Patient Preferences on Clinical Decision-Making in Myeloma: A 747-Patient Survey

Introduction: The advent of many novel myeloma treatments has increased the complexity of decision making for doctors and patients. Multiple available treatment options (some longstanding and others newly approved) lead to improved surrogate outcomes such as progression-free survival (PFS) but have not been shown to extend overall survival (OS). To better understand the priorities that underpin myeloma patients' preferences and guide their treatment decisions, we surveyed patients with myeloma about four different treatment scenarios, each with similar OS but substantially different surrogate outcomes. Methods: The HealthTree® Cure Hub by the HealthTree Foundation is the largest online portal designed to help people with plasma cell dyscrasias navigate their disease. Using this platform, we conducted a 4-question survey to investigate patient preferences. Question 1 presented the choice of a 3 drug versus a 4 drug option for newly diagnosed myeloma. Both options provided patients with a similar quality of life and 4-year OS, but the 4-drug option improved PFS (84% vs 67% at 4 years, similar to the PERSEUS trial), with increased risk of infection and neutropenia. We did not use the term “PFS” but rather described the concept in simple terms. Question 2 assumed a good response to induction therapy and presented a choice of a one-time treatment to deepen remission (analogous to melphalan autograft), or a continuation of the induction treatment once weekly for 4 additional months. The one-time treatment had a high likelihood of causing 3 months of decreased quality of life due to short term side effects. This one-time treatment increased the PFS at 5 years from 65% to 81% with no change in 5 year OS (similar to the DETERMINATION trial). Question 3 presented a choice between 1 drug or 2 drug maintenance. The 2 drug option increased 3 year PFS from 65% to 75% (similar to the FORTE trial), with similar 3 year OS. The 2 drug option had greater risks and side effects, such as those seen with the addition of carfilzomib (increased blood pressure, fevers, and cardiac toxicity). Question 4 presented the option of a treatment for multiply relapsed disease that improved PFS by 2 months and increased the likelihood of side effects but did not improve OS. The IRB-approved survey was open from April-May 2024. Statistics were assessed descriptively with Chi squares performed for between-group comparisons. Results: 747 responses were obtained. The median age of participants was 66 years (interquartile range 60-71), and most participants were female (59%) and white (91%). Of those who provided complete diagnostic information (405/747), 83% have MM and 17% have MM precursor conditions. No statistically significant differences were seen between the responses of those of have MM and those who have MM precursors. Further subset analysis will be presented at the meeting. In the 3 drug versus 4 drug scenario, 51% of participants chose 4 drug induction, and 49% chose the three drug option. For the one-time consolidation versus 4 months of weekly therapy to deepen response, 47% of participants chose a one-time treatment with 3 months of side effects, and 53% of participants chose the 4-month treatment option. 84% of participants chose 1 drug maintenance with shorter PFS compared to just 16% who chose 2 drug maintenance, with greater risks and side effects but a 10-month longer PFS. When evaluating an option for multiply relapsed disease that improved PFS but had no impact on OS, 93% of participants chose not to receive this treatment. Conclusion: Our analysis demonstrates substantial heterogeneity in patient preferences and priorities when choosing between treatments that improve PFS but not OS. For induction regimens (triplet versus quadruplet) and the use of a one-time intervention analogous to transplant to deepen response, approximately half of patients chose each option in both cases. In comparison, when evaluating long-term maintenance therapy with 2 drugs vs a single drug, or when evaluating options for multiply relapsed disease that improve PFS but not OS, the vast majority of patients choose the less toxic option that offers similar OS, even when PFS is inferior. These results provide guidance to other patients, clinicians (especially when counseling patients), industry, and regulators on patient preferences for therapies in newly diagnosed myeloma, facilitating patient-centered clinical trial design and drug development in future.

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