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更新于：2026-04-03

Lisocabtagene maraleucel

利基迈仑赛

更新于：2026-04-03

概要

基本信息

药物类型

自体CAR-T

别名

anti-CD19-EGFRt-4-1BB-zeta modified CAR CD8+ and CD4+ T lymphocyte therapy、anti-CD19/EGFRt/4-1BB/zeta modified CAR CD8+ and CD4+ T lymphocyte therapy、CD19-directed chimeric antigen receptor T cell therapy

+ [7]

靶点

CD19

作用方式

调节剂

作用机制

CD19调节剂(B淋巴细胞抗原CD19调节剂)、T淋巴细胞替代物

治疗领域

肿瘤免疫系统疾病血液及淋巴系统疾病+ [1]

在研适应症

复发性边缘区淋巴瘤难治性边缘区淋巴瘤复发性套细胞淋巴瘤+ [27]

非在研适应症

侵袭性 B 细胞非霍奇金淋巴瘤前体B细胞成淋巴细胞白血病淋巴瘤难治性侵袭性非霍奇金淋巴瘤+ [2]

原研机构

Fred Hutchinson Cancer Research Center

Bristol Myers Squibb Co.

在研机构

Bristol-Myers Squibb Pharma EEIG

Celgene Corp.

Bristol Myers Squibb Co.

+ [3]

非在研机构-

权益机构

Fred Hutchinson Cancer Research Center

Juno Therapeutics, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2021-02-05),

弥漫性大B细胞淋巴瘤滤泡性淋巴瘤高级别B细胞淋巴瘤+ [1]

最高研发阶段(中国)-

特殊审评优先审评 (美国)、加速批准 (美国)、孤儿药 (美国)、孤儿药 (欧盟)

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结构/序列

Sequence Code 133531

来源: *****

Sequence Code 133534

来源: *****

Sequence Code 133555

来源: *****

Sequence Code 133567

来源: *****

Sequence Code 133586

来源: *****

Sequence Code 171111

来源: *****

关联

项与利基迈仑赛相关的临床试验

NCT07194980

/ Not yet recruiting临床2期IIT

Safety and Efficacy of the Addition of Nemtabrutinib to Lisocabtagene Maraleucel in Patients With Relapsed/Refractory Chronic Lymphocytic Leukemia

This phase II trial studies how well the addition of nemtabrutinib to lisocabtagene maraleucel in treating patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). Nemtabrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Lisocabtagene maraleucel is a type of treatment called chimeric antigen receptor (CAR) T-cell therapy, in which a patient's T-cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T-cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein on the patient's cancer cells is added to the T-cells in the laboratory. The special receptor is called a chimeric antigen receptor (CAR). Large numbers of the CAR T-cells are grown in the laboratory and given to the patient by infusion for treatment. Adding nemtabrutinib to lisocabtagene maraleucel may be an effective treatment for relapsed/refractory CLL/SLL.

开始日期2026-04-01

申办/合作机构

Fred Hutchinson Cancer Research Center

[+1]

ChiCTR2600121485

/ Not yet recruiting临床4期IIT

The Efficacy and Safety of Lisaftoclax (APG-2575) Monotherapy in Patients with Indolent Lymphoma

开始日期2026-03-01

申办/合作机构

河南省肿瘤医院

NCT07098364

/ Recruiting临床1/2期IIT

A Phase 1/2 Open-Label Study Evaluating the Safety and Efficacy of ST-067 in Combination With CD19-Directed CAR T-Cell Therapy in Patients With Relapsed/Refractory (R/R) Large B-Cell Lymphoma (LBCL)

This phase I/II trial tests the safety, side effects, and best dose/regimen of ST-067 in combination with CD19-directed chimeric antigen receptor (CAR) T-cell therapy (liso-cel) and how well it works in treating patients with large B-cell lymphoma (LBCL) that has come back after a period of improvement (recurrent) or LBCL that has not responded to previous treatment (refractory). ST-067 is an engineered variant of the human cytokine interleukin-18 that may help the immune system kill cancer cells. Lisocabtagene maraleucel (liso-cel) is an autologous CAR T-cell therapy prepared using the person's own immune system (a group of cells, tissues, and organs that protect the body from attack by bacteria, viruses, and cancer cells) to fight the cancer. Giving ST-067 in combination with liso-cel may better treat patients with relapsed/refractory LBCL.

开始日期2025-12-27

申办/合作机构

Fred Hutchinson Cancer Research Center

[+1]

100 项与利基迈仑赛相关的临床结果

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100 项与利基迈仑赛相关的转化医学

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100 项与利基迈仑赛相关的专利（医药）

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199

项与利基迈仑赛相关的文献（医药）

2026-03-01·Transplantation and Cellular Therapy

Immune Effector Cell-Associated HLH-Like Syndrome (IEC-HS) in CAR-T and TCE-Treated Myeloma and B-Cell Malignancies: Insights from a Pharmacovigilance Study

Article

作者: Irfan, Sohaib ; Kamboj, Ishita ; Rahman, Raeef ; Qureshi, Raabia ; Ahmed, Nausheen ; Abdallah, Al-Ola ; Lutfi, Forat ; Ayoobkhan, Fathima Shehnaz ; Mahmoudjafari, Zahra ; McGuirk, Joseph ; Vojjala, Nikhil

