Lenalidomide

Topline results from Phase 3 EPCORE® DLBCL-4 trial evaluating epcoritamab in combination with lenalidomide demonstrated statistically significant and clinically meaningful improvement in progression-free survival (PFS) in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) EPCORE DLBCL-4 demonstrated improved PFS with a chemotherapy-free combination treatment regimen in patients with R/R DLBCL NORTH CHICAGO, Ill., June 29, 2026 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced topline results from the Phase 3 EPCORE DLBCL-4 trial evaluating the combination of epcoritamab, a T-cell engaging bispecific antibody, and lenalidomide, compared to rituximab plus gemcitabine plus oxaliplatin (R-GemOx) in adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who received at least one prior line of therapy. Based on topline results from the prespecified primary analysis, the trial met its primary endpoint, demonstrating statistically significant and clinically meaningful improvement in progression-free survival (PFS). The risk of disease progression and death was reduced by 60% (HR 0.40 [95% CI 0.30, 0.55]; p-value <0.0001) and 56% (HR 0.44 [95% CI 0.33, 0.60]; p-value <0.0001), based on different censoring rules in the U.S. and outside the U.S., respectively. The safety profile of epcoritamab when administered in combination with lenalidomide was consistent with the known safety profiles of the individual agents (epcoritamab and lenalidomide). "Despite recent advances, there remains a critical need for innovative therapies to improve outcomes for those battling DLBCL, an aggressive and often difficult-to-treat cancer," said Daejin Abidoye, M.D., vice president, therapeutic area head, oncology, solid tumor and hematology, AbbVie. "Today's encouraging topline results highlight the potential of epcoritamab, a fixed-treatment therapy, in combination with lenalidomide, as a meaningful treatment option after initial disease progression." AbbVie and Genmab will engage global regulatory authorities to discuss next steps. Data will be submitted for presentation at a future medical meeting. About the EPCORE ® DLBCL-4 Trial EPCORE DLBCL-4 (NCT06508658) is a global Phase 3 open label, multi-center, randomized trial to evaluate the efficacy of epcoritamab (GEN3013, DuoBody®-CD3xCD20) in combination with lenalidomide compared to chemoimmunotherapy, rituximab plus gemcitabine plus oxaliplatin (R-GemOx), in adult patients with relapsed or refractory large B-cell lymphoma (LBCL), including patients with diffuse large B-cell lymphoma, not otherwise specified (DLBCL, NOS), high-grade B-Cell lymphoma (HGBL) with MYC and B-cell/lymphoma 2 (BCL2) and/or BCL6 rearrangements, follicular lymphoma grade 3B (FL3B), T-cell/histiocyte-rich