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:Establishing a successful outpatient CAR T-Cell program with cilta-cel: real-world experience from an expert roundtable.
Establishing a successful outpatient CAR T-Cell program with cilta-cel: real-world experience from an expert roundtable.
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CAR-T 细胞疗法首次获批不到十年,治疗线次已逐渐前移,给药场所从住院转向门诊,患者需求也随之增加。为满足患者和医疗机构需要,多个 CAR-T 门诊项目已启动,带来资源节约、治疗能力提高、住院时间缩短及经济方面的优势。Ciltacabtagene autoleucel(cilta-cel)尤其适合门诊给药,因为在复发/难治性多发性骨髓瘤(MM)成人患者中,其细胞因子释放综合征和免疫效应细胞相关神经毒性综合征通常具有可预测的延迟发生特点。考虑到 MM 治疗的发展、cilta-cel 独特的安全性特征、住院能力有限、成本因素及患者偏好,门诊给予 cilta-cel 正成为及时扩大 CAR-T 治疗可及性的关键环节,使尽可能多的合格患者能够接受治疗。为介绍 cilta-cel 门诊项目的实践要点,并帮助其他诊所建立类似项目,一组具有骨髓瘤和细胞疗法经验的血液科/肿瘤科专家参加了圆桌讨论。他们分享真实世界经验,提供对可行性、安全性和疗效的见解,并确定了机构、患者和照护者准备工作的关键成功因素。
In less than a decade since their first approval, chimeric antigen receptor T-cell (CAR-T) therapies have moved into earlier lines of therapy, shifting administration from an inpatient to an outpatient setting and increasing patient demand. Several CAR-T outpatient programs have been initiated to meet patient and institution needs, resulting in resource conservation, increased treatment capacity, reduced hospitalization times, and financial advantages. Ciltacabtagene autoleucel (cilta-cel) is uniquely suited for outpatient administration, given its predictably delayed cytokine release syndrome and immune-effector cell-associated neurotoxicity syndrome profile in adult patients with relapsed or refractory multiple myeloma (MM).
Given the evolution of MM treatment, the distinct safety profile of cilta-cel, limited hospital capacities, cost considerations, and patient preference, outpatient administration of cilta-cel is becoming a critical part of ensuring increased access to CAR-T therapy in a timely manner to most, if not all, eligible patients.
To highlight the practical aspects of cilta-cel outpatient programs and to facilitate the establishment of similar initiatives at other clinics, a group of hematology/oncology specialists with expertise in myeloma and cellular therapies participated in a roundtable discussion. They shared their real-world experiences, provided insights on feasibility, safety, and effectiveness, and identified key success factors to prepare institutions, patients, and caregivers.
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