Patient perspectives on BCMA-targeted therapies for multiple myeloma: A survey conducted in a patient advocacy group.
Authors: Ahlstrom Jennifer M. , Lin Dee , Sweeney Nathan W. , Wu Bingcao , Gries Katharine , Kim Nina , Patel Saurabh , Sborov Douglas W. , Berdeja Jesus G. , Anderson, Jr Larry D. , Huo Jinhai , Hydren Jay R 
Published: June 2023

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Background: The emergence of B-cell maturation antigen (BCMA) targeted therapies in multiple myeloma (MM) has led to improvements in clinical outcomes in patients with heavily pretreated disease. Little is known about patients’ knowledge, decision-making process, and openness to these novel therapies that include chimeric antigen receptor (CAR) T-cell therapy and bispecific antibodies. This study aims to understand patient perspectives on these BCMA-targeted therapies. Methods: This is a cross-sectional survey completed by patients with MM enrolled in HealthTree Cure Hub, an online portal for patients with plasma cell dyscrasias to help navigate their disease. An 18-question survey was developed by the research team, then reviewed and adjusted by HealthTree’s Patient Advocacy Panel and a physician panel. Responses were analyzed descriptively and reported in aggregate. Results: From 10/28/2022 to 1/12/2023, 325 patients with MM participated in the survey (mean age: 66±8 years; 54% female; 90% White; years since diagnosis: 6.6±4.7; lines of therapy: 3.0±2.6). In general, 26% of patients were open to trying a new therapy right away and 43% were open but would like more information on safety and efficacy. BCMA-targeted therapies were well known with 92% of patients having heard about them. Among respondents for each question, 65% were likely or very likely to try a CAR T-cell therapy if offered and 74% were likely or very likely to try a bispecific therapy; while 17% and 13%, respectively, needed more information to decide. The most requested domains of information were efficacy, side effects (SEs), eligibility, and administration process for both CAR T-Cell and bispecific therapies. "How soon can I receive it" was ranked higher for bispecific therapy while "where can I receive it" was ranked higher for CAR T-cell therapy, relatively. The top attributes of therapy profiles were prolonged life, improved quality of life, longer progression-free survival, and tolerable SEs. To receive an effective therapy, the top acceptable trade-offs included frequent monitoring of SEs and initiating a new drug in a hospital setting, while patients were least willing to compromise on caregiver burden. Additionally, the most acceptable SEs were those that were asymptomatic but would need routine monitoring to prevent serious complications, and those that were cosmetic but non-life-threatening. Conclusions: This study found a high level of openness in patients with MM to try BCMA-targeted therapies if offered. Information on efficacy, safety, availability, and eligibility may assist patients on their decision-making. Patients were willing to accept certain trade-offs for a treatment that could be clinically beneficial. Incorporating patients’ goals, values, and preferences alongside clinical factors and other considerations may further optimize treatment decisions and improve patient outcomes.