Phase 2 Trial of Total Marrow and Lymphoid Irradiation (TMLI) as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplantation (PBSC-HCT) From a Match Donor With Fludarabine and Melphalan in Older Patients With Refractory Acute Myeloid Leukemia and MDS

This phase II trial tests the effect of total marrow and lymphoid irradiation (TMLI) in combination with fludarabine and melphalan as conditioning regimen in older patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that has not responded to previous treatment (refractory) and that has come back after a period of improvement (relapsed) and are undergoing a donor (allogeneic) peripheral blood stem cell (PBSC) hematopoietic cell transplant (HCT) from a matched related or unrelated donor. HCT is the only curative treatment for high-risk patients, but the side effects related to the current conditioning treatments limit the use to younger and more fit patients. TMLI is a targeted form of total body radiation that uses intensity-modulated radiation therapy to target marrow, lymph node chains, and the spleen. It is designed to reduce radiation-associated side effects and maximize the radiation therapeutic effect. Fludarabine blocks cells from making deoxyribonucleic acid (DNA) and may kill cancer cells. It is a type of purine antagonist and a type of ribonucleotide reductase inhibitor. Melphalan is in a class of medications called alkylating agents. It may kill cancer cells by damaging their DNA and stopping them from dividing. Giving chemotherapy, such as fludarabine and melphalan, and TMLI before an allogeneic transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells to grow. When healthy stem cells from a related or unrelated donor, such as PBSC HCT, that closely match the patient's blood, are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets, an may help destroy any remaining cancer cells. Giving TMLI in combination with fludarabine and melphalan as conditioning treatment for an allogeneic PBSC HCT from a matched related or unrelated donor may be safe, tolerable, and/or effective in treating high-risk older patients with relapsed and refractory acute myeloid leukemia or high-risk myelodysplastic syndrome.

开始日期2027-04-05

申办/合作机构

City of Hope National Medical Center

[+1]

NCT07605416

/ Not yet recruiting临床2期IIT

Quadruplet Induction Followed by Teclistamab Consolidation and Doublet Maintenance in Patients With Primary Plasma Cell Leukemia: The QUANTUM Trial

This phase II trial compares standard consolidation with daratumumab, carfilzomib, lenalidomide, and dexamethasone to consolidation with teclistamab following standard induction therapy and autologous hematopoietic stem cell transplant for improving overall survival of patients with plasma cell leukemia. Consolidation therapy is treatment given after initial therapy to kill any cancer cells that may remain in the body. Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells, including myeloma cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Carfilzomib inhibits protein complexes called proteasomes, which inhibits cancer cell growth and leads to cancer cell death. Lenalidomide may help kill cancer cells and prevents the growth of blood vessels that cancer cells need to survive. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Teclistamab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Giving teclistamab as consolidation therapy after induction and autologous hematopoietic stem cell transplant may improve survival outcomes in patients with plasma cell leukemia, compared to standard consolidation with daratumumab, carfilzomib, lenalidomide, and dexamethasone.

开始日期2026-07-29

申办/合作机构

National Cancer Institute

NCT06954987

/ Not yet recruiting临床2期IIT

A Two Cohort, Randomized, Double-Blind, Placebo-Controlled, Phase II Multi-Center Signal-Finding Study of Venetoclax Combined With Reduced-Intensity Conditioning Followed by Allogeneic Hematopoietic Cell Transplantation Then Venetoclax Maintenance in Adult Patients With Acute Myeloid Leukemia in First Complete Remission: A MyeloMATCH Sub-Study

This phase II MyeloMATCH treatment trial compares the effect of adding venetoclax or placebo to reduced intensity conditioning chemotherapy with fludarabine and busulfan or melphalan, with or without total body irradiation, followed by hematopoietic stem cell transplant and either venetoclax or placebo maintenance therapy after transplant, for the treatment of patients with acute myeloid leukemia (AML). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving chemotherapy and total body irradiation before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets and may help destroy any remaining cancer cells. Adding venetoclax to conditioning therapy before, and giving it as maintenance therapy after, a hematopoietic stem cell transplant may be a more effective treatment option than the usual approach in patients with AML.