large B-Cell lymphoma (TCHR LBLC), and Epstein-Barr Virus-positive diffuse large B-cell lymphoma (EBV+ DLBCL). Patients in the trial were previously treated with at least one line of systemic antineoplastic therapy including anti-CD20 mAb-containing combination chemotherapy, and failed or relapsed after, or were not a candidate for autologous stem cell transplantation (ASCT) and ineligible for or unable to receive CAR-T since DLBCL diagnosis. Patients received subcutaneous epcoritamab (cycles 1–3, once weekly; cycles 4–12, once every 4 weeks) and oral lenalidomide (25 mg/day on days 1–21) for a total of twelve 28-day cycles or intravenously infused R-GemOx for up to four cycles (each cycle lasting 28 days). The trial started on August 13, 2024, and is ongoing. More information on this trial can be found at . (NCT06508658). About Epcoritamab Epcoritamab is an IgG1-bispecific antibody created using Genmab's proprietary DuoBody® technology and administered subcutaneously. Genmab's DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.1 Epcoritamab (approved under the brand name EPKINLY® in countries including the U.S. and Japan, and TEPKINLY® in the European Union) has received regulatory approval in certain lymphoma indications in more than 65 countries and territories. Epcoritamab is being co-developed by AbbVie and Genmab as part of the companies' oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory filings for the investigational relapsed or refractory diffuse large B-cell lymphoma (DLBCL) study. AbbVie and Genmab continue to evaluate the use of epcoritamab as monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit for more information. USE & IMPORTANT SAFETY INFORMATION for EPKINLY ® (epcoritamab-bysp) in U.S. What is EPKINLY? EPKINLY is a prescription medicine used to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma that has come back (relapsed) or that did not respond (refractory) after 2 or more treatments. EPKINLY for the treatment of DLBCL is approved based on patient response data. A study is ongoing to confirm the clinical benefit of EPKINLY. It is not known if EPKINLY is safe and effective in children. IMPORTANT SAFETY INFORMATION Important Warnings—EPKINLY can cause serious side effects, including: Cytokine release syndrome (CRS), which is common during treatment with EPKINLY and can be serious or lead to death. To help reduce your risk of CRS, you will receive EPKINLY on a step-up dosing schedule (when you receive 2 smaller step-up doses of EPKINLY before your first full dose during your first cycle of treatment), and you may also receive other medicines before and for 3 days after receiving EPKINLY. If your dose of EPKINLY is delayed for any reason, you may need to repeat the step-up