探索复发/难治性多发性骨髓瘤治疗中患者最看重的因素：一项针对患者、照护者与医生的加拿大离散选择实验

Relapsed/refractory multiple myeloma (RRMM) significantly impacts patients' quality of life. This study investigated treatment characteristic preferences for RRMM in Canada, comparing attributes of conventional treatments to those of Chimeric antigen receptor (CAR) T-cell therapy. It aimed to understand patient preferences and how caregivers and healthcare professionals (HCPs) perceive these preferences.

A discrete choice experiment (DCE) was conducted with 101 RRMM patients, 46 caregivers, and 49 HCPs. Five attributes were explored: progression-free survival (PFS), 10-year survival chance, risk of serious side effects, risk of long-term moderate side effects, and treatment administration. Participants were asked to choose between two hypothetical treatments in a series of 12 questions. Hypothetical treatments included CAR-T-administered treatments and alternative treatments administered by conventional means ("conventional treatments") with varying levels of performance across remaining attributes.

Prolonged PFS and 10-year survival chance were the most critical factors in treatment decisions across all groups. Long-term moderate side effects were more concerning than serious short-term side effects, while treatment administration was least important. Patients were willing to accept higher side effect risks for improved survival. High agreement was found between patients' preferences and caregivers' and HCPs' perceptions of these preferences.

While participants showed no significant preference between the administration profiles of CAR-T therapy and conventional treatments, CAR-T therapy was favored for its clinical benefits of potentially increased survival and lower long-term side effect risks. These findings are valuable for Canadian patient-centered clinical and reimbursement decision-making, highlighting the potential role of CAR-T therapy in addressing patient needs.