开始日期2026-07-17

申办/合作机构

National Cancer Institute

100 项与美法仑相关的临床结果

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100 项与美法仑相关的转化医学

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100 项与美法仑相关的专利（医药）

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10,639

项与美法仑相关的文献（医药）

2026-09-01·SPECTROCHIMICA ACTA PART A-MOLECULAR AND BIOMOLECULAR SPECTROSCOPY

A computational study of perylene diimide as a potential nanocarrier for multiple drugs: adsorption, stability and release mechanisms

Article

作者: Ayub, Ali Raza ; Nabat, Karim Youssef ; Iqbal, Javed ; Salba ; Li, Hui ; Bensahbane, Imane ; Zubair, Sadia ; Sabir, Muhammad Zohaib

Our investigation involves the analysis of perylene diimide (PDI) through DFT estimation, evaluating its potential as a drug carrier framework for Hesperetin, Melphalan, Mercaptopurine, Resveratrol, and Zidovudine. Analysis of the bond lengths between the interacting atoms indicates that PDI demonstrates exceptional carrier capabilities for the delivery of Hesperetin, Melphalan, Mercaptopurine, Resveratrol, and Zidovudine, with measurements of 2.10, 1.82, 2.24, 2.01, and 2.46 Å, respectively. The presence of hydrogen bonding interactions can be analyzed through Noncovalent Interaction (NCI) and Quantum Theory of Atoms in Molecules (QTAIM) methodologies. The dipole moments and chemical descriptors indicate the significant reactivity of the PDI nanocarriers with Hesperetin, Melphalan, Mercaptopurine, Resveratrol, and Zidovudine drugs. According to the analysis of optoelectronic properties, PDI@Mercaptopurine demonstrates a significant adsorption affinity for the perylene diimide (PDI) complex in comparison to other complexes. Moreover, the release of Mercaptopurine from the nanocarrier is enhanced by the stability of adsorption during protonation in an acidic environment, in conjunction with the brief recovery time anticipated for the PDI nanocarrier surface. After considering all the factors mentioned before, it is clear that PDI nanocarriers have a lot of promise as anticancer drug delivery methods.

2026-08-01·AMERICAN JOURNAL OF HEMATOLOGY

Determinants of Long‐Term Benefit From High Dose Melphalan With Autologous Stem Cell Transplant in AL Amyloidosis

Article

作者: Kumar, Shaji ; Zanwar, Saurabh S. ; Schönland, Stefan ; Buadi, Francis ; Oubari, Sara ; Cejka, Vladimir ; Hellou, Tamer ; Kortum, K. Martin ; Dispenzieri, Angela ; Schwotzer, Rahel ; Muchtar, Eli ; Steinhardt, Maximilian J. ; Binder, Moritz ; Gerber, Bernhard ; Roussel, Murielle ; Dingli, David ; Kourelis, Taxiarchis ; Störk, Stefan ; Carpinteiro, Alexander ; Rieger, Max J. ; Hegenbart, Ute ; Einsele, Hermann ; Morbach, Caroline ; Gertz, Morie

ABSTRACT:

High dose melphalan (HDM) with autologous stem cell transplant is an established treatment for systemic light chain amyloidosis, but its incremental benefit in the era of effective standard intensity therapy is unknown. We retrospectively analyzed 475 transplant‐eligible patients who completed standard intensity treatment with or without HDM within 12 months at six centers in Europe and the United States between 2010 and 2024 to evaluate outcomes by baseline risk factors, hematologic response, and receipt of HDM. Death was an equally competing risk after the 12‐month landmark chosen to address early non‐proportional hazards and to ensure completion of first‐line therapy. BMPC > 20% was associated with inferior outcomes (HR 1.7), and gain/amp 1q with shorter TTP (HR 2.15), whereas other high‐risk FISH abnormalities were not. In multivariable models, VGPR/CR after standard intensity therapy was associated with longer TTP (HR 0.49/0.29), while gain/amp 1q remained independently associated with shorter TTP (HR 2.18). Receipt of daratumumab and HDM were associated with longer TTP (HR 0.44/0.61). HDM benefited patients who had not achieved CR after standard intensity therapy (mTTP 21.0 vs. 5.7 months without HDM), whereas patients already in CR did not benefit. Higher BMPC at diagnosis was associated with earlier progression in patients not receiving HDM (HR 1.9 for 5%–20% and 4.09 for > 20%), a risk attenuated in the HDM cohort. Gain/amp 1q remained associated with shorter TTP regardless of HDM exposure. These findings support a selective role for HDM, primarily benefiting patients with residual disease after standard intensity therapy or higher BMPC burden at diagnosis.