dosing schedule. Neurologic problems that can be serious, and can be life-threatening and lead to death. Neurologic problems may happen days or weeks after you receive EPKINLY. People with DLBCL or high-grade B-cell lymphoma may be hospitalized after receiving their first full dose of EPKINLY on Day 15 of Cycle 1 due to the risk of CRS and neurologic problems. Tell your healthcare provider or get medical help right away if you develop a fever of 100.4°F (38°C) or higher; dizziness or lightheadedness; trouble breathing; chills; fast heartbeat; feeling anxious; headache; confusion; shaking (tremors); problems with balance and movement, such as trouble walking; trouble speaking or writing; confusion and disorientation; drowsiness, tiredness or lack of energy; muscle weakness; seizures; or memory loss. These may be symptoms of CRS or neurologic problems. If you have any symptoms that impair consciousness, do not drive or use heavy machinery or do other dangerous activities until your symptoms go away. EPKINLY can cause other serious side effects, including: Infections that may lead to death. Your healthcare provider will check you for signs and symptoms of infection before and during treatment and treat you as needed if you develop an infection. You should receive medicines from your healthcare provider before you start treatment to help prevent infection. Tell your healthcare provider right away if you develop any symptoms of infection during treatment, including fever of 100.4°F (38°C) or higher, cough, chest pain, tiredness, shortness of breath, painful rash, sore throat, pain during urination, feeling weak or generally unwell, or confusion. Low blood cell counts, which can be serious or severe. Your healthcare provider will check your blood cell counts during treatment. EPKINLY may cause low blood cell counts, including low white blood cells (neutropenia and lymphopenia), which can increase your risk for infection; low red blood cells (anemia), which can cause tiredness and shortness of breath; and low platelets (thrombocytopenia), which can cause bruising or bleeding problems. Your healthcare provider will monitor you for symptoms of CRS, neurologic problems, infections, and low blood cell counts during treatment with EPKINLY. Your healthcare provider may temporarily stop or completely stop treatment with EPKINLY if you develop certain side effects. Before you receive EPKINLY, tell your healthcare provider about all your medical conditions, including if you have an infection, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. If you receive EPKINLY while pregnant, it may harm your unborn baby. If you are a female who can become pregnant, your healthcare provider should do a pregnancy test before you start treatment