Treatment preferences in Waldenström macroglobulinemia: an international discrete choice experiment in 1455 patients

For Waldenström macroglobulinemia (WM), there is currently no uniform preferred therapeutic approach, with options differing in efficacy, administration, and toxicity profiles. Understanding patient preferences is essential for optimizing treatment strategies. The WM-VOICE study used a discrete choice experiment (DCE) to quantify treatment preferences in WM patients from five high-income countries. The DCE, implemented via a web-based survey, comprised 16 choice sets requiring participants to select between two hypothetical treatment options, characterized by varying properties. The properties and values (‘attributes and levels’) identified through patient interviews, included response duration (2, 4, 6, or 8 years), administration mode (hospital infusions or pills at home), treatment duration (6 months, 2 years, or ongoing), temporary toxicities (grade 0-1, 2, or 3-4), persistent toxicities (grade 0-1 or 2), and risk of secondary malignancies (increased or not). Repeated choice data were analyzed using a panel mixed logit model. A total of 1,455 WM patients participated; 75% aged 61-80 years, 56% male, and 79% previously treated. All attribute levels significantly influenced choices, with preferences consistent across all countries. Response duration and temporary toxicity emerged as the most influential attributes driving treatment preferences, followed by secondary malignancy and persistent toxicity . Administration mode and treatment duration had relatively little impact on decision-making, although there was a preference for oral, fixed-duration therapy. In conclusion, WM patients prioritize long responses and avoidance of severe toxicity, whereas mode of administration and treatment duration are less influential. These insights can support shared decision-making and inform drug development and clinical trial design in WM, within settings with similar healthcare resources and infrastructure.