2026-07-14·Blood Advances

Infections in patients receiving daratumumab for newly diagnosed multiple myeloma: a pooled analysis of MAIA and ALCYONE

Article

作者: Carson, Robin ; Cavo, Michele ; Zweegman, Sonja ; Borgsten, Fredrik ; Leleu, Xavier ; Facon, Thierry ; Weisel, Katja ; Mateos, Maria-Victoria ; Raje, Noopur ; Cook, Gordon ; Dimopoulos, Meletios A. ; Usmani, Saad Z. ; Basu, Supratik ; Quach, Hang ; Kumar, Shaji K. ; Plesner, Torben ; Ngo, Mai ; Orlowski, Robert Z. ; Bahlis, Nizar ; Jakubowiak, Andrzej ; Manier, Salomon ; Venner, Christopher P. ; Perrot, Aurore ; Moreau, Philippe ; Goldschmidt, Hartmut ; Bolyard, Kasey ; San-Miguel, Jesus ; Hulin, Cyrille ; Chari, Ajai ; Mohty, Mohamad

Abstract:

Both the phase 3 MAIA and ALCYONE studies demonstrated significant survival benefit with the addition of daratumumab to standard-of-care Rd (Revlimid [lenalidomide] and dexamethasone; D-Rd) or to VMP (Velcade [bortezomib], melphalan, and prednisone; D-VMP), respectively, vs Rd or VMP alone in transplant-ineligible newly diagnosed multiple myeloma (NDMM). Patients with NDMM are highly susceptible to infection; therefore, gaining a greater understanding of infection incidence may help to mitigate future risk. We conducted a pooled analysis of infection incidence, timing, and management strategies from MAIA and ALCYONE. The median (range) age of the pooled population was 72 (40-93) years. Incidence was higher with D-Rd/D-VMP vs Rd/VMP for grade 3/4 (36.9% vs 22.4%) and grade 5 (3.0% vs 1.5%) infections; however, exposure-adjusted incidence rates were generally comparable between groups. Any-grade infections led to the discontinuation of study treatment in ∼2% of patients across treatment groups. Of the timing intervals explored, the highest incidence of grade 3/4 infection occurred within the first 6-month interval of study treatment initiation in both groups. Median time to the first onset of grade 3/4 infections (per Kaplan-Meier estimates) was 83.3 months and not estimable in the D-Rd/D-VMP and Rd/VMP groups, respectively. Although the rates of D-Rd/D-VMP treatment discontinuation due to infection remained low in MAIA and ALCYONE, clinicians should remain vigilant about infections throughout treatment and follow local guidelines and International Myeloma Working Group recommendations to minimize the risk of infection. These trials were registered at www.ClinicalTrials.gov as NCT02252172 (MAIA) and NCT02195479 (ALCYONE).

