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

For Waldenström macroglobulinemia (WM), there is currently no uniform 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 among patients with WM from 5 high-income countries. The DCE, implemented via a web-based survey, comprised 16 choice sets requiring participants to select between 2 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 1455 patients with WM participated, with 75% aged 61 to 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 less impact on decision-making, although there was a preference for oral, fixed-duration therapy. In conclusion, patients with WM prioritize longer response durations 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 health care resources and infrastructure.