Hall Rebekah, Chua Gin Nie, Macfarlane Bonnie, Plante Richard K, Shi Kun, Soltys Stephanie, McCurdy Arleigh, Elias Martine, Ranger Jessy
Acaster Lloyd Consulting Ltd, London, UK.
Johnson & Johnson, Ottawa, Canada.
BMC Cancer. 2025 Jul 1;25(1):1095. doi: 10.1186/s12885-025-14490-5.
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.
复发/难治性多发性骨髓瘤(RRMM)对患者的生活质量有重大影响。本研究调查了加拿大RRMM患者的治疗特征偏好,比较了传统治疗与嵌合抗原受体(CAR)T细胞疗法的属性。其目的是了解患者的偏好以及护理人员和医疗保健专业人员(HCPs)如何看待这些偏好。
对101名RRMM患者、46名护理人员和49名HCPs进行了一项离散选择实验(DCE)。探讨了五个属性:无进展生存期(PFS)、10年生存机会、严重副作用风险、长期中度副作用风险和治疗给药方式。在一系列12个问题中,要求参与者在两种假设治疗方案之间进行选择。假设治疗方案包括CAR-T给药治疗和通过传统方式给药的替代治疗方案(“传统治疗”),在其余属性方面具有不同的表现水平。
延长的PFS和10年生存机会是所有组治疗决策中最关键的因素。长期中度副作用比严重短期副作用更令人担忧,而治疗给药方式最不重要。患者愿意为提高生存率接受更高的副作用风险。患者的偏好与护理人员和HCPs对这些偏好的看法之间存在高度一致性。
虽然参与者在CAR-T疗法和传统治疗的给药方式上没有表现出明显的偏好,但CAR-T疗法因其潜在提高生存率和降低长期副作用风险的临床益处而受到青睐。这些发现对于以加拿大患者为中心的临床和报销决策具有重要价值,突出了CAR-T疗法在满足患者需求方面的潜在作用。