408

项与美法仑相关的新闻（医药）

2026-07-13

·文献计量Pro

【罕见病专题|模板146】2005—2024全球AL淀粉样变性研究图谱：热点、团队与未来方向全解析

Healsan™技术驱动，让科研和SCI论文成为临床工作的副产品。 ▼做您定制的类似分析，请与Jason老师联系。医学圈正在悄悄流行的文献计量分析SCI论文引言淀粉样变性（Amyloidosis）是一类由于异常淀粉样蛋白沉积于组织和器官细胞外，引起器官结构和功能受损的疾病。目前已发现60余种具有形成淀粉样蛋白能力的蛋白，其中30余种与人类疾病密切相关。免疫球蛋白轻链型淀粉样变性（Immunoglobulin Light Chain Amyloidosis，AL amyloidosis），又称原发性淀粉样变性，是最常见的一种系统性淀粉样变性。美国每年约新增4,500例患者，主要发生于50～80岁人群，中位发病年龄约64岁，男性约占三分之二。 AL淀粉样变性的本质是一种浆细胞克隆性疾病。异常浆细胞产生错误折叠的免疫球蛋白轻链，这些轻链以淀粉样纤维形式沉积于心脏、肾脏、神经、肝脏等多个器官。其中，心脏受累最为常见，也是患者死亡的主要原因。过去20年，AL淀粉样变性的诊断和治疗取得了显著进展，新药不断出现，患者生存率持续提高。然而，对于晚期患者而言，该病仍然具有较高死亡率，其发生发展机制也尚未完全阐明。与此同时，随着相关研究数量不断增加，传统综述已难以全面展示整个领域的发展全貌。文献计量学（Bibliometrics）作为一种基于大数据的科研分析方法，能够从发文趋势、国家合作、机构布局、作者影响力、关键词演化等多个维度，系统揭示一个学科的发展历程、知识结构及未来研究方向。目前，针对AL淀粉样变性的文献计量学研究仍然较少，缺乏对整个领域发展格局的系统总结。因此，本研究利用 CiteSpace、VOSviewer、Bibliometrix（R）以及Microsoft Excel 等工具，对2005—2024年全球AL淀粉样变性研究进行了全面的文献计量学分析，从发文趋势、国际合作、核心作者、研究机构、热点主题及前沿方向等多个方面，系统梳理该领域近20年的发展脉络，为未来科研和临床研究提供参考。中文标题：2005—2024年轻链型淀粉样变性研究的文献计量学分析：研究趋势与热点该文2024年发表于 Frontiers in Medicine Impact Factor≈ 3.6；JCR分区：Q1▼ 想发表自己的文献计量分析论文？点击👇获得更多高质量文献计量分析模板。（👆经典文献计量分析模板，持续更新中）以下分为三个方面进行解读。一. 方法2.1 数据来源（Data Sources）本研究以 Web of Science Core Collection（WOSCC）为数据来源，检索时间范围为 2005年1月1日至2024年4月28日。检索主题包括： AL amyloidosis Primary amyloidosis Light chain amyloidosis Amyloid light chain Amyloidogenic light chain Light chain aggregation Immunoglobulin light chain amyloidosis 初步共检索获得 4,980篇文献。随后按照以下标准进行筛选：纳入标准：英文文献 Original Article（原创研究） Review（综述）排除标准：会议摘要社论书评会议论文 Letter Early Access 撤稿图书章节新闻 Expression of Concern等非正式学术文献最终共纳入 2,864篇文献，包括原创研究（Article）：2,277篇; 综述（Review）：587篇，所有文献均利用 CiteSpace 进行重复文献检测，未发现重复记录，最终全部纳入分析。 2.2 数据分析（Data Analysis）本研究综合采用多种文献计量学软件开展分析，各软件承担不同功能：CiteSpace（6.3.R1）：用于关键词聚类分析、参考文献共被引分析以及关键词和文献突现（Burst）分析。VOSviewer（1.6.20）：用于构建国家、机构、作者及关键词合作网络，并分析期刊和机构之间的关联关系。Bibliometrix / Biblioshiny（R 4.3.3）：用于分析作者发文趋势、国家间合作情况及作者—国家—期刊三域关联（Three-field Plot）。Microsoft Excel：用于整理数据并绘制基础统计图表。本研究以2005年1月至2024年4月为时间跨度，对全球AL淀粉样变性领域的发文趋势、国家与机构合作、作者影响力、核心期刊、关键词演化及研究热点进行了系统分析，全面揭示该领域近20年的发展现状及未来方向。