Chimeric antigen receptor T-cell therapy (CAR-T) and T-cell engager antibody (TCE) revolutionized relapsed refractory multiple myeloma (RRMM), non-Hodgkin lymphoma (NHL), and acute lymphoblastic leukemia (ALL) treatment. Immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) is a potentially fatal rare complication characterized by cytopenia, coagulopathy, transaminasemia, and hyperferritinemia. We aim to analyze the IEC-HS reported cases, patterns, and outcomes in RRMM and NHL patients undergoing CAR-T and TCE therapy utilizing the US Food and Drug Administration (FDA) Adverse Event Reporting System (FAERS) database. We conducted a retrospective postmarketing pharmacovigilance inquiry using the FAERS database and the Medical Dictionary for Regulatory Activities (MEDRA). The database was accessed on March 20, 2025 to examine the adverse effects of CAR-T: idecabtagene vicleucel (ide-cel), ciltacabtagene autoleucel (cilta-cel), axicabtagene ciloleucel (axi-cel), brexucabtagene autoleucel (brexu-cel), tisagenlecleucel (tisa-cel) and lisocabtagene maraleucel (liso-cel), and TCE (teclistamab, elranatamab, talquetamab, mosunetuzumab, glofitamab, and epcoritamab) since their FDA approval in the United States and non-US populations. Descriptive analysis was used to describe reported proportions and outcomes. Reported mortality per product was calculated for patients with IEC-HS. Cases reported in 2024, were used to calculate reporting odds ratio (ROR) and empirical Bayesian geometric mean (EBGM) to compare the drugs for IECHS events reported. A total of 23,315 unique case safety reports (ICSRs) were analyzed, identifying 378 (1.62%) reports of IEC-HS. Reported mortality was high among these patients (n = 232/378, 61.3%). A formal disproportionality analysis, limited to 2024 reports to ensure a contemporary comparison, was performed. Tisa-cel (ROR 2.20) and cilta-cel (ROR 2.24) both demonstrated statistically significant disproportionate reporting signals for IEC-HS. In contrast, axi-cel, which accounted for the highest absolute number of reports (n = 114/378), did not show a significant signal in this analysis (ROR 1.02), suggesting its high case volume is proportional to its high overall reporting frequency. Teclistamab showed a significantly reduced likelihood of HLH reporting (ROR 0.43). IEC-HS is a rare but potentially fatal complication. Our disproportionality analysis identifies significant reporting signals for tisa-cel and cilta-cel. The high absolute number of cases observed for products like axi-cel appears to reflect high utilization rather than a disproportionate risk, underscoring the necessity of using statistical signal detection methods when interpreting spontaneous reports.

2026-03-01·PHARMACOECONOMICS

Comparative Economic Evaluations of CAR-T Therapies for Relapsed or Refractory Diffuse Large B-Cell Lymphoma: A Systematic Review

Review

作者: Kim, Hyung Seok John ; Tran, Tien Hoang

BACKGROUND AND OBJECTIVE:

Chimeric antigen receptor T-cell therapies have changed the treatment paradigm of relapsed or refractory large B-cell lymphoma but are among the most expensive treatments. Moreover, their comparative economic value remains uncertain in adults with relapsed or refractory large B-cell lymphoma. The objective of this study was to understand the comparative value of these therapies in this population and the main factors that influenced conclusions on which intervention was considered more cost effective than others.

METHODS:

To assess the comparative cost effectiveness of chimeric antigen receptor T-cell therapies and the drivers of cost-effectiveness results, a systematic literature search of Embase, Scopus, and PubMed was conducted from inception to December 2024 and updated in October 2025. Studies were selected if they were full economic evaluations of head-to-head comparisons of chimeric antigen receptor T-cell therapies for relapsed or refractory large B-cell lymphoma. Two reviewers independently extracted data on key information such as population characteristics and model structure. Results were reported in their original format, and conclusions on cost effectiveness were evaluated based on country-specific willingness-to-pay thresholds. The reporting quality of the included studies was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist and the Drummond 10-Item Checklist.

RESULTS:

Eight full economic evaluations across the USA, Spain, France, and Japan were included in this systematic review. All studies modeled an adult population or patients with relapsed or refractory large B-cell lymphoma who had failed two or more lines of prior therapy, were conducted over a lifetime horizon, and mostly used the payer perspective (n = 6). All studies utilized three-state partitioned survival models but relied heavily on indirect comparison methods such as matching-adjusted indirect comparison. Three chimeric antigen receptor T-cell therapies were evaluated: axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), and lisocabtagene maraleucel (liso-cel). Axi-cel was the most frequently reported cost-effective option (n = 7) with two studies concluding it was the dominant strategy. There was substantial uncertainty in the model results given the lack of individual patient-level data for each arm and reliance on indirect comparisons.

CONCLUSIONS:

In this systematic review of economic evaluations of head-to-head comparisons of chimeric antigen receptor T-cell therapies for relapsed or refractory large B-cell lymphoma, current evidence suggests that axi-cel may be the most cost-effective option. However, given the studies' reliance on indirect comparisons and the absence of any study conducted in low- to middle-income countries, these results must be carefully interpreted until additional evaluations or clinical trials are conducted.

2026-03-01·International Journal of Clinical Oncology

Real-world effectiveness and safety of lisocabtagene maraleucel for relapsed/refractory large B cell lymphoma in Japan

Article

作者: Ochi, Tetsuro ; Ito, Kimiteru ; Izutsu, Koji ; Makita, Shinichi ; Hiratsuka, Anna ; Munakata, Wataru ; Maeshima, Akiko Miyagi ; Nishiyama, Risa ; Iwaki, Noriko ; Takeda, Wataru ; Fukuhara, Suguru

BACKGROUND:

Lisocabtagene maraleucel (liso-cel), an anti-CD19 CAR T cell therapy, has demonstrated efficacy in relapsed/refractory (R/R) large B cell lymphoma (LBCL). However, real-world data from commercial settings outside the United States remain limited.

METHODS:

To evaluate the safety and effectiveness of commercial-use liso-cel in Japan, we conducted a single-center retrospective study of patients with R/R LBCL who received commercial-use liso-cel at our institution between November 2021 and November 2024.

