Abstract Topic: 14. Myeloma and other monoclonal gammopathies - Clinical
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.
Aims: This study aimed to gain insights into perspectives of patients with MM on BCMA-targeted therapies. By understanding patient preferences, values, knowledge gaps and willingness to try new therapies, healthcare providers can better support patients in making informed treatment decisions.
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 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. When two therapies with the same efficacy and duration of response were offered, 69% of patients would prefer the therapy with lower risk of severe SEs but requires continuous dosing with no treatment-free interval, as opposed to the therapy that is given once followed by a treatment-free interval but with a potentially higher risk of severe SEs (31%).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.
Summary/Conclusion: 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.
Keywords: B-cell maturation antigen, Multiple myeloma, Bispecific, CAR-T