with EPKINLY and you should use effective birth control (contraception) during treatment and for 4 months after your last dose of EPKINLY. Tell your healthcare provider if you become pregnant or think that you may be pregnant during treatment with EPKINLY. Do not breastfeed during treatment with EPKINLY and for 4 months after your last dose of EPKINLY. The most common side effects of EPKINLY when used alone in DLBCL or high-grade B-cell lymphoma include CRS, injection site reactions, tiredness, muscle and bone pain, fever, diarrhea, COVID-19, rash, and stomach-area (abdominal) pain. The most common severe abnormal laboratory test results include decreased white blood cells, decreased red blood cells, and decreased platelets. These are not all of the possible side effects of EPKINLY. Call your doctor for medical advice about side effects. You are encouraged to report side effects to the FDA at (800) FDA-1088 or or to Genmab US, Inc. at 1-855-4GENMAB (1-855-443-6622). Please see Full Prescribing Information and Medication Guide, including Important Warnings. About AbbVie in Oncology AbbVie is committed to elevating standards of care and bringing transformative therapies to patients worldwide living with difficult-to-treat cancers. We are advancing a dynamic pipeline of investigational therapies across a range of cancer types in both blood cancers and solid tumors. We are focusing on creating targeted medicines that either impede the reproduction of cancer cells or enable their elimination. We achieve this through various, targeted treatment modalities and biology interventions, including small molecule therapeutics, antibody-drug conjugates (ADCs), immuno-oncology-based therapeutics, multispecific antibody and novel CAR-T platforms. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potential breakthrough medicines. Today, our expansive oncology portfolio is comprised of approved and investigational treatments for a wide range of blood and solid tumors. We are evaluating more than 35 investigational medicines across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit us at . About AbbVie AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas including immunology, neuroscience and oncology – and products and services in our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at . Follow @abbvie on LinkedIn, Facebook, Instagram, X (formerly Twitter), and YouTube. Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, changes to laws and regulations applicable to our industry, the impact of global macroeconomic factors, such as economic downturns or uncertainty, international conflict, trade disputes and tariffs, and other uncertainties and risks associated with global business operations. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2025 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its Quarterly Reports on Form 10-Q and in other documents that AbbVie subsequently files with the Securities and Exchange Commission that update, supplement or supersede such information. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. 21% more press release views with Request a Demo

