Patients identify efficacy as the highest priority for the treatment of Waldenström’s macroglobulinemia


The findings may have important implications for shared decision-making between providers and their patients, as well as for future trials of treatments for the disease.

For the first time, researchers have collected and analyzed systematic data on treatment preferences for Waldenström’s macroglobulinemia, uncovering the main factors in patients’ treatment choices.

According to the researchers, their findings may have important implications for shared decision-making between providers and their patients, as well as for future trials of disease treatments.

“A better understanding of patient treatment preferences can help physicians and patients make shared decisions,” the researchers wrote. “Patients who are involved in clinical decision-making are more likely to express satisfaction with therapy that leads to better adherence. For example, data on preferences can provide valuable information from the patient’s perspective to help select clinical trial outcomes that might otherwise be overlooked.

Among the 214 respondents to the discrete-choice experiment questionnaire, progression-free survival (PFS) was the most important factor identified by patients in deciding. For example, the probability of choosing a treatment increased by 26% if the PFS at 5 years increased from 50% to 70%. Patients indicated a willingness to trade efficacy for certain attributes, including no increased risk of secondary malignancy (-16.3% efficacy; 95% CI 16.1% at 16 .5%) and treatment containing targeted therapy (-5.8%; 95% CI, 5% to 6.4%).

When it comes to safety, patients said neuropathy was the side effect they most wanted to avoid, indicating a willingness to trade off 6.5% efficacy to avoid neuropathy. The researchers highlighted this finding, noting the importance of rapid dose reduction in patients with neuropathy due to proteasome inhibitors and for consideration of less toxic options.

Patients also expressed a preference for treatment administered intravenously (IV) or subcutaneously (SC) in the clinic.

“It may seem surprising that patients prefer IV/SC administration in hospital to oral intake at home, but this could be explained by the fixed duration of IV/SC treatment, which patients seem to value most” , the researchers explained. test the ‘duration of treatment’ attribute separately since the oral treatments currently available for WM do not have a fixed duration.

The researchers found that patients’ preferences were not statistically different based on gender, age, or whether they had been treated before. Education level influenced patient preferences, with a slight difference observed in preference for adverse events.

The researchers noted that their survey results, posted on a patient organization’s website, may have selection bias, as they may have only reached a certain subset of patients.


Amaador K, Nieuwkerk, Minnema M, Kersten M, Vos J. Patient preferences regarding treatment options for Waldenstrom’s macroglobulinemia: a discrete choice experiment. CancerMed. Published online July 26, 2022. doi:10.1002/cam4.5080


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