二. 主要结果3.1 年度发文情况及发展趋势（Annual Publications and Growth Trend） 2005—2024年，全球AL淀粉样变性领域共发表SCI论文2,864篇。整体来看，该领域发文量呈持续增长趋势，说明AL淀粉样变性正逐渐成为血液病学和系统性疾病研究的重要方向。其中，2020年发文量达到峰值，共发表271篇，占全部文献的9.46%。近年来，虽然年度发文数量略有波动，但总体仍保持较高水平，反映出国际学术界对AL淀粉样变性的关注持续提升。 3.2 主要国家（地区）及国际合作（Leading Countries/Regions and Their Collaboration）过去20年，共有70个国家或地区开展了AL淀粉样变性研究，但整体呈现明显的"头部国家集中"现象，仅有8个国家累计发表论文超过100篇。美国以1,182篇论文位居全球第一，遥遥领先；随后依次为：意大利（395篇）英国（278篇）德国（266篇）法国（215篇）日本（214篇）中国（196篇）从论文影响力来看，美国不仅发文最多，总引用次数（46,900次）也位居世界第一；而英国虽然发文数量不及美国和意大利，但平均每篇论文引用次数（75.95次）位居前列，体现出较高的科研质量。此外，荷兰、瑞典、奥地利等国家虽然整体发文量较少，但论文平均引用率较高，说明这些国家更注重高质量研究。国际合作网络分析进一步显示，美国仍然是全球合作网络的核心。通过总合作强度（Total Link Strength，TLS）分析发现：美国合作强度最高（TLS=34,766）意大利位居第二（TLS=21,431）英国排名第三（TLS=17,640）说明欧美国家已经形成稳定而成熟的国际合作体系。国家合作网络图显示，美国与英国、意大利、德国、法国等欧洲国家之间联系最为紧密，共同构成全球AL淀粉样变性研究的核心合作网络。近年来，中国、日本等亚洲国家也逐渐加入国际合作体系，但整体合作规模仍有进一步提升空间。进一步分析单国合作（SCP）与国际合作（MCP）发现：美国拥有最多国际合作论文（MCP）英国国际合作程度同样较高美国和意大利同时也是单国独立发表论文最多的国家说明美国不仅科研产出最高，也是推动国际合作的重要枢纽。 3.3 主要研究机构分布（Publication Distribution of Organization） 2005—2024年，共有2,896家科研机构参与AL淀粉样变性相关研究，其中285家机构累计发表论文超过5篇。全球发文量排名前五的机构分别为： Mayo Clinic（394篇） University of Pavia（190篇） Boston University（175篇） University College London（153篇） Fondazione IRCCS Policlinico San Matteo（69篇）从论文引用情况来看，University College London、Columbia University、Brigham and Women's Hospital等机构的平均引用次数均超过80次/篇，显示出较高的国际学术影响力。总体来看，美国机构占据明显优势，全球Top20机构中有超过一半来自美国，进一步体现了美国在AL淀粉样变性研究中的主导地位。机构合作网络显示，Mayo Clinic、Boston University、University of Pavia等国际知名中心之间合作十分密切，已经形成多个稳定的国际研究团队，为推动该领域的发展发挥了重要作用。 3.4 高产作者及合作关系（Most Productive Authors and Co-author Relationship）本研究共纳入12,366位作者，其中有44位作者累计发表论文超过30篇。按照发文数量排名： Dispenzieri A.（199篇） Gertz M. A.（165篇） Merlini G.（135篇）位居全球前三。若按照论文引用次数统计： Dispenzieri A.（10,529次） Merlini G.（9,080次） Hawkins P. N.（9,035次）三位作者的学术影响力最高。此外，所有Top20作者的H指数均超过30，其中： Dispenzieri A.拥有最高H指数（65） Merlini G.拥有最高G指数（117）说明这些研究者不仅持续高产，而且长期引领该领域的发展。作者合作网络显示，以Dispenzieri、Gertz、Merlini等专家为核心，形成了多个国际合作团队，彼此之间合作频繁，构成了AL淀粉样变性研究的重要学术共同体。