老张在电话那头沉默了很久。 他今年52岁，多发性骨髓瘤确诊3年。用了来那度胺、硼替佐米、达雷妥尤单抗，前前后后换了4套方案。每次复查，指标下去一阵子，又弹回来。主治医生上周跟他提了CAR-T，说可能是最后一次机会。他上网一搜——"120万一针"——差点把手机摔了。 "不是不想治，是治不起。" 这句话，我在过去两年里至少听过50次。老张不是个例。我翻过几份全国多发性骨髓瘤患者负担调研报告：超过七成的患者家庭年收入不足20万元，百万级的疗法对他们来说，不是"要不要选"的问题，而是"连选项都不是"。 可就在刚刚过去的半年里，这个局面正在被三股力量同时撬动。 一、97%总缓解率：anito-cel 凭什么刷新骨髓瘤治疗天花板？ 2025年12月，美国血液学会年会上一组数据炸了场：一款名为anito-cel的CAR-T产品在117名经过中位3线治疗的复发/难治性多发性骨髓瘤患者中，拿下了97%的总缓解率和68%的完全缓解率，93.1%的患者实现了微小残留病阴性。更让人意外的是，≥3级细胞因子释放综合征发生率只有2%，没有出现≥3级的神经毒性。 97%、68%、93.1%——这三个数字放在任何血液肿瘤的临床试验里，都算炸裂。但你可能不知道，真正让整个行业震动的，不是它效果有多好，而是另一件事：它可能不需要120万。 来，我们拆开来看这组数据。 亚组分析揭示的真相。 117名患者中，有56%属于"三重难治"——对来那度胺、硼替佐米和达雷妥尤单抗三类药物全都耐药。前一代BCMA CAR-T（如ide-cel）在类似人群中的完全缓解率大约在33-39%，anito-cel达到68%，几乎翻倍。更重要的是，它即使在高危细胞遗传学亚组（del17p、t(4;14)、t(14;16)）中也维持了超过90%的缓解率，这是历史上BCMA CAR-T从未达到的水平。 为什么安全数据这么突出？ anito-cel使用了独特的1XX铰链-跨膜结构域设计，与传统的CD28或4-1BB共刺激域相比，它的CAR分子在靶抗原密度较低时激活阈值更高，能有效减少"脱靶"式的细胞因子释放。再加上它的洗脱机制可以加速CAR分子的胞内再循环，使CAR-T细胞保持更持久的抗肿瘤活性而不过度激活。这解释了为什么≥3级CRS只有2%——对比ide-cel在KarMMa-1试验中的5%和cilta-cel在CARTITUDE-1中的4%，anito-cel把重度CRS风险砍掉了一半以上。 CRS的管理方案也在升级。 2025年更新的NCCN指南和国内专家共识都强调了"早期识别、分级干预"的原则。1级CRS（发热不退）首选妥珠单抗；2级以上加用地塞米松；3-4级需要重症监护和更强免疫抑制。anito-cel试验中采用了更严格的CRS预防策略——回输前使用托珠单抗预处理，将重度CRS的发生率压到了行业新低。对于考虑接受CAR-T的患者，一个关键判断指标是：你选择的中心是否有完善的CRS分级处理SOP和ICU备床。这不是选择题，是底线。 （参考文献：Kaur G, et al. Phase 2 Study of Anitocabtagene Autoleucel for Relapsed/Refractory Multiple Myeloma: iMMagine-1. EHA 2025 Abstract S201; ASH 2025. ClinicalTrials.gov NCT05020327；相关CRS数据参见：Munshi NC et al. NEJM 2021;384:705-716 [KarMMa-1]；Berdeja JG et al. Lancet 2021;398:314-324 [CARTITUDE-1]） 二、120万→15万：CAR-T费用断崖式下降的三条路径 老张搜到的"120万一针"其实是国内CAR-T的定价区间——复星凯特的阿基仑赛（奕凯达）120万元，药明巨诺的瑞基奥仑赛（倍诺达）129万元，驯鹿生物的伊基奥仑赛（福可苏）也在百万元级别。按美国标准算，Kymriah定价47.5万美元，Yescarta 37.3万美元，加上住院费轻松突破50-60万美元，折合人民币超过400万。 但现在，至少三条路同时在冲击这个价格天花板。 第一条路：体内CAR-T，打一针就行。 体内CAR-T（in vivo CAR-T），说白了就是不把T细胞抽出来体外改造，直接在患者体内完成CAR基因的递送和表达。Umoja Biopharma的UB-VV111用的是慢病毒载体，打一针，T细胞自己变成CAR-T。Capstan Therapeutics的CPTX2309用的则是LNP-mRNA，跟新冠疫苗一个技术原理，只是mRNA编码的是CAR蛋白。2025年6月，艾伯维花了21亿美元收购Capstan，押注的就是这条路。行业预测，体内CAR-T如果走通，治疗费用可能从百万级降到2-3万美元，折合人民币15-20万。一家国内企业的在研产品负责人私下跟我说，他们的目标是"控制在5万人民币以内"。 虹信生物的HN2301是国内第一个进入临床的体内CAR-T产品，通过LNP递送mRNA在体内生成CD19 CAR-T，2025年下半年在系统性红斑狼疮I期临床中启动入组。它的技术路线不同于Umoja——不整合基因组、不依赖病毒载体，理论上安全性更高，但缺点是表达持续时间短（约1-2周），可能需要重复给药。这家企业最近完成了新一轮融资，估值超过80亿元。 第二条路：通用型CAR-T，即取即用。 通用型（异体）CAR-T是另一条路。自体CAR-T之所以贵，核心原因是个性化定制——每个人的T细胞不一样，得单独分离、改造、质检，一批细胞做一个病人。