RESULTS:

56 patients received liso-cel infusion. The median age was 66.5 years, and 55.4% had primary refractory disease. Liso-cel was administered as second-line therapy in 14 patients (25.0%) and as third-line or later therapy in 42 patients (75.0%). The best overall response rate was 80.4%, with a complete response rate of 78.6%. At a median follow-up of 12.3 months, 1 year progression-free survival (PFS) and overall survival rates were 69.5% and 86.4%, respectively. The 1 year PFS rates were 68.5% for diffuse large B cell lymphoma not otherwise specified, 100% for primary mediastinal large B cell lymphoma (PMBCL), and 30% for high-grade B cell lymphoma (HGBCL). Grade ≥ 3 cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome were observed in one patient each. Multivariate analysis identified HGBCL subtype and higher pre-infusion metabolic tumor volume as independent adverse factors for PFS.

CONCLUSION:

Commercial-use liso-cel demonstrated high response rates and favorable safety in Japanese patients with R/R LBCL. Patients with PMBCL had excellent outcomes, whereas those with HGBCL showed poorer prognosis, indicating a need for further therapeutic strategies.

928

项与利基迈仑赛相关的新闻（医药）

2026-04-01

·愈见血业

骨髓移植：一个人如何和旧的造血系统告别

2026年3月，全球血液与骨髓移植领域的学者相聚于欧洲血液和骨髓移植学会（EBMT）年会，讨论移植相关血栓性微血管病（TA-TMA）等并发症的最新干预手段。一个月前，2026年移植与细胞治疗会议（Tandem Meetings）也在美国盐湖城公布了Orca-T等细胞疗法的新数据，涉及非复发死亡率（NRM）和无复发生存率（RFS）等关键指标。对研究者和医生而言，这些进展意味着移植治疗正在变得更精准，也更可控。但对每一个躺在层流病房——也就是需要尽可能隔绝病原体、维持高度无菌环境的移植病房——里的患者而言，造血干细胞移植（hematopoietic stem cell transplantation, HSCT）不仅仅是一项高度复杂的现代医疗技术，更是一段必须亲身穿过的过程：旧的造血系统被清除，新的造血系统重新生根，像一场漫长、决绝且充满痛楚的告别。就像自然界在春日破土前必须经历最刺骨的凛冬，一个人要想挣脱恶性血液病（如白血病、骨髓增生异常综合征等）的泥沼，就必须与体内那个曾经维系生命运转、如今却发生叛变或衰竭的旧造血系统彻底诀别。这不仅是细胞层面的生死交替，更是一场涉及生物学机制、心理学重建与全球医疗产业链协同的宏大重塑工程。预处理与“空龛”的营建：一场漫长告别的凛冬序曲在新的生命种子被播撒之前，必须先将原本被污染的土壤彻底翻耕。在造血干细胞真正注入患者体内之前，患者需要经历一段被称为“负数日”（Minus days）的准备期。这一阶段的核心任务，是利用超大剂量的化学药物或全身放射线，对体内的恶性细胞进行无差别的“地毯式轰炸”。在医学上，这一过程被称为预处理（Conditioning）。预处理的本质，是人为地在患者体内制造一场严酷的凛冬，以摧毁原有的造血和免疫系统。从微观生物学的角度来看，哺乳动物的造血系统受到骨髓微环境（即骨髓龛，Bone Marrow Niche）的精密调控。骨髓龛不仅为造血干细胞提供栖息地，还通过分泌各种趋化因子与生长因子维持其静息、自我更新与分化。然而，在恶性血液病患者体内，这种生理平衡已被打破。白血病干细胞（LSCs）会劫持并重塑骨髓微环境，分泌大量的促炎因子（如G-CSF、IL-1α等），不仅占据了有限的生存空间，还加速了正常造血干细胞的衰老与凋亡。图左显示骨组织与骨髓内部环境，右上展示造血干细胞如何分化为红细胞、血小板和不同类型白细胞；下方则是成骨细胞、脂肪细胞、基质细胞等“龛位”组成部分。可把骨髓龛理解为一间会发信号、会分配位置、也会影响细胞命运的“育苗室”。图源Greim H et al 预处理方案的引入，正是为了打破这一被深度污染的生态系统。高强度的放化疗能够诱导旧骨髓微环境中的细胞发生广泛的DNA损伤反应，进而引发不可逆的细胞凋亡与衰老。伴随着旧造血干细胞和部分基质细胞的消亡，骨髓内原本拥挤的物理与化学空间被释放出来，形成了生物学意义上的“空龛”（Empty Niche）。复杂的数学建模与体内生物学示踪研究表明，这些被刻意制造出的空位，是后续供者干细胞成功植入的先决条件。它们使得外来干细胞得以在不与原生残存细胞发生直接物理竞争的情况下，顺利驻留并开启增殖周期。为了尽量减轻这场“凛冬”对心脏、肺、肝脏等非靶向正常器官的毁灭性打击，全球的血液病学家在预处理方案的精准度上进行了不懈的探索。例如，含全骨髓照射（Targeted Marrow Irradiation, TMI）的预处理方案正在被广泛投入应用于临床试验中。TMI技术通过高精度的靶向放射，能够在精准摧毁骨髓靶组织、肃清恶性细胞的同时，最大程度地避开正常器官。