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:SOHO State of the Art Updates and Next Questions | Accessing BTK Inhibitors and Other Novel Therapies for Mantle Cell Lymphoma: Are We All Invited to the Party?
SOHO State of the Art Updates and Next Questions | Accessing BTK Inhibitors and Other Novel Therapies for Mantle Cell Lymphoma: Are We All Invited to the Party?
分数与星级只用于站内排序 —— 不代表疗效、安全性或个人适用性。
过去20年间,套细胞淋巴瘤(MCL)的治疗发生了深刻变化。MCL既往以频繁复发和生存期有限为特征;随着含大剂量阿糖胞苷的诱导治疗、利妥昔单抗维持治疗、自体干细胞移植(ASCT)及近年的靶向治疗应用,患者结局得到改善。当代试验已挑战长期沿用的治疗范式:在适合和不适合移植的患者中,基于BTK抑制剂(BTKi)的策略均显示前所未有的无进展生存期;近期随机研究也质疑在优化诱导和维持治疗时代是否所有患者都必须接受ASCT巩固。复发/难治性疾病中,共价和非共价BTKi以及后续CAR-T 细胞疗法已重塑治疗预期,即使在高危人群中也可带来有意义且持久的疗效。
然而,这些进步并未在不同医疗体系中均等实现。来自拉丁美洲和其他中低收入地区的真实世界证据显示,利妥昔单抗、移植、BTKi和细胞疗法的可及性差异显著,并伴随生存差距。随着一线治疗日益采用持续口服药物和资源密集型细胞平台,药物可及性和报销可能成为决定结局的主要因素,其影响或可与疾病生物学本身相当。本综述回顾一线及复发MCL管理的演变,批判性探讨当代ASCT的作用和BTKi序贯治疗,并介绍新兴细胞疗法和抗体疗法。
我们还讨论核心公平性问题:卫生系统如何优先配置高影响力干预,以最大化人群层面的获益?务实且高收益的策略包括确保稳定获得利妥昔单抗为基础的化学免疫治疗、扩大至少一种BTKi的可及性,以及建立先进疗法的结构化转诊网络。在MCL领域,创新如今必须与落实相结合,才能实现全球进展。
Mantle cell lymphoma (MCL) has undergone a profound therapeutic transformation over the past 2 decades. Historically characterized by frequent relapse and limited survival, MCL outcomes have improved with the incorporation of high-dose cytarabine-containing induction, maintenance rituximab, autologous stem cell transplantation (ASCT), and, more recently, targeted therapies. Contemporary trials have challenged long-standing paradigms: BTK inhibitor-based strategies in both transplant-eligible and transplant-ineligible patients have demonstrated unprecedented progression-free survival, while recent randomized data question the universal need for consolidative ASCT in the era of optimized induction and maintenance. In relapsed/refractory disease, covalent and non-covalent BTKi, followed by CAR T-cell therapies, have redefined expectations, offering meaningful durability even in high-risk populations.
Yet, these advances have not translated uniformly across healthcare systems. Real-world evidence from Latin America and other low- and middle-income regions demonstrates substantial variability in access to rituximab, transplantation, BTKi, and cellular therapies, with corresponding survival disparities.
As frontline therapy increasingly incorporates continuous oral agents and resource-intensive cellular platforms, access and reimbursement may become dominant determinants of outcome, rivaling disease biology itself. This update reviews the evolution of frontline and relapsed MCL management, critically examines the contemporary role of ASCT and BTKi sequencing, and highlights emerging cellular and antibody-based therapies.
We further address the central equity question: how can health systems prioritize high-impact interventions to maximize population-level benefit? Ensuring consistent access to rituximab-based chemoimmunotherapy, expanding availability of at least one BTKi, and developing structured referral networks for advanced therapies represent pragmatic, high-yield strategies. In MCL, innovation must now be paired with implementation to achieve global progress.
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