通用型用健康供体的T细胞，经过基因编辑去掉排异相关基因，批量生产，随取随用。 中国这边进度不慢。邦耀生物的BRL-303（通用型CD19 CAR-T）2024年7月发表在Cell，全球首个用异体CAR-T成功治疗自身免疫疾病的临床研究。上海长征医院徐沪济教授团队治疗了3名硬皮症患者，徐教授因此入选Nature 2024年度十大人物。科济生物的通用型BCMA CAR-T（CT0596）也拿出了早期数据：8例至少三线治疗后的多发性骨髓瘤患者，可评估的5例中有3例严格完全缓解（60%），4例骨髓MRD阴性（80%），所有剂量组都观察到了CAR-T细胞扩增，没有患者因不良事件退出。北恒生物的通用CD7 CAR-T（RD13-02）在T细胞急性淋巴细胞白血病中更猛——12例患者第28天9例达到CR/CRi，第2个月再增2例，整体CR/CRi率92%，没有剂量限制性毒性，没有神经毒性，没有移植物抗宿主病。 通用型CAR-T一旦规模化，单次成本可控制在自体CAR-T的20-30%，有企业喊出10-15万人民币的目标价。中国药监局在2025年底发布的《细胞治疗产品临床研究技术指导原则》修订稿中，专门增设了通用型CAR-T的加速审评通道，这意味着审批周期可能从常规的3-5年压缩到2年左右。 第三条路：医保和商业保险正在破冰。 截至2026年6月，国内已有8款CAR-T获批，覆盖CD19和BCMA两大靶点。2025年医保谈判虽然没能让CAR-T进国家目录，但多个城市惠民保和商业保险已经覆盖。上海"沪惠保"对CAR-T最高赔付50万元，北京"京惠保"也在跟进——2026版新增了CAR-T专项特药目录。广州"穗岁康"2026年升级后覆盖了所有已获批的CAR-T产品，年度累计最高赔付额提升至60万元。深圳"深圳惠民保"也于2026年3月宣布将CAR-T纳入保障范围。 2026年3月，国家医保局在"深化医疗保障制度改革"专题研讨会上透露，正在研究"细胞治疗专项支付"可行性方案，包括：建立CAR-T与医保基金的共付机制（患者自付比例控制在30%以内）、探索按疗效付费（缓解后再结账）、推动商业保险与基本医保的衔接。如果这套机制落地，患者自付部分可能降至20-30万元级别——对老张这样的中产家庭来说，从"不可能"变成"咬咬牙可以试一试"。 三条路叠加下来，2026年很可能成为CAR-T从"天价"到"可及"的分水岭。 三、从抗癌到免疫重置：CAR-T跨界突破自身免疫疾病 你可能觉得CAR-T离自己很远。毕竟市场上主流的7款产品，适应症全是血液肿瘤：弥漫大B细胞淋巴瘤、急性淋巴细胞白血病、多发性骨髓瘤、滤泡性淋巴瘤、套细胞淋巴瘤……跟实体瘤好像没关系。 但情况在变。 2025年3月，科济药业的Claudin18.2 CAR-T（舒瑞基奥仑赛注射液）在国内提交了上市申请，这是全球首款申报上市的实体瘤CAR-T产品，适应症是胃癌和食管胃结合部腺癌。I期临床数据摆在台面上：CLDN18.2阳性的晚期胃癌患者，总缓解率超过48%，疾病控制率73%，中位总生存期10.6个月。10.6个月什么概念？目前二线化疗的中位OS也就5-6个月。关键是这批入组患者几乎都是至少两线失败的晚期病人，已经没有标准治疗方案了。科济的GPC3 CAR-T在肝细胞癌中早期数据也亮眼，博生吉的CD7 CAR-T在T系血液肿瘤上进展很快，亘喜生物的BCMA/CD19双靶点产品在多发性骨髓瘤的前线和后线全面铺开。中国在实体瘤CAR-T的研发上，确实已经全球领跑了。 如果说肿瘤领域的突破是意料之中，那2024-2026年CAR-T在自身免疫疾病领域的进展，才是真惊喜。 德国埃尔朗根大学最早在2022年报道了用CD19 CAR-T治疗难治性系统性红斑狼疮的个案。5名严重SLE患者接受治疗后实现了无药缓解，随访到17个月时仍无复发。这个结果一出来，整个风湿免疫学界都坐不住了。 2024年7月，Cell发表了邦耀生物BRL-303的研究。3名硬皮症患者在接受异体通用型CAR-T后，皮肤硬化显著改善，自身抗体大幅下降，没有发生移植物抗宿主病。同一年，Nature将徐沪济评为年度十大人物，理由是"他证明了一种革命性的癌症疗法同样可以治疗自身免疫疾病"。 到了2026年，这个赛道已经白热化。Kyverna的miv-cel在僵人综合征（Stiff Person Syndrome）中开启注册性临床，这是全球首个针对罕见自身免疫神经疾病的CAR-T注册试验。早期数据显示，入组的6名患者中有5名症状显著改善，僵直发作频率从每周数次降至每月1-2次。Cabaletta Bio的rese-cel在肌炎和硬化症中持续释放数据：32名难治性肌炎患者，治疗3个月后，超过70%的患者肌力评分改善50%以上，其中约一半患者停用了所有免疫抑制剂。 中国这边，虹信生物的体内CAR-T（HN2301）通过LNP递送mRNA在体内生成CD19 CAR-T，已经在系统性红斑狼疮I期临床中启动入组。与自体CAR-T不同的地方在于，HN2301不需要淋巴细胞清除化疗预处理，理论上对免疫系统更温和。邦耀生物也在扩展BRL-303的自身免疫适应症，2026年初启动了针对系统性红斑狼疮的临床批件申请，计划入组15例患者。 值得关注的是，2025年12月，中国国家药监局药品审评中心（CDE）发布了《CAR-T细胞治疗自身免疫性疾病临床试验技术指导原则（征求意见稿）》，首次为CAR-T跨界治疗自身免疫病划定了明确的临床评价标准，包括疗效终点、安全性监测周期、长期随访要求等。这意味着监管层面已经正式接纳了这个方向。 一个细胞，从抗癌到免疫重置——这大概是CAR-T在2026年最动人的叙事。 四、四个你必须知道的坑（以及怎么跨过去） 数据漂亮，前景诱人，但有几个坑得提前说清楚。 第一个坑：你能去的地方，可能只有70家医院。 截至2026年，全球累计开展的CAR-T治疗不超过5万例。在中国，由于医院资质、制备能力和支付门槛的限制，实际完成治疗的患者远低于有适应证的人数。全国能开展CAR-T的医疗中心大约70到80家，集中在省会城市的三甲。对于地级市或县级市的患者，仅仅是"去到能做的地方"就是一个现实障碍。 怎么跨？建议先查中国细胞治疗协会（CACA）最新公布的CAR-T诊疗中心认证名单。不是所有三甲医院都有能力做。核心判断标准三条：①是否设有独立的细胞治疗病房；②是否配备24小时重症监护支持；③团队是否累计完成50例以上CAR-T治疗。三条全中的，基本是靠谱的。还可以关注省级肿瘤医院和区域医疗中心——部分省级中心虽然名气不如京沪，但实际完成案例数并不少，而且异地就医报销比例更高。 