove and return the blood to the person. If calcium levels drop, the person may have numbness and tingling, which doctors address by giving calcium.

Learn more about plasmapheresis.

Stem cell transplant is uncommon for WM, but doctors may recommend it for younger people whose treatment has been unsuccessful.

Autologous stem cell transplants involve taking stem cells from the blood or bone marrow of the individual receiving the treatment. Allogeneic stem cell transplants involve taking the stem cells from a donor.

Allogeneic transplants have higher risks and side effects than autologous stem cell transplants. One of the most severe side effects of a donor transplant is when the immune system of the person with WM believes the tissues to be foreign and attacks them, which can be life threatening.

Numerous medical professionals may be involved in the treatment of WM. These include:

  • a hematologist, who specializes in blood disorders
  • a medical oncologist, who treats cancer with chemotherapy and other drugs
  • a radiation oncologist, who treats cancer with radiation therapy

A person can discuss treatment options, goals, and potential side effects with their healthcare team to decide on which treatments are right for them.

Their doctor will help them to understand the information about the treatments and answer any questions they may have. Sometimes, people may seek a second professional opinion about which treatment option is suitable.

Treatment success may depend on the individual and the stage of cancer. According to a 2023 article, about 4 in 10 people with WM survive for 10 years or more.

It is important to note that the outlook will be different for each individual. A person’s doctor can provide them with more accurate information about how successful they expect treatment to be on the basis of the individual’s circumstances.

There are numerous possible treatments for Waldenstrom macroglobulinemia (WM). If a person has no symptoms, doctors may observe them closely, monitoring them with tests.

However, for people with symptoms, treatment for WM can include chemotherapy, targeted drug therapy, immune-modulating drugs, or plasma exchange.

It is best for a person to discuss treatment options with their healthcare team to understand what the best decision is for them.