Ailawadhi Sikander, Biru Yelak, Clavreul Solène, San Miguel Maite, Cormier Nicolas, Efebera Yvonne, Merz Maximilian, Sato Anna, Zeanah Cathy, Watkins Jack L, Farrell James, Goldman Erinn Hoag, Popat Rakesh
Division of Hematology and Medical Oncology, Mayo Clinic, Jacksonville, FL, USA.
International Myeloma Foundation, Studio City, CA, USA.
Patient Prefer Adherence. 2025 Apr 16;19:1089-1104. doi: 10.2147/PPA.S496106. eCollection 2025.
With novel therapies including chimeric antigen receptor T-cell (CAR-T) therapy and bispecific antibodies (BsAbs), healthcare providers (HCPs) face complexities managing treatment for patients with relapsed/refractory multiple myeloma (RRMM). This study, among the largest surveys on RRMM, examined unmet needs in care access, barriers to novel therapy use, and treatment decision-making.
This survey-based study (March-June 2024) enrolled 2284 participants (patients: 1301; HCPs: 983) across 7 countries. Patients with >1 relapse/progression and HCPs managing ≥3 patients were included. Data were analyzed using descriptive statistics and Χ tests.
For patients, treatment priorities included slowing disease progression (second line [2L], 47%; third or later line [≥3L], 49%), minimizing adverse events (AEs; 2L, 43%; ≥3L, 49%), and extending life (2L, 39%; ≥3L, 38%). HCPs prioritized prolonging survival and controlling disease. Younger patients (<65 vs ≥65 years) prioritized convenience (40% vs 24%; <0.01) and avoiding referrals to new institutions for therapies (32% vs 20%; <0.01). Across geographies, HCPs reported logistical challenges as key reasons that CAR-T (38%) or BsAb (34%) therapy was not offered. Novel therapies were offered to patients more frequently in the US vs EU (CAR-T, 84% vs 77%, =0.023; BsAbs, 84% vs 76%, =0.011), with a similar trend in the US vs Japan for CAR-T; however, across all geographies, few patients recalled being offered CAR-T (17%) or BsAbs (13%). Patients receiving BsAbs prioritized efficacy-related reasons (25-35%) and nonclinical factors like less time and financial impact (27-29%), whereas those who received CAR-T prioritized patient success stories (50%), efficacy-related factors (48-50%), and minimal financial burden (43%).
This study revealed gaps in treatment priorities; patients valued quality of life and AE management, while HCPs focused on efficacy and delaying progression. There is a significant need to educate HCPs and patients on the impact of shared decision-making when considering novel treatments for RRMM.
随着嵌合抗原受体T细胞（CAR-T）疗法和双特异性抗体（BsAbs）等新型疗法的出现，医疗服务提供者（HCPs）在管理复发/难治性多发性骨髓瘤（RRMM）患者的治疗方面面临复杂性。这项在RRMM领域规模较大的调查研究，探讨了护理获取方面未满足的需求、新型疗法使用的障碍以及治疗决策过程。
这项基于调查的研究（2024年3月至6月）在7个国家招募了2284名参与者（患者：1301名；HCPs：983名）。纳入了有超过1次复发/进展的患者以及管理≥3名患者的HCPs。使用描述性统计和卡方检验对数据进行分析。
对于患者而言，治疗重点包括减缓疾病进展（二线[2L]，47%；三线或更后线[≥3L]，49%）、将不良事件（AEs）降至最低（2L，43%；≥3L，49%）以及延长生命（2L，39%；≥3L，38%）。HCPs则将延长生存期和控制疾病作为优先事项。较年轻的患者（<65岁与≥65岁）更看重便利性（40%对24%；<0.01）以及避免因治疗转诊至新机构（32%对20%；<0.01）。在不同地区，HCPs报告称后勤方面的挑战是未提供CAR-T（38%）或BsAb（34%）疗法的关键原因。在美国，与欧盟相比，新型疗法向患者提供的频率更高（CAR-T，84%对77%，P = 0.023；BsAbs，84%对76%，P = 0.011），在美国与日本之间，CAR-T也有类似趋势；然而，在所有地区，很少有患者回忆起曾被提供过CAR-T（17%）或BsAbs（13%）。接受BsAbs治疗的患者将疗效相关原因（25 - 35%）以及诸如时间和经济影响较小等非临床因素（27 - 29%）作为优先考虑因素，而接受CAR-T治疗的患者则将患者成功案例（50%）、疗效相关因素（48 - 50%）以及最小经济负担（43%）作为优先考虑因素。
这项研究揭示了治疗重点方面的差距；患者重视生活质量和不良事件管理，而HCPs则关注疗效和延缓疾病进展。在考虑为RRMM患者采用新型治疗方法时，非常有必要就共同决策的影响对HCPs和患者进行教育。