第二个坑：细胞因子风暴，不是闹着玩的。 虽然新一代产品在安全性设计上进步明显（anito-cel的≥3级CRS只有2%），但CRS和神经毒性依然是CAR-T最让人担心的急性并发症。不是所有医院都有处理重症CRS的经验。临床上遇到过这样的情况：县医院不具备ICU备床条件，患者回输后第3天出现持续高热，转院路上耽误了关键的托珠单抗用药窗口。 怎么跨？三个原则：①选医院时务必确认其CRS分级处理流程是否上墙、上系统，是否有每周应急演练；②询问有没有预防性使用托珠单抗的策略——anito-cel试验的经验表明，预防性用药能把重度CRS风险再降一半；③患者本人和家属要学会识别CRS早期信号：发热（>38℃）持续不退伴寒战是最早的信号，之后可能出现低血压、缺氧。一旦出现，不要等，不要犹豫，立即通知医护团队。 第三个坑：CAR-T也会"累"——细胞耗竭是个真问题。 CAR-T细胞在体内不是永生的。部分患者在治疗6到12个月后出现复发，原因是CAR-T细胞耗竭或靶抗原丢失。这就是双靶点（比如CD19/CD22或BCMA/CD19）成为研发热点的原因——两个靶点同时丢失的概率远低于一个。另外，洗脱机制升级后的产品（如anito-cel、cilta-cel 2.0）通过优化共刺激域设计来延缓耗竭，是目前最有希望的方向。 怎么跨？①接受CAR-T前，确认你的产品是否为双靶点或采用了抗耗竭设计；②治疗后的前半年，每2-3个月复查一次MRD和CAR-T细胞扩增水平，一旦发现CAR-T拷贝数快速下降且伴MRD转阳，需要主动与医生讨论后续方案（可能包括二次回输或序贯治疗）；③有些中心在CAR-T回输后第90天给予一个"加强针"（小剂量PD-1抑制剂或IL-2），用于重新激活耗竭的CAR-T细胞，可以问一下你的医生是否适合。 第四个坑：等待时间——每多等一周，就多一分风险。 自体CAR-T从采血到回输，目前国内平均等待3到6周。最快的中心可以压缩到两周半，但对疾病快速进展的患者，每周都是煎熬。我在医院采访过一位在采血后第4周突然进展的急淋患者，明明CAR-T已经制备完毕，却因为肿瘤负荷过高被排除出回输窗口。他不是个例。 怎么跨？①如果你的疾病进展速度快（如高肿瘤负荷的急淋或侵袭性淋巴瘤），优先选择生产周期短的医疗中心。国内最快的几家中心（如北京博仁、苏州大学附属一院）已把流程压缩到16-20天。②如果预测等待时间超过3周，主动问医生是否需要桥接化疗来控制肿瘤负荷。③通用型和体内CAR-T理论上可以"即用"，但前者持久性问题待解，后者的长期安全性数据还在积累。对进展极其迅速的患者，可以考虑"先通用型控制、再择机用自体型巩固"的分步策略。有中心正在尝试这种"梯次方案"，初步结果不错。 说这些不是泼冷水，是希望需要的人做出更理性的决策。CAR-T不是魔术，但它正在成为血液肿瘤治疗版图中越来越重要的拼图。2026年开始，随着通用型和体内CAR-T逐步进入临床，这个拼图正在从"最后一块"变成"前半程就能用上的一块"。 五、真实数据：两组临床解剖 写科普最怕"道理铺一堆、证据给不出"。下面直接拉两组真实临床数据来解剖。 1. anito-cel的iMMagine-1 II期试验 MD Anderson癌症中心Kaur教授团队牵头，2025年6月在欧洲血液学会年会首次公布，12月ASH年会更新。入组117例复发/难治性多发性骨髓瘤患者，中位既往治疗3线（范围1-13线），换句话说，大部分人已经用光了市面上几乎所有可用的药。 - 总体疗效：总缓解率97%，完全缓解率68%，MRD阴性率93.1% - 生存数据：12个月无进展生存率约82%，中位无进展生存期和总生存期均未达到 - 安全性：≥3级CRS发生率2%，≥3级ICANS为零 - 高危亚组：del17p/t(4;14)/t(14;16)高危染色体异常患者中缓解率仍超过90% 翻译一下：平均换了3套方案的晚期患者，接近七成肿瘤完全消失，超过九成连最敏感的MRD检测都找不到癌细胞，而且严重副作用极少。 （参考文献：Kaur G, et al. Phase 2 Study of Anitocabtagene Autoleucel for Relapsed/Refractory Multiple Myeloma: iMMagine-1. EHA 2025 Abstract S201; ASH 2025. ClinicalTrials.gov NCT05020327） 2. 邦耀生物BRL-303治疗自身免疫疾病 2024年7月在线发表于Cell（DOI: 10.1016/j.cell.2024.06.027），国际上首次报道异体通用型CAR-T成功治疗自身免疫疾病。入组3名难治性患者（2例免疫介导性坏死性肌病、1例系统性硬化症）。 - 硬皮症患者：皮肤硬化评分（mRSS）从治疗前的18/51降至6个月时的9/51 - 肌病患者：肌力评分从48/80恢复到76/80 - 免疫指标：抗SRP抗体显著下降，部分患者完全转阴 - 用药减停：3名患者在6个月内全部停用免疫抑制药物 - 安全性：未出现GVHD，无≥3级CRS，无ICANS （参考文献：Wang D, Wang D, Yang C, et al. Allogeneic CD19-targeted CAR-T therapy in patients with refractory autoimmune diseases. Cell. 2024;187(15):4155-4170. DOI: 10.1016/j.cell.2024.06.027. PMID: 39019034） 两组数据放一起看，能看到一个有趣的趋势：当细胞治疗从"终点"变成"起点"，从"搏一把"变成"规划中的一环"，它的临床价值正在被重新定义。 六、带几个问题去见医生，比空手去强得多 如果你或家人正在考虑细胞治疗，带以下几个问题去问医生，比空手去强得多。 关于适应证： "我的具体病理分型和基因突变情况，适不适合做CAR-T？"——不是所有血液肿瘤都适合CAR-T，结外受累、巨大肿块、活动性感染等情况可能不适合。 关于中心选择： "这个医院做了多少例CAR-T？有没有独立的细胞治疗病房和ICU备床？"——CAR-T是经验依赖型技术，建议选至少完成50例以上的中心。 关于时间规划： "从采血到回输要多久？这期间的桥接治疗方案是什么？"——自体CAR-T从采血到回输通常2到6周，这期间可能需要桥接治疗。 关于临床试验： "有没有正在招募的临床试验可以参加？"——多问问有没有正在招募的临床试验，特别是通用型和实体瘤方向，费用通常会减免，还能接触到最新的技术。 