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.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Patient perspectives on BCMA-targeted therapies for multiple myeloma: a survey conducted in a patient advocacy group.
Patient perspectives on BCMA-targeted therapies for multiple myeloma: a survey conducted in a patient advocacy group.
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本研究发现,MM 患者对尝试 BCMA 靶向治疗持高度开放态度。
多发性骨髓瘤(MM)治疗进展已改变治疗格局。了解患者观点可帮助医生支持患者做出知情决定。本研究旨在了解患者决策过程,并深入认识患者对 MM B 细胞成熟抗原(BCMA)靶向疗法的看法。
参与者为 HealthTree Cure Hub 注册的 MM 患者。该在线平台帮助浆细胞疾病患者管理疾病;患者完成一份 18 题问卷。
2022 年 10 月 28 日至 2023 年 1 月 12 日,共有 325 例 MM 患者参与。受访者平均年龄(标准差)为 66(8)岁;54% 为女性,90% 为白人。数据库中有完整临床记录的 218 例患者,既往治疗线数中位数(最小值,最大值)为 2(1,16)。61 例(28%)既往接受 4 线治疗,其中 55 例(90%)曾暴露于三类药物。290 例回答是否愿意尝试新疗法的患者中,76 例(26%)愿意立即尝试,125 例(43%)希望进一步了解安全性和疗效。多数受访者表示很可能或可能尝试 BCMA CAR-T(60%)或双特异性抗体(74%);另有部分患者需要更多信息再决定(CAR-T 16%,双特异性抗体 13%)。最希望了解的信息包括疗效、副作用、治疗资格和给药流程。当两种疗效和应答持续时间相同的疗法供选择时,69% 更倾向于严重副作用风险较低但需持续给药、没有停药期的疗法;31% 更倾向于只给药一次、随后有停药期但严重副作用风险可能较高的疗法。为获得有效治疗,患者最能接受的妥协包括频繁监测副作用和在医院启动新疗法;最难接受的是照护者负担。
MM 患者对尝试 BCMA 靶向疗法的接受度较高。疗效、安全性、可及性和治疗资格等信息可帮助患者做出决定。
Advances in multiple myeloma (MM) treatment have shifted the therapeutic landscape. Understanding patients' perspectives can assist physicians in helping patients make informed decisions. This study aimed to understand the patient decision-making process and gain insights into patient perspectives on B-cell maturation antigen (BCMA)-targeted therapies for MM.
An 18-question survey was completed by patients with MM enrolled in HealthTree Cure Hub, an online portal helping patients with plasma cell dyscrasias navigate their disease.
From October 28, 2022, to January 12, 2023, 325 patients with MM participated in the survey. The mean age (standard deviation) of the respondents was 66 (8) years; 54% were female and 90% were White. Among 218 patients with complete clinical records in the database, the median (min, max) lines of therapy (LOT) was 2 (1,16). Among 61 (28%) patients who had received 4 LOTs, 55 (90%) were triple-class exposed. Of the 290 patients who responded to the question about openness to new therapies, 76 (26%) were open to trying a new therapy immediately and 125 (43%) wanted more information on safety and efficacy. Most respondents reported likely or very likely to try a BCMA CAR T-cell therapy (60%) or a bispecific antibody (74%) and some needed more information to decide (16% for CAR T-cell therapy and 13% for bispecific antibody). The most requested information included efficacy, side effects (SEs), eligibility, and administration process for both CAR T-cell and bispecific therapies. When 2 therapies with the same efficacy and duration of response were offered, 69% of respondents would prefer the therapy with a lower risk of severe SEs but requires continuous dosing with no treatment-free interval, and 31% preferred a therapy given once followed by a treatment-free interval but with a potentially higher risk of severe SEs. To receive an effective therapy, the top acceptable trade-offs included frequent monitoring of SEs and initiating a new therapy in a hospital setting, and the least acceptable compromise was caregiver burden.
This study found a high level of openness in patients with MM to try BCMA-targeted therapies. Information on efficacy, safety, availability, and eligibility may assist patients on decision-making.
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