这不仅有效减轻了放疗的毒性副作用，还进一步拓宽了移植技术在身体机能较弱或高龄患者群体中的适用边界。这张图的重点是看“照射范围”的变化：传统 TBI 是全身照射，正常器官也会承受较多辐射；IM-TBI 与 TMI/TMLI 则更强调把剂量集中到骨髓、淋巴结和脾脏等目标区域，从而尽量减少肺等重要器官的额外损伤。图源Wong JYC et al 零点时刻与无边长夜：身心剥离的幽谷随着预处理剂量的持续累积，患者体内的白细胞数量会发生断崖式的下降，最终触及绝对的谷底。在医学监测的图表上，这是一个趋近于零的冰冷数值；在重症监护的语境中，它被称为“零点时刻”或骨髓抑制期（Nadir period）。这是整个移植周期里最难熬的一段时间。失去了免疫系统的庇护，患者必须被安置在高度无菌的层流病房中，与外界进行物理隔离。此时，任何微小的病原体——空气中悬浮的真菌孢子、消化道内原本温和的共生细菌——都可能瞬间演变为夺命的梦魇。与此同时，大剂量化疗带来的消化道黏膜大面积受损、持续的高热、难以遏制的出血倾向，也在无情地逼近患者的生理极限。然而，这场痛苦的告别不仅发生在肉体层面，更深深刻印在患者的心理轨迹中。精神病学与肿瘤学领域的交叉研究指出，造血干细胞移植患者在经历躯体折磨的同时，普遍伴随着极高的心理痛苦。这些心理创伤包括严重的抑郁、焦虑、谵妄甚至创伤后应激障碍（PTSD）。这种心理层面的崩塌如果不加干预，不仅会严重降低患者的治疗依从性，甚至可能通过复杂的神经内分泌途径触发或加重移植物抗宿主病（GVHD），从而直接威胁最终的生存结局。为了在这漫长且孤独的黑夜中为患者点燃一盏心理明灯，哈佛大学丹娜-法伯癌症研究所（Dana-Farber Cancer Institute）的研究人员进行了富有成效的尝试。由Hermioni L. Amonoo博士领导的团队提出了一项名为PATH（Positive Affect for the Transplantation of Hematopoietic Stem Cells）的积极心理学干预方案。在一项针对70名移植患者的随机对照试验中，研究人员摒弃了传统耗时的心理咨询，转而采用为期9周、每周仅需15至20分钟的电话心理疏导，引导患者在病床上完成关于感恩、个人优势认知与意义感寻觅的特定练习。这项研究的成果令人振奋：这种专门针对免疫力低下、身体极度虚弱的移植患者量身定制的轻量级干预，显著改善了患者的疲劳感、心理压力与整体生活质量。高达90%的干预完成率也有力地证明，在极端残酷的医疗情境下，温柔而克制的心理抚慰同样具有扭转乾坤的临床价值，它帮助患者在与旧我剥离的剧痛中，树立找到了自洽而坚韧的心理支撑，走出人生极夜下去的微光。精密归巢与破土迎春：微观世界的生命重塑当“负数日”清零，时间轴进入“正数”阶段（Day0及以后），来自供者的造血干细胞悬液将顺着中心静脉导管，缓缓滴入患者体内。在冰冷的病房里，这是新生命的种子，也是微观世界中春意萌动的开始。令人惊叹的是，这些新生的干细胞并没有被直接注射进骨髓，而是进入了外周血循环。它们是如何在浩瀚的血液河流中，精准地找到位于骨髓深处的“空龛”并安营扎寨的？这涉及到一个被生物学家称为“归巢”（Homing）的极其精巧的分子导航机制。归巢机制的核心，是一对被称为SDF-1（基质细胞衍生因子-1，又称CXCL12）和CXCR4的趋化因子-受体轴。预处理后的骨髓微环境中，由CXCL12丰富网状细胞和血管内皮细胞分泌的SDF-1浓度急剧升高，在血液与骨髓之间形成了一道强烈的化学浓度梯度。与此同时，供者造血干细胞表面高表达的CXCR4受体在捕捉到这一化学信号后，会被迅速激活，进而启动细胞内的一系列级联反应，包括PI3K、PKC以及NF-κB等核心信号通路。图中央的 CXCL12（ SDF-1）为骨髓释放出的“导航信号”，下方细胞膜上的 CXCR4 则像干细胞表面的“接收器”。当两者结合后，干细胞会更容易朝骨髓方向移动、黏附并进入骨髓微环境。周围的英文分子名称代表的是一些能增强这条导航通路的辅助因素。图源Ratajczak MZ et al 在这股无形信号的强力牵引下，外周血中的造血干细胞跟随着低切应力（Shear stress）的水流，开始向骨髓血管的内皮壁靠拢。在极迟现抗原-4（VLA-4）、淋巴细胞功能相关抗原-1（LFA-1）以及多种整合素和基质金属蛋白酶（如MMP-2, MMP-9）的协同切割与粘附作用下，干细胞成功突破了血管内皮的致密屏障，穿越至骨髓实质内部。一旦进入骨髓并稳稳占据了预先清理出的“空龛”，这些干细胞便如同度过严冬、深扎于土壤的树根，开始在破土前汲取养分。它们在干细胞因子（SCF）等关键因子的共同滋养下，开启了旺盛的分裂与分化进程。大约两到三周后，当查房医生的生化报表上第一次出现中性粒细胞和血小板稳步攀升的绝对数值时，这意味着新的造血系统已经成功生根发芽。患者体内的血液循环系统，从此流淌着另一个人的基因密码。旧的疾病基因被彻底清洗，衰败的躯体迎来了真正意义上的早春。跨越免疫的鸿沟：从“北京方案”到全球普惠的春天这场微观世界的春之复苏，其绝对前提是必须找到一颗与患者免疫系统足够契合的“种子”。在人类白细胞抗原（HLA）高度多态性的遗传限制下，同胞兄弟姐妹间完全相合的概率仅为25%，而要在毫无血缘关系的茫茫人海中寻找完全匹配的骨髓供者，概率往往低至数万甚至数十万分之一。在很长一段历史时期内，找不到合适的供者，意味着无数绝望的家庭只能眼睁睁看着生命凋零。然而，这一阻碍人类大半个世纪的免疫鸿沟，正在被现代医学以气吞山河之势迅速填平。在这一波澜壮阔的伟大历史进程中，由北京大学血液病研究所黄晓军院士团队开创的“北京方案”（Beijing Protocol）留下了不可磨灭的划时代印记。画面中的赛道代表单倍型移植技术的发展历程。它想表达的是，随着 T 细胞去除技术、北京方案和巴尔的摩方案等平台成熟，半相合移植的疗效、安全性和生活质量结局，已经逐步追上甚至在部分场景中优先于传统 HLA 全相合移植。图源Lv M et al 传统血液学观念认为，非体外去除T细胞的单倍型（即半相合）移植会引发无法控制的致命性GVHD。但“北京方案”通过极具开创性的G-CSF（粒细胞集落刺激因子）体内动员与预激策略，结合精准的免疫抑制网络综合调控，成功攻克了这一长期被视为禁区的医学难题。这一方案使得患者的父母、子女或堂表亲属均可无缝成为供者。