作者发文时间分析进一步发现，Dispenzieri、Merlini、Gertz等核心学者几乎贯穿整个研究发展历程，持续推动该领域不断前进，而近年来也不断有新的青年学者加入这一研究方向，为领域注入新的活力。 3.5 核心期刊分布（Primary Academic Journals） 2005—2024年，AL淀粉样变性相关研究共发表于733种期刊。其中，累计发文超过10篇的期刊共有63种。发文量排名前五的期刊分别为：Amyloid – Journal of Protein Folding Disorders（152篇）Blood（95篇）American Journal of Hematology（57篇）British Journal of Haematology（48篇）Clinical Lymphoma Myeloma & Leukemia（41篇）其中，Blood不仅是血液学领域的顶级期刊，也是本领域引用次数最高的期刊，共获得9,384次引用，显示出极高的学术影响力。此外，《American Journal of Hematology》《Leukemia》《Haematologica》等国际知名血液学期刊也是AL淀粉样变性研究的重要发表平台。 3.6 高被引文献及参考文献共被引分析（Documents Co-citation and References Burst Analysis）研究共分析了39,672篇参考文献，其中77篇文献累计被引用超过100次。引用次数最高的是： **Gertz（2005）**发表于《American Journal of Hematology》，累计引用695次。其次分别为： Kumar（2012，Journal of Clinical Oncology） Dispenzieri（2004，Journal of Clinical Oncology）这些经典文献构成了AL淀粉样变性研究的重要知识基础。参考文献共被引分析共形成9个主要知识聚类，包括： #0 AL amyloidosis #1 高剂量马法兰（High-dose melphalan） #2 心脏淀粉样变性（Cardiac amyloidosis）聚类质量较高，说明整个知识结构较为稳定。突现文献（Citation Burst）分析进一步揭示了推动领域发展的关键研究。其中，Palladini团队2012年发表于《Journal of Clinical Oncology》的论文具有最强突现强度，提出了基于游离轻链（FLC）和心脏生物标志物的疗效评价标准，对临床治疗评估产生了重要影响。 3.7 关键词分析：研究热点与演化趋势（Analysis of Burst Words, Keyword Clusters and Co-occurrence Network）研究共提取6,147个关键词，其中216个关键词出现次数超过20次。出现频率最高的关键词分别为： AL amyloidosis（1,048次） Light-chain amyloidosis（715次） Amyloidosis（701次）说明疾病本身仍是整个领域研究的核心。关键词聚类共形成7个主要研究方向： #0 AL淀粉样变性 #1 心脏淀粉样变性 #2 自体造血干细胞移植 #3 轻链型心脏淀粉样变性 #4 单克隆免疫球蛋白病 #5 支持治疗 #6 肾脏疾病可以看出，目前研究重点主要围绕疾病诊断、器官受累及治疗策略展开。时间线分析显示，近年来研究热点逐渐由传统治疗转向疾病分型、疗效评价、生物标志物及器官保护等方向，反映出AL淀粉样变性研究正不断向精准医学发展。突现关键词分析显示，近年来持续受到关注的主题包括： Cardiac amyloidosis（心脏淀粉样变性） Biomarkers（生物标志物） Outcome（预后） Recommendations（指南） Light-chain amyloidosis（轻链型淀粉样变性）提示精准诊断、风险评估和规范化治疗已成为当前研究热点。 3.8 三域图分析：关键词—期刊—国家（Three-field Plot Analysis Representing Keywords–Sources–Countries）三域图分析进一步揭示了研究主题、发表期刊及国家之间的联系。结果显示，“AL amyloidosis”是连接最广泛的核心关键词，与期刊Amyloid – Journal of Protein Folding Disorders联系最为紧密。此外，该期刊还与： Light-chain amyloidosis Survival Primary systemic amyloidosis 等关键词高度相关，是全球AL淀粉样变性研究的重要发表平台。