Assistant Professor Moffiitt Cancer Center Tampa, Florida, United States
Introduction: Multiple myeloma (MM) is a generally incurable, heterogeneous disease affecting >35,000 patients annually in the United States (US). Chimeric antigen receptor T-cell (CAR-T) therapy, a novel treatment with demonstrated depth of response and durability, is now approved for patients as early as 2nd line (2L). Recent data from CARTITUDE-4 and KarMMa-3 show meaningful benefits in clinical and health-related quality of life metrics over current options for patients in earlier lines. However, patient preferences for the use of CAR-T in early lines remain unclear. Therefore, the objective of this study was to evaluate patient preferences for early-line CAR-T therapy use in MM.
Methods: Patients with relapsed or refractory MM from the US, who had completed ≥1 line of treatment, were recruited via convenience sampling to participate in a cross-sectional survey between December 2023-March 2024. The survey included a Discrete Choice Experiment (DCE) comprised of a series of choice tasks in which patients chose between 2 hypothetical 2L treatment profiles. These profiles varied on 7 treatment attributes: median progression free survival (mPFS), median overall survival (mOS), treatment response, serious adverse events (SAEs), neurological events, cytokine release syndrome (CRS), and treatment administration (i.e., one-time infusion vs. various routes and schedules for agents). Attributes and levels were selected based on a targeted literature review, clinical data on standard 2L therapies in MM, including CAR-T, and insights gathered from patient focus groups. Patient characteristics were described, and attribute-level preference weights were estimated using hierarchical Bayesian modeling.
Results: The sample comprised 127 patients with a mean age of 67 years. Most patients were female (54%), White (61%) and on active MM treatment (96%). The majority had received 2 lines of therapy (45%), mean time since MM diagnosis was 71 months, and nearly 10% received CAR-T. Patients’ preferences were most influenced by extending mOS from 2 to 6 years, increasing mPFS from 7 months to 4 years, reducing CRS events from 95% to 0%, and decreasing SAEs from 73% to 28%. In contrast, attributes such as neurological events (decreasing risk from 9% to 0%), and treatment response (increasing response rate from 55% to 95%) had less influence. While not the most influential factor, patients preferred a one-time infusion over other modes of administration (e.g., weekly or every other day injection).
Conclusions: This study indicates that patients’ treatment preferences were predominantly influenced by survival metrics in early lines of treatment for relapsed myeloma, suggesting a potential preference for CAR-T therapy given the demonstrated PFS benefit in as early as 2L+ patients in CARTITUDE-4. However, the safety profiles associated with more effective treatments like CAR-T should be clearly communicated, as the associated risks may influence preferences and treatment decisions.