长期的多中心临床随访数据证实，对于高危白血病患者，“北京方案”将移植后的复发率从55%大幅降低至19%，3年无白血病生存率（LFS）更是从可怜的33%跃升至73%。其卓越的临床疗效不仅远超传统化疗，甚至在多项核心生存指标上媲美了全相合同胞移植。在“北京方案”以及随后发展的“巴尔的摩方案”（基于移植后环磷酰胺的免疫耐受机制）等新型非T细胞去除技术的共同推动下，全球造血干细胞移植的供者池格局已被彻底颠覆。更为激动人心的突破发生在2025年的美国血液学会（ASH）年会上。由全球非营利性细胞治疗领导机构NMDP（美国国家骨髓捐赠项目）和国际血液与骨髓移植研究中心（CIBMTR）联合公布的ACCESS II期临床试验最新数据，为全人类解答了寻找供者的终极难题。NMDP首席医疗官Steven M. Devine博士在大会上庄严宣告，医学界正在从根本上改变移植医学的可能边界。数据显示，在深度不相合的无关供者移植中，只要辅以合理的移植后化学预防机制，患者依然能够获得令人瞩目的生存结局。为了更直观地展现这一历史性跨越，以下汇总了近期权威临床研究中不同匹配程度下的生存表现：供者匹配程度与干预方案一年总生存率 (OS) 临床意义与趋势解析全相合同胞/无关供者(8/8匹配) 参照基准 (>80%) 长期以来的“黄金标准”，但受限于人口结构变迁与独生子女政策，寻找难度日益攀升。高水平不相合无关供者 (7/8匹配, 结合PTCy) 79% ACCESS试验证实，一个位点的不相合在现代免疫抑制剂的干预下，已被有效克服，临床预后高度稳定。深度不相合无关供者 (<7/8匹配, 结合PTCy) 86% 颠覆性突破：在4/8至6/8位点不合的极端情况下，一年总生存率不仅未降，反而表现出极高的生存韧性。单倍型亲属供者 (“北京方案”) 3年 LFS 达 73% (针对高危白血病) 彻底解决了“无供者可用”的绝境，使得人人皆有供者成为现实，复发率大幅降至19% 。如今，供者来源匮乏这一长期困扰全球血液学家的瓶颈已被彻底打碎。无论是国际顶级专家还是基层的临床医生，都可以基于坚实的循证医学数据向患者保证：在当今世界，只要你需要，超过99%的患者都能在国际骨髓库或亲属中快速找到合适的血细胞供者并安全地接受移植。这是现代医学向人类生命尊严献上的一份最厚重礼物。智库视野：细胞疗法的未来当我们从个体微观的细胞更迭与临床免疫的跨越，将视线拉升至全球宏观的产业版图时，造血干细胞移植及其衍生出的高级细胞与基因疗法（CGT），正展现出前所未有的经济潜能与社会价值。国际权威智库与咨询机构的系列深度报告均指出，这一领域正在经历从“小众医学奇迹”向“可负担普惠医疗”的历史性重构。德勤（Deloitte）健康解决方案中心在2024年底至2025年期间，对全球14位生物制药与生态系统合作组织的高管进行了深度访谈。其行业前瞻报告明确指出，随着底层科研的纵深推进，细胞和基因疗法已经跨越了单纯的血液肿瘤边界，正大步扩展至自身免疫性疾病、1型糖尿病及复杂的神经系统疾病领域。尽管在宏观经济逆风的背景下，全球CGT领域的早期投资额从2021年的230亿美元回落至2024年的150亿美元，但产业基本盘依然强劲。得益于目前超140种已获批疗法和超4000种在研管线的双重驱动，该领域预计到2033年将保持18%的惊人复合年增长率（CAGR）。图源德勤行业前瞻报告麦肯锡（McKinsey & Company）发布的《2026年医疗保健与生命科学行业展望》同样佐证了这一趋势。报告强调，由于CAR-T等基因修饰细胞疗法（如Carvykti, Breyanzi）在多发性骨髓瘤及淋巴瘤的治疗策略中持续向一线或二线前移，全球细胞疗法的商业化进程正在提速。为了抵御传统药物研发中高达90%的失败率与日益严苛的定价压力，全球领先的生物药企正在将人工智能（AI）、生成式AI以及数字孪生（Digital Twins）技术深度整合进研发与制造的各个环节。数字孪生作为虚拟的患者副本，允许研究人员在硅基环境中早期测试新靶点与新药效，从而大幅缩短了漫长且昂贵的临床开发周期。图源麦肯锡（McKinsey & Company）发布的《2026年医疗保健与生命科学行业展望》摘要伴随着产业的狂飙突进，过去只有少数中心、少数家庭能够承接的高复杂度治疗，未来有望借助更成熟的制造体系、更稳定的物流和更清晰的支付方式，被更多医院提供、被更多患者用上。永恒的更迭：生命的韧性与春日的回响对于此刻正在无菌舱或层流病房里的患者而言，前面的数据、报告和行业判断都很遥远。更贴近他们身体的，是预处理期化疗药物涌入静脉时的冰凉，是白细胞降到谷底后反复升起的高热，是口腔和消化道黏膜受损带来的疼痛，也是监护单上那些缓慢、却终于开始向上走的细胞计数。也许此刻躺在那里的人，是母亲，是姐姐，是弟弟，是外公；也可能只是某个被家人和朋友在心里默念过很多遍名字的人。对他们来说，移植不是一段宏大的医学叙事，而是一夜一夜地熬过去：等感染风险过去，等新细胞归巢，等血象回升，等身体重新认出自己。旧叶已落，新芽初绽，每一次干细胞在骨髓深处的成功破土，都是现代科学体系的又一次迈进。然而，医学当然在进步，人也始终脆弱。正因为如此，所有关于移植的知识、照护与祈愿，才格外值得被认真对待。参考文献 Greim H et al. The bone marrow niche, stem cells, and leukemia. Ann N Y Acad Sci. 2014; PMCID: PMC4002179. Wong JYC et al. Total marrow irradiation (TMI): Addressing an unmet need in hematopoietic cell transplantation. Front Oncol. 2022; PMCID: PMC9574324. Ratajczak MZ et al. CXCL12-CXCR4 Axis and Their Role in Stem Cell Homing and Mobilization. Theranostics. 2013; PMCID: PMC3563075. Lv M et al. A perfect mismatch: haploidentical hematopoietic stem cell transplantation overtakes a bend. Cell Death Discovery. 2023; PMCID: PMC10387465. 分享，关注，点赞，一同愈见治愈的期盼

