Patients Identify Efficacy as Biggest Priority for Waldenström's Macroglobulinemia Treatment

The findings can 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 preferences for Waldenström's macroglobulinemia treatment, uncovering the biggest drivers of treatment choice for patients.

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

“A better understanding of patient treatment preferences can aid physicians and patients in shareddecision-making,” wrote the researchers. “Patients who are involved in clinicaldecision-making are more likely to express satisfactionwith therapy which leads to improved adherence.Inaddition, better insight in patients' treatment views couldhelp direct future clinical trials in WM. For instance,preference data can provide valuable information froma patient's perspective to help select clinical trial outcomes that may otherwise be overlooked.”

Among the 214 respondents of the discrete choice experiment questionnaire, progression-free survival (PFS) was the most important factor identified by patients for deciding on. For example, the probability of choosing a treatment increased by 26% if the 5-year PFS increased from 50% to 70%. Patients indicated a willingness to trade efficacy for certain attributes, including no increased risk of a secondary malignancy (−16.3% efficacy; 95% CI, 16.1% to 16.5%) and a treatment containing targeted therapy (−5.8%; 95% CI, 5% to 6.4%).

When it came to safety, patients expressed neuropathy as the side effect they want to avoid most, indicating a willingness to trade 6.5% efficacy to avoid neuropathy. The researchers highlighted this finding, noting the importance of timely dose reduction in patients who experience neuropathy from proteasome inhibitors and for the consideration of less toxic options.

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

“It may seem surprising thatpatients would prefer IV/SC administration at hospitalover oral intake at home, but this could be explained bythe fixed duration of the IV/SC treatment, which patientsseem to value the most,” explained the researchers.“We did not test the attribute ‘duration of treatment’ separately since the oral treatmentscurrently available for WM do not have a fixed duration.Interestingly, fixed-duration treatments for WM usuallycontain cytotoxic drugs that predispose for secondary malignancies, a risk that respondents also wanted to avoid.”

The researchers found that patient preferences were not statistically difference based on gender, age, or whether they had been previously treated. Education level did influence patient preferences, with a slight difference observed in the preference of adverse events.

The researchers noted that the findings of their survey, disseminated on a website of a patient organization, could have selection bias as it may have reached just a certain subset of patients.


Amaador K, Nieuwkerk, Minnema M, Kersten M, Vos J. Patient preferences regarding treatment options for Waldenström's macroglobulinemia: A discrete choice experiment. Cancer Med. Published online July 26, 2022. doi:10.1002/cam4.5080

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