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Innovative Treatment Options for Waldenstrom

Innovative Treatment Options for Waldenstrom
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Published on June 23, 2020

Innovative Treatment Options for Waldenstrom

Waldenstrom macroglobulinemia (WM) is not only a mouthful to say, but it's also a rare type of non-Hodgkin lymphoma (NHL). Approximately 1,500 patients are diagnosed each year in the United States. Also called lymphoplasmacytic lymphoma, this condition occurs when cancer cells make large amounts of an abnormal protein, called a macroglobulin, which pushes out healthy blood cells.

So, what’s the best treatment for this rare disease?

For patients who prefer, or cannot tolerate, IV chemotherapy, an oral pill called ibrutinib (Imbruvica) has been a good option for many, and several new therapies are in clinical trials.

Two Therapies Go Head to Head

A recent randomized Phase III study compared two Bruton’s tyrosine kinase (BTK) inhibitors, zanubrutinib (Brukinsa), and ibrutinib. Here’s the breakdown:

BTK inhibitors slow down the enzyme BTK, which is a crucial part of the B-cell receptor signaling pathway. Certain B-cell lymphomas use B-cell receptor signaling for growth. In the Phase III study, 201 patients were randomly selected to participate from January 2017 to July 2018. Side effects included rates of atrial fibrillation — a heart arrhythmia sometimes call aFib — bruising, diarrhea, swelling, hemorrhage, muscle spasms, and pneumonia, but were less in the zanubrutinib group.

Although not statistically significant, zanubrutinib was associated with a higher complete response (CR), or very good partial response (VGPR) than ibrutinib. The verdict is still out, however, on whether zanubrutinib will lead to longer progression-free survival (PFS). Follow up studies are needed.

Treatment strategies for WM are personalized and vary by symptoms and which side effects a patient can tolerate, according to Dr. Larry Anderson Jr., Associate Professor in UT Southwestern Medical Center's Department of Internal Medicine, Division of Hematology/Oncology.

“Some patients with Waldenstrom's may have a smoldering disease that's sort of like a precancer where they have a detectable disease in the bone marrow, but no symptoms. And the symptoms that we look for that would require treatment would be anemia, large spleen, something called hyperviscosity,” he said.

Hyperviscosity happens when blood isn't able to flow freely through your arteries. Arterial blockages can happen when you have too many red blood cells, white blood cells, or proteins in your bloodstream. Having the right expert in your corner is important to help you understand this condition.

To learn more from Dr. Anderson, watch Waldenstrom Macroglobulinemia: Symptoms and Treatment.

Find an Expert in WM

With any cancer diagnosis, it’s important to consult a specialist. This becomes increasingly important when you’re diagnosed with a rare disease like Waldenstrom. Chances are, your local oncologist has seen few if any, cases.

Dr. Sikander Ailawadhi, a hematologist/oncologist from the Mayo Clinic, says it’s critical for those diagnosed with this rare disease to consult a Waldenstrom expert for help. He encourages collaboration between academic centers and community doctors to ensure patients receive the best care possible and says most physicians are very comfortable with their patients getting a second opinion.

“We expect our patients to seek out the right care for themselves,” he said.

You want a physician who understands the rarity and specific needs of this condition and how to help you determine the next steps, whether that is a watch and wait for approach or seeking treatment soon after diagnosis. Whatever your approach, Patient Power is here for you.

To learn more from Dr. Ailawadhi about working with a Waldenstrom expert and how to find one, watch Waldenstrom Diagnosis: Consulting a Specialist for Help.

Looking for more information about Waldenstrom Macroglobulinemia? Please sign up for e-news and we’ll send it directly to your inbox.

~Lauren Evoy Davis


Constantine S, et al. ASPEN: Results of a phase III randomized trial of zanubrutinib versus ibrutinib for patients with Waldenström macroglobulinemia (WM). J Clin Oncol 38: 2020 (suppl; abstr 8007)


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