2026-03-31

·百度百家

全球十大药企营收与研发实力大比拼

全球十大药企营收与研发实力大比拼 ■ 强生总营收：2025年总营收达942亿美元，稳居全球首位。其中处方药销售额为557.5亿美元，占总营收的59.2%。肿瘤药物表现尤为亮眼，达雷妥尤单抗实现143.51亿美元销售额，CART疗法Carvykti也贡献了18.87亿美元。研发投入：全年研发预算为146.6亿美元，同比下降14.9%。制药研发投入占比达81%，并购与研发总投入高达320亿美元。公司采取多元化布局战略。市场表现：中国区具体收入数据未公开，通过西达基奥仑赛等产品强化国际合作。预计2026年公司总营收将突破千亿美元大关。 ■ 罗氏总营收：以743.67亿美元位列第二，制药业务收入524.6亿美元，同比增长7%。奥瑞利珠单抗表现突出，五大核心产品合计贡献257亿美元收入。研发投入：研发预算达147.3亿美元，占收入比重19.3%。在肿瘤治疗领域保持领先优势，ADC药物恩美曲妥珠单抗实现24.5亿美元销售额。市场表现：中国市场销售额51.22亿美元，保持稳定增长。依托基因泰克平台持续创新，阿替利珠单抗维持46.3亿美元销售规模。 ■ 礼来总营收：651.79亿美元跃居第三位，同比大幅增长44%。替尔泊肽（降糖+减肥版）合计销售额达365.07亿美元，成为新一代"药王"。研发投入：研发费用133.4亿美元，同比增长21.4%，增速位居行业第一。专注代谢疾病领域，同时拓展乳腺癌和自身免疫治疗业务。市场表现：公司市值突破万亿美元里程碑，GLP1药物占营收比重达34.2%。德国工厂23亿欧元扩产计划推进，预计2026年制药收入将超过700亿美元。 ■ 默沙东总营收：650.1亿美元排名第四，Keytruda单款产品销售额高达316.8亿美元，占公司总收入48.7%。研发投入：157.9亿美元的研发预算位居行业首位，占营收比例24.3%。全年完成21项Ⅲ期临床试验，肿瘤治疗管线布局持续深化。市场表现：PD1抑制剂全球领先地位稳固，但面临专利到期压力。中国区具体数据未披露，公司战略正处于加速调整期。 ■ 辉瑞总营收：625.7亿美元，其中制药业务收入519.5亿美元。新冠疫苗业务下滑后，重点转向肿瘤和罕见病领域。研发投入：104.4亿美元研发预算同比下降4%，连续四年保持百亿美元以上规模。重点投入RSV疫苗和基因治疗技术研发。市场表现：总营收增速放缓至7.8%，中国市场策略持续调整，尚未进入在华营收前列药企行列。 ■ 艾伯维总营收：611.6亿美元，处方药销售额544.8亿美元。利生奇珠单抗单一产品贡献175.6亿美元收入。研发投入：91亿美元研发预算同比下降28.9%，呈现战略收缩态势。以免疫治疗领域为核心业务，但受到GLP1药物的市场冲击。市场表现：总营收增长3.3%，对明星单品的依赖风险显现。中国市场表现逊色于主要竞争对手。 ■ 阿斯利康总营收：587.4亿美元，制药业务收入509.5亿美元。中国区收入66.54亿美元，占比11%，连续两年领跑在华跨国药企业绩。研发投入：142.3亿美元研发预算同比增长5%，占收入比例24.2%保持行业高位。肿瘤与呼吸系统管线并重，计划2030年前在华追加150亿美元投资。市场表现：奥希替尼等核心产品驱动增长，处方药业务增速达16.3%。新兴市场战略成效显著。 ■ 诺华总营收：545亿美元，处方药销售额501.9亿美元同比下降4.4%。多发性硬化症药物业务占比有所收缩。研发投入：112亿美元研发预算同比增长8.2%，重点布局基因治疗和放射性配体疗法领域。市场表现：中国市场收入42亿美元，心血管药物Entresto保持稳定表现。公司正持续优化研发效率。 ■ 诺和诺德总营收：421.2亿美元（仅处方药业务），首次跻身前十。司美格鲁肽系列产品贡献361亿美元，占比高达85.7%。研发投入：虽未进入研发投入前十，但增长势头显著。专注剂型创新，口服版Rybelsus成功解决注射给药问题。市场表现：GLP1市场份额面临挤压，但在糖尿病治疗领域仍具优势。全球肥胖治疗需求持续支撑业务增长。 ■ 百时美施贵宝总营收：482亿美元，处方药销售额478.2亿美元。欧狄沃实现102亿美元销售额，CART疗法Breyanzi达43.7亿美元。研发投入：99.5亿美元研发预算同比下降11%，处于战略调整期。面临Keytruda的激烈竞争，实体瘤领域突破成为关键。市场表现：预测2026年可能跌出前十。血液肿瘤领域优势稳固，但在代谢疾病领域布局存在缺失。

免疫疗法疫苗抗体药物偶联物细胞疗法财报

2026-03-31

·FiercePharma

BMS, Novartis, Gilead, Iovance dinged over biologics promos in rare spate of CBER untitled letters

