Survival, Symptoms and Support in Waldenström Macroglobulinemia

Waldenström macroglobulinemia (WM) is often slow growing, with many patients experiencing no symptoms for years, according to Alisha Kimble, senior outcomes researcher at Ontada.

A study which Kimble conducted demonstrated a five-year survival rate of 78% among this patient population; however, quality of life can decline over time due to chronic conditions. Early use of risk scoring tools, like the Revised International Prognostic Scoring System, can help guide treatment decisions. The study also confirmed that patients treated in U.S. community clinics received care aligned with national guidelines, thanks to built-in clinical pathways in electronic records. Patients in underserved areas had worse outcomes, highlighting the need for early support.

Kimble encouraged newly diagnosed patients to ask about risk scoring and use trusted resources to stay informed. She also noted a need for more clinical trials in community settings, where over 2,500 patients with WM were treated in the past decade.

CURE: What do patients with WM need to know about the natural course of this disease, based on what your study found?

Kimble: Typically, this disease is indolent in nature, meaning patients may not have symptoms for the first few years before requiring treatment. This makes it difficult to identify, which is why research like this is so important — not only because the disease is hard to detect, but also because it can be challenging to find a large enough patient population to allow for statistically meaningful analysis. That’s what adds value to this work.

Some other key things for patients to know: this disease tends to have high survival rates. We identified a five-year survival rate of about 78%, which aligns with SEER estimates from the American Cancer Society and WHO estimates of about 76%. It’s higher than some other rare diseases. However, patients often experience a lower quality of life due to chronic conditions that develop over time, which can offset that long-term survival.

Another key takeaway is the use of a risk prognostic tool. For Waldenström, there’s the Revised International Prognostic Scoring System and a modified version — both are risk calculators that use lab values to assess disease severity. If patients receive one of these risk scores early in the clinic, it can help predict the course of the disease and assist physicians in guiding treatment.

How does the modified staging system for WM help patients and care teams better understand prognosis or guide treatment decisions?

It’s a newer prognostic tool, but our research was able to externally validate its use in a large population. We analyzed about 917 patients who had all necessary lab values to derive their risk scores and assess severity. What we found was that patients with higher risk scores had worse outcomes than those with lower scores. We confirmed these associations through multivariate analysis. So overall, this tool can help identify disease severity early, inform treatment patterns, and guide clinical decision-making.

Your study showed that patients received guideline-recommended treatments. What does this mean for those being treated outside major cancer centers?

Yes. This study was unique because patients were assessed within U.S. community oncology clinics — outpatient settings that are different from larger academic or hospital-based institutions. We wanted to highlight both the depth of data in these settings and the breadth of patients receiving care there.

The data came from the iKnowMed EHR system used across the U.S. Oncology Network. One unique feature of this system is the inclusion of clinical pathways — evidence-based tools built into the EHR. When a provider enters a patient’s diagnosis and staging information, the system suggests treatment options aligned with NCCN guidelines and established research. That ties into our results: not only were physicians treating patients appropriately, but patients were also receiving guideline-recommended care based on national standards.

Could you explain how certain social and economic factors affected outcomes in your study, and what patients can do if they're concerned about these barriers?

Yes. We conducted a multivariate analysis to assess associations between patient characteristics and outcomes. One important factor we found was the Area Deprivation Index, which is based on patients’ zip codes. Patients living in more deprived or rural areas had worse outcomes than those in less deprived areas.

This is really about spreading awareness. If a provider can identify that a patient lives in a rural or underserved area — where there may be food deserts, transportation barriers, or limited access to care — they might be able to offer more support from the beginning. Early identification of these social challenges can help ensure that patients receive the follow-up care they need.

What are the most important things a newly diagnosed patient with WM should discuss with their doctor in light of your study’s findings?

Two of the most important things are, first, the use of the risk prognostic tool to assess disease severity early. That can help guide treatment decisions and potentially improve outcomes.

Second, because this is a rare and often misunderstood disease, it’s important for patients to advocate for themselves and stay informed. Resources like the International Waldenström Foundation and the national patient registry can help patients connect with others who have been diagnosed. The disease can present differently in different people, so doing your own research and learning from others can be very helpful. Again, understanding and using a risk score early on is also key.

Were there any findings in your research that surprised you or deserve more attention from the patient community?

Not necessarily surprising, but something important we found was that more than 2,500 patients were diagnosed with Waldenström macroglobulinemia over the past 10 years within U.S. community oncology settings. There aren’t many clinical trials in these environments, so our findings show a large patient population with a potential unmet need. This opens the door for more clinical trials to take place closer to where patients live, instead of requiring them to travel to major academic centers. It’s an opportunity to bring research to more patients

and inform future drug development by focusing on this underserved group.

Transcript has been edited for clarity and conciseness

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