Study Investigates Patient Preferences in Waldenström Macroglobulinemia Treatment

A mixed-method study of Dutch patients, presented at the 63rd American Society of Hematology Annual Meeting & Exhibition, examined treatment preferences among patients with Waldenström macroglobulinemia (WM).

This study comprised a literature review, qualitative interviews, and expert discussions. A discrete choice experiment questionnaire was developed to assess treatment-related characteristics, including:

  • five-year progression-free survival (PFS)
  • frequency and type of administration (intravenous, subcutaneous, or oral)
  • treatment setting (clinic or home)
  • adverse events
  • risk of future secondary malignancies
  • type of treatment agent (chemotherapy or targeted therapy)

Respondents were presented with two hypothetical, but realistic, treatment options and asked to choose between them. Questionnaires were disseminated nationally either online, via a patient organization’s website, or paper assessments completed at outpatient clinics.

In total, 330 online and 17 paper-based questionnaires were completed; 214 (65%) had complete data and were included in the analysis. The average age of respondents was 66 years, and 54% of the cohort were male. Average time since diagnosis was 6.9 years. Regarding disease status, 31% were in the wait-and-see period, 39% were in remission, 10% had progressive disease, and 20% were currently being treated.

Respondents rated five-year PFS as the most important attribute for making treatment decisions. The probability of choosing a treatment option increased by 26% if the five-year PFS rate rose from 50% to 70%. The risk of secondary malignancies was the second most highly rated attribute. Selection of a treatment rose by 22% if the chance of secondary malignancy decreased from high to low. Risk of neuropathy was a greater consideration compared to nausea, vomiting, and extreme fatigue. To avoid risk of neuropathy, patients were willing to accept a 7.2% reduction in treatment efficacy. Selection of a treatment was 8% more likely with a fixed-duration, intravenous/subcutaneous administration at a hospital compared to an ongoing oral regimen at home.

The authors reported that patients’ demographic characteristics and treatment status were not significantly associated with treatment preferences. However, educational status was the one exception: patients with a higher education status were more accepting of a treatment with a lower five-year PFS than patients with lower education levels (p < 0.0001). Those with higher education tended to prefer targeted therapy and treatments with low risk of secondary malignancy (p < 0.0001).

“These are the first data on WM patients’ preferences on treatment characteristics,” wrote the authors in conclusion. “These data can be used in discussions with individual patients about their treatment preference and help direct future clinical trials that optimally connect to WM patients’ preferences.”