The letters acknowledge footnotes and fine print from the companies noting that the datapoints in question should be interpreted with caution, but countered that they “do not sufficiently mitigate the overall misleading message.” Promotional materials for a handful of biologics have found their way into the FDA’s hot seat.The regulator’s avowed crackdown on direct-to-consumer pharmaceutical advertising that began in earnest last fall has resulted in dozens of untitled letters flagging perceived issues in marketing materials. The vast majority of those missives, however, have centered on conventional small-molecule drugs; when the frenzy began in September, only one untitled letter went to a product under the purview of the FDA’s Center for Biologics Evaluation and Research (CBER), and before that, CBER’s last untitled letter dated back to 2018.The group of four untitled letters uploaded to the CBER database this month, then, is a rarity. All are dated March 9, and each takes issue with promo materials for a different biologic medicine—from the likes of Iovance Biotherapeutics, Bristol Myers Squibb, Novartis and Gilead Sciences’ Kite Pharma—that the agency has deemed “false or misleading.”All four of the letters single out mentions of or references to “exploratory analyses” beyond the outcome measures in single-arm studies that earned each biologic’s FDA approval. Each missive includes a nearly identical statement that its listed violations “are particularly concerning from a public health perspective because the promotional materials make misleading representations about [the biologic] being more effective or having greater clinical benefit than has been demonstrated. This may cause doctors and patients to inaccurately weigh the risks versus benefits of treatment with [the biologic], which can be fatal or life-threatening.”The letters acknowledge footnotes and fine print from the companies noting that the datapoints in question should be interpreted with caution, but countered that they “do not sufficiently mitigate the overall misleading message conveyed by these materials.” Iovance’s letter (PDF) flags a laundry list of materials regarding its skin cancer med Amtagvi, including slide decks, conference booth panels, branded emails, a clinician-facing website and more.All of the identified items reportedly include references to overall survival (OS) data from studies of Amtagvi. That’s potentially misleading, according to the FDA, because the biologic was approved based on objective response rate, with additional studies still ongoing. BMS, meanwhile, received a letter (PDF) about its B-cell lymphoma treatment Breyanzi. Per the FDA, the healthcare provider-facing website and promotional labeling include representations of OS and progression-free survival (PFS) benefits for Breyanzi, despite the med being approved based only on response rate and duration of response.Novartis’ letter (PDF) homes in on half a dozen sales and marketing materials for its own blood cancer med Kymriah, which the FDA said also incorrectly reference OS and PFS data, since the biologic was approved in multiple indications based on various combinations of ORR, duration of response, complete remission (CR) and minimal residual disease.Finally, the letter (PDF) sent to Gilead’s Kite subsidiary points to an HCP website and sales aid for yet another blood cancer med, Tecartus. Those materials reportedly refer to PFS, OS and relapse-free survival benefits, while Tecartus was greenlit based on ORR and duration of response in one indication and CR and duration of CR in another.In each case, the companies were given 15 working days to respond to the FDA’s listed concerns. As of publication time Monday, only Gilead had replied to Fierce Pharma Marketing’s request for comment.In a statement sent to Fierce, a Gilead spokesperson confirmed receipt of the letter, adding, “The materials were previously reviewed by the FDA, and Kite followed established regulatory procedures. We take patient safety and compliance seriously and are working to ensure our communications are accurate, balanced, and consistent with the Tecartus prescribing information. We are reviewing the FDA’s feedback and working cooperatively with the Agency to address its comments.”

上市批准

100 项与利基迈仑赛相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11990	-	L01XX	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
复发性边缘区淋巴瘤	美国	Juno Therapeutics, Inc.	2025-12-04
难治性边缘区淋巴瘤	美国	Juno Therapeutics, Inc.	2025-12-04
复发性套细胞淋巴瘤	欧盟	Bristol-Myers Squibb Pharma EEIG	2025-11-25
复发性套细胞淋巴瘤	冰岛	Bristol-Myers Squibb Pharma EEIG	2025-11-25
复发性套细胞淋巴瘤	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2025-11-25
复发性套细胞淋巴瘤	挪威	Bristol-Myers Squibb Pharma EEIG	2025-11-25
难治性套细胞淋巴瘤	欧盟	Bristol-Myers Squibb Pharma EEIG	2025-11-25
难治性套细胞淋巴瘤	冰岛	Bristol-Myers Squibb Pharma EEIG	2025-11-25
难治性套细胞淋巴瘤	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2025-11-25
难治性套细胞淋巴瘤	挪威	Bristol-Myers Squibb Pharma EEIG	2025-11-25
复发性滤泡性淋巴瘤	欧盟	Bristol-Myers Squibb Pharma EEIG	2025-03-17
复发性滤泡性淋巴瘤	冰岛	Bristol-Myers Squibb Pharma EEIG	2025-03-17
复发性滤泡性淋巴瘤	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2025-03-17
复发性滤泡性淋巴瘤	挪威	Bristol-Myers Squibb Pharma EEIG	2025-03-17
难治性滤泡性淋巴瘤	欧盟	Bristol-Myers Squibb Pharma EEIG	2025-03-17
难治性滤泡性淋巴瘤	冰岛	Bristol-Myers Squibb Pharma EEIG	2025-03-17
难治性滤泡性淋巴瘤	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2025-03-17
难治性滤泡性淋巴瘤	挪威	Bristol-Myers Squibb Pharma EEIG	2025-03-17
套细胞淋巴瘤	美国	Juno Therapeutics, Inc.	2024-05-31
慢性淋巴细胞白血病	美国	Juno Therapeutics, Inc.	2024-03-18

