CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Perspectives of Healthcare Providers and Patients with Relapsed/Refractory Multiple Myeloma on Treatment Priorities and Novel Therapies.
Perspectives of Healthcare Providers and Patients with Relapsed/Refractory Multiple Myeloma on Treatment Priorities and Novel Therapies.
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本研究揭示了治疗优先事项上的差异:患者重视生活质量和 AE 管理,而 HCP 则关注疗效和延缓进展。
随着CAR-T 细胞疗法和双特异性抗体(BsAb)等新疗法出现,医疗服务提供者(HCP)在管理复发/难治性多发性骨髓瘤(RRMM)患者治疗时面临更复杂的决策。本研究是规模最大的RRMM调查之一,考察照护可及性、使用新疗法的障碍及治疗决策中的未满足需求。
2024年3月至6月在7个国家开展问卷调查,共纳入2284人(患者1301人,HCP 983人)。纳入经历过超过一次复发/进展的患者,以及管理至少3名此类患者的HCP。采用描述性统计和χ²检验分析数据。
患者最关注的治疗目标包括减缓疾病进展(二线[2L] 47%,三线及以后[≥3L] 49%)、尽量减少不良事件(AE;2L 43%,≥3L 49%)和延长生存(2L 39%,≥3L 38%)。HCP则优先考虑延长生存和控制疾病。与≥65岁患者相比,较年轻患者(<65岁)更重视治疗便利性(40%比24%,P<.01)和避免为治疗转诊至新机构(32%比20%,P<.01)。在不同地区,HCP均将物流困难列为未提供CAR-T(38%)或BsAb(34%)治疗的主要原因。美国向患者提供新疗法的比例高于欧盟(CAR-T 84%比77%,P=.023;BsAb 84%比76%,P=.011);美国与日本相比,CAR-T 也呈类似趋势。然而,各地区回忆曾获提供CAR-T 或BsAb的患者比例均较低,分别为17%和13%。接受BsAb的患者优先考虑疗效相关因素(25%–35%),以及治疗时间较少、经济影响较小等非临床因素(27%–29%);接受CAR-T 者则重视患者成功案例(50%)、疗效因素(48%–50%)和经济负担较轻(43%)。
本研究揭示治疗优先事项存在差距:患者重视生活质量和AE管理,而HCP更关注疗效及延缓疾病进展。考虑RRMM新疗法时,亟需教育HCP和患者认识共同决策的重要性。
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 2 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%; P <0.01) and avoiding referrals to new institutions for therapies (32% vs 20%; P <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%, P =0.023; BsAbs, 84% vs 76%, P =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.
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