Patient Preferences for Treatment in Relapsed/Refractory Acute Leukemia: A Multinational Discrete Choice Experiment

Acute leukemia (AL) is an aggressive cancer of white blood cells that progresses rapidly and requires intensive treatment. While first-line therapies can induce remission, this is not the case for all patients (refractory disease), and many patients experience a relapse. In these situations, treatment decisions become complex due to trade-offs between efficacy, toxicity and quality of life. Despite recognition of the importance of patient preferences in treatment decision-making, limited evidence exists on how individuals with AL value different treatment features.

A web-based discrete choice experiment (DCE) was conducted across the UK, USA, France, Germany and Italy to quantify treatment preferences among adults with AL. Attributes were identified through literature review, formative qualitative research and patient input. Each participant completed 12 choice tasks comparing hypothetical treatment profiles varying by chance of response, duration of response, quality of life during treatment and response and mode of administration. Data were analyzed using mixed logit and latent class models to estimate conditional relative attribute importance (RAI) scores, explore preference heterogeneity and assess trade-offs.

A total of 267 respondents completed the study. Across the full sample, the chance of response was the most influential treatment attribute (RAI=62%), followed by quality of life during response (14%), duration of response (9%), quality of life during treatment (8%) and mode of administration (7%). Three latent preference classes were identified: efficacy-focused (47%), convenience- and efficacy-focused (21%) and balanced decision-makers (32%). Respondents in the USA were more likely to belong to the convenience- and efficacy-focused group, suggesting that contextual factors influence preferences.

Patients with AL largely prioritize treatment efficacy but also value quality of life and convenience, indicating that treatment decisions are multifaceted and context dependent. Incorporating patient preferences into clinical development and health technology assessment can promote more person-centered approaches to care.