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
难治性慢性淋巴细胞白血病	申请上市	美国	Bristol Myers Squibb Co.	2023-11-18
复发性慢性淋巴细胞白血病	申请上市	美国	Bristol Myers Squibb Co.	2023-11-18
难治性侵袭性非霍奇金淋巴瘤	临床3期	美国	Celgene Corp.	2018-10-23
难治性侵袭性非霍奇金淋巴瘤	临床3期	日本	Celgene Corp.	2018-10-23
难治性侵袭性非霍奇金淋巴瘤	临床3期	比利时	Celgene Corp.	2018-10-23
难治性侵袭性非霍奇金淋巴瘤	临床3期	芬兰	Celgene Corp.	2018-10-23
难治性侵袭性非霍奇金淋巴瘤	临床3期	法国	Celgene Corp.	2018-10-23
难治性侵袭性非霍奇金淋巴瘤	临床3期	德国	Celgene Corp.	2018-10-23
难治性侵袭性非霍奇金淋巴瘤	临床3期	意大利	Celgene Corp.	2018-10-23
难治性侵袭性非霍奇金淋巴瘤	临床3期	荷兰	Celgene Corp.	2018-10-23

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04245839 (TRANSCEND FL, Pubmed \| Lancet)	临床2期	难治性边缘区淋巴瘤 CD19	66	Lisocabtagene maraleucel	構鏇膚遞衊鑰膚餘壓積(夢觸遞淵繭壓鹽淵憲膚) = 選觸膚襯夢鬱壓糧鏇構鑰積鹽齋構繭蓋鏇願夢 (網憲廠構願製顧襯衊網, 87.3 ~ 99.1) 更多	积极	2026-03-01
Tandem Meetings ASTCT and CIBMTR2026	N/A	多发性骨髓瘤 \| 非霍奇金淋巴瘤	3	Lisocabtagene maraleucel	鬱醖糧顧憲壓齋襯糧蓋(衊鹹衊繭糧醖蓋鑰顧齋) = No ICU transfers for cytokine release syndrome or immune-effector cell associated neurotoxicity syndrome occurred. Readmissions after discharge were not seen. 憲鬱艱鬱積壓鏇憲鹽遞 (醖夢網觸齋廠糧鑰願願 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	多发性骨髓瘤 \| 非霍奇金淋巴瘤	3	Idecabtagene vicleucel		积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	大B细胞淋巴瘤	127	Lisocabtagene Maraleucel (liso-cel)	壓鹹餘網鬱範窪鹽衊鑰(簾構遞壓繭築構齋艱鹹) = numerically fewer liso-cel recipients had any-grade or grade ≥ 3 CRS or ICANS versus axi-cel, and fewer liso-cel recipients needed pharmacologic management with tocilizumab or dexamethasone/other steroids 鬱積夢醖鑰鬱鑰選憲廠 (觸廠選構蓋醖窪鏇壓範 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	大B细胞淋巴瘤	127	Axicabtagene Ciloleucel (axi-cel)		积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	大B细胞淋巴瘤二线	74	Lisocabtagene Maraleucel (liso-cel)	膚選顧鑰製繭簾觸顧鹽(顧選憲築鹽構窪鏇鏇範) = 構壓鬱網鏇願醖窪憲繭願鬱蓋衊鏇簾餘鏇製築 (鏇選願繭鏇齋膚糧顧鑰 ) 更多	积极	2026-02-04
NCT04245839 (TRANSCEND FL, Tandem Meetings ASTCT and CIBMTR2026)	临床1期	滤泡性淋巴瘤三线	107	Lisocabtagene Maraleucel (liso-cel)	憲網鏇襯製蓋選獵獵製(構顧範廠簾選淵壓繭餘) = 齋醖糧顧餘淵觸糧網鹽憲繭醖鹽鑰顧繭繭鹹鑰 (蓋網夢簾憲蓋鹹選艱齋 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	慢性淋巴细胞白血病	100	Liso-cel	憲膚範構簾廠簾選餘艱(積鏇鹽襯襯鹹築夢範蓋) = 鹹顧齋憲鹽襯蓋蓋鑰淵顧築蓋構選艱範鬱製觸 (鏇膚憲繭糧夢鑰獵積齋 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	大B细胞淋巴瘤二线	473	Liso-cel	壓壓餘鬱糧廠窪壓糧憲(夢繭繭繭衊鹹積膚鏇製) = 構蓋範鏇獵衊壓鏇鬱範壓壓願膚觸膚糧遞糧網 (壓廠糧觸鏇夢襯範衊膚 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	大B细胞淋巴瘤二线	473	Axicabtagene Ciloleucel	壓壓餘鬱糧廠窪壓糧憲(夢繭繭繭衊鹹積膚鏇製) = 顧糧顧網構遞夢鹽積鬱壓壓願膚觸膚糧遞糧網 (壓廠糧觸鏇夢襯範衊膚 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	慢性淋巴细胞白血病	41	Lisocabtagene Maraleucel	選夢觸窪繭廠積鬱蓋壓(襯遞鹽網觸膚鹽壓積積) = 獵繭膚憲製蓋獵蓋鑰襯糧鏇積簾衊淵觸積製鹽 (餘鑰選鑰壓構顧觸齋遞 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	非霍奇金淋巴瘤	601	Axicabtagene ciloleucel (axi-cel)	醖顧憲淵鹽膚醖構壓鏇(壓繭簾鏇襯襯鹹齋膚鏇) = 醖衊艱製齋窪襯願廠夢醖鏇壓選鏇簾糧淵襯餘 (鏇夢繭選遞淵醖觸膚鬱 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	非霍奇金淋巴瘤	601	Lisocabtagene maraleucel (liso-cel)	醖顧憲淵鹽膚醖構壓鏇(壓繭簾鏇襯襯鹹齋膚鏇) = 淵廠齋鏇夢窪願鹽鹽襯醖鏇壓選鏇簾糧淵襯餘 (鏇夢繭選遞淵醖觸膚鬱 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	Carney复合症	26	Liso-cel	簾範艱繭艱糧觸淵膚糧(網鏇憲蓋蓋顧夢鑰齋憲) = 獵憲積簾憲壓鬱顧獵壓艱觸鬱製網夢醖艱鹹壓 (選願簾鏇壓蓋憲構廠鏇 ) 更多	积极	2026-02-04