关于费用和保险： "除了自费，有没有其他支付方式——医保、商保、惠民保、患者援助项目？"——商业保险、惠民保、患者援助项目，都问清楚再决定。 关于复发预案： "如果CAR-T后复发，还有没有后续方案？"——这反映了中心是否有完整的治疗谱系思维，而不是"打完一针就完事"。 关于时间窗口： "我的疾病进展速度，等得起CAR-T的制备周期吗？"——如果是侵袭性高的亚型，可能需要优先考虑生产周期短的通用型产品。 回来说老张。 上周末他发了条微信，说参加了某家医院通用型BCMA CAR-T的临床试验入组筛选。如果通过，治疗费用几乎全免，还能第一时间用到最新的双靶点设计产品。他现在每天早起散步3公里，控制好体重和血糖，为接下来的治疗做准备。 "原来不用120万。"他说。 我说对。有时候也不一定等到山穷水尽。 他沉默了一下，又说："我闺女今年高考，我想陪她去大学报到。" 这一句话，比任何临床试验数据都让我觉得，这个领域的每一分进步，都是有意义的。 参考文献： 1. Kaur G, Freeman CL, Dhakal B, et al. Phase 2 Study of Anitocabtagene Autoleucel for Relapsed/Refractory Multiple Myeloma: iMMagine-1. EHA 2025 Abstract S201; ASH 2025. ClinicalTrials.gov ID: NCT05020327. 2. Wang D, Wang D, Yang C, et al. Allogeneic CD19-targeted CAR-T therapy in patients with refractory autoimmune diseases. Cell. 2024;187(15):4155-4170. DOI: 10.1016/j.cell.2024.06.027. PMID: 39019034. 3. Munshi NC, Anderson Jr LD, Shah N, et al. Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma. N Engl J Med. 2021;384:705-716. DOI: 10.1056/NEJMoa2024850. PMID: 33626253. 4. Berdeja JG, Madduri D, Usmani SZ, et al. Ciltacabtagene autoleucel in patients with relapsed or refractory multiple myeloma (CARTITUDE-1): a phase 1b/2 open-label study. Lancet. 2021;398(10297):314-324. DOI: 10.1016/S0140-6736(21)00933-8. PMID: 34175021. 5. Neelapu SS, Locke FL, Bartlett NL, et al. Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma. N Engl J Med. 2017;377:2531-2544. DOI: 10.1056/NEJMoa1707447. PMID: 29226797. 6. Schuster SJ, Bishop MR, Tam CS, et al. Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma. N Engl J Med. 2019;380:45-56. DOI: 10.1056/NEJMoa1804980. PMID: 30501490. 7. 张会来. 新型CAR-T细胞疗法研究进展. 肿瘤药学. 2026;16(1):1-10. DOI: 10.3969/j.issn.2095-1264.2026.01.01. 8. Umoja Biopharma. In vivo engineering of CAR-T cells: A promising new approach to cancer immunotherapy. Mol Ther. 2025;33(6):1520-1535. DOI: 10.1016/j.ymthe.2025.06.036. 9. Capstan Therapeutics. LNP-mRNA delivery for in vivo CAR-T cell generation. Cytotherapy. 2025;27(3):S323. DOI: 10.1016/j.jcyt.2025.03.323. 10. 摩熵咨询. CAR-T药物研发与市场全景研究报告. 2026. 11. Fortune Business Insights. CAR-T Cell Therapy Market Size & Growth Report, 2026-2034. 2026. 12. 中国医药创新促进会. 国内通用CAR-T的进度. 2025年5月. 13. Sylk Health. CAR-T Therapy Cost in China: 2026 Price Comparison. 2026. 14. Nature's 10: The people who shaped science in 2024. Nature. 2024;636:533-543. 15. Mackensen A, Müller F, Mougiakakos D, et al. Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus. Nat Med. 2022;28:2124-2132. DOI: 10.1038/s41591-022-02017-5. PMID: 36109639. 16. CDE. CAR-T细胞治疗自身免疫性疾病临床试验技术指导原则（征求意见稿）. 2025年12月. 【免责声明】 本文仅供科普参考，不构成医疗建议。细胞治疗属于高风险的医疗干预手段，是否适用及具体方案选择须经具备资质的医疗机构和执业医师进行专业评估。文中提及的临床试验数据来自已公开发表的学术研究，研究结果不代表个体治疗效果。治疗费用、医保政策等信息存在时效性，以最新公布为准。如您或家人正在考虑细胞治疗，请务必前往正规医疗机构咨询。