从国家分布来看，美国、意大利和法国与上述核心研究主题联系最密切，进一步反映了欧美国家在该领域的主导地位。三. 讨论4.1 全球AL淀粉样变性研究进入快速发展阶段本研究对2005—2024年全球AL淀粉样变性文献进行了系统分析，结果显示，该领域发文量持续增长，尤其近十年增长明显，说明AL淀粉样变性已成为血液病学和精准医学的重要研究方向。从全球科研格局来看，美国始终处于主导地位，在发文数量、学术影响力和国际合作方面均位居世界第一；意大利、英国、德国等欧洲国家同样具有较强科研实力。近年来，中国、日本等亚洲国家发文量不断增加，国际影响力也在逐步提升，全球研究正呈现多中心协同发展的趋势。4.2 研究热点与未来方向关键词分析显示，过去20年AL淀粉样变性的研究主要集中在生物标志物、心脏淀粉样变性和治疗策略三个方向。4.2.1 生物标志物：推动疾病早诊和精准评估由于AL淀粉样变性的临床表现缺乏特异性，患者往往在出现严重器官损伤后才被确诊，因此寻找更加敏感、准确的生物标志物一直是研究重点。目前，NT-proBNP、心肌肌钙蛋白和游离轻链（FLC）等指标已广泛应用于疾病分期和预后评估。近年来，GDF-15、Galectin-3、D-二聚体等新型标志物也受到越来越多关注。未来，多种生物标志物联合应用，并结合人工智能、大数据分析和多中心临床数据库，有望进一步提高疾病的早期诊断和精准预测能力。4.2.2 心脏受累：决定患者预后的关键因素心脏受累是AL淀粉样变性最常见、也是最致命的表现，因此成为近年来增长最快的研究热点。由于临床表现容易与肥厚型心肌病或心力衰竭混淆，早期识别仍是当前最大的挑战。目前，组织活检结合质谱分析仍是确诊和分型的金标准，而超声心动图、心脏磁共振及多种血清学指标则为早期筛查提供了重要依据。未来，建立更加准确的早期诊断体系和疾病监测指标，将有助于改善患者预后。4.2.3 治疗策略：迈向精准治疗新时代过去20年，AL淀粉样变性的治疗不断升级。从高剂量马法兰联合自体造血干细胞移植，到硼替佐米为代表的蛋白酶体抑制剂，再到近年来广泛应用的CD38单克隆抗体达雷妥尤单抗，患者生存率得到明显改善。与此同时，CAR-T细胞治疗、双特异性抗体、抗体偶联药物等新一代免疫治疗技术正在快速发展，为复发或难治患者带来了新的希望。值得关注的是，目前研究重点已不仅局限于清除异常浆细胞，而是逐渐延伸至异常轻链形成、蛋白聚集及淀粉样沉积机制等基础研究，为开发新的靶向治疗提供理论基础。总体来看，未来AL淀粉样变性的研究将进一步向精准诊断、精准治疗和多学科协作方向发展，并依托国际合作和临床研究不断推动诊疗水平提升。编者按对于许多患者而言，AL淀粉样变性（免疫球蛋白轻链型淀粉样变性）的起点，往往只是乏力、水肿、蛋白尿或心功能异常等看似普通的症状；然而，当真正确诊时，心脏、肾脏等重要器官往往已经受到不可逆损伤。由于临床表现复杂、诊断困难，这种罕见病长期被称为“最容易漏诊的血液系统疾病”之一。过去20年，随着浆细胞疾病研究、蛋白质错误折叠机制以及精准医学的快速发展，AL淀粉样变性的诊疗理念正在发生深刻变化。从传统化疗到蛋白酶体抑制剂、CD38单克隆抗体，再到CAR-T等新型免疫治疗；从依赖组织活检到生物标志物、影像学和人工智能辅助诊断，整个领域正迈向更加精准、高效的新时代。本期，我们结合最新发表的文献计量学研究，系统梳理2005—2024年全球AL淀粉样变性的科研格局，从发文趋势、国际合作、核心团队、研究热点到未来方向，全景展现这一领域近二十年的演进轨迹，希望为临床医生、科研人员以及关注罕见病的读者提供一份值得收藏的研究指南。原文链接：DOI: 10.3389/fmed.2024.1441032 关键词：#文献计量学；#Hanson临床科研；#罕见病广而告之：如果您也需要对自己公司的产品或者对自己研究的领域做系统梳理，可以找美国Healsan恒祥医学科技做文献计量分析。编译：Shutong Liu，微信号：Healsanz。美国Healsan (恒祥医学科技)，专长于Healsan医学大数据分析（Healsan™）、及基于大数据的Hanson临床科研服务（HansonCR™）和医学编辑服务（MedEditing™）。主要为医生科学家、生物制药公司和医院科研处等提供文献计量分析和SCI论文润色、编辑、选刊等服务，成为诸多机构的“临床科研外挂”。网址：https://healsan.com/ （点击👆，获得持续的医学大数据分析报告） ▼ Healsan医路成长更多免费资源：（点击👆图片，进入自己感兴趣的专辑。或点击“资源”，浏览本公众号所有资源） #文献计量分析 #文献计量Pro #Hanson临床科研

2026-06-26

·李氏大药厂

快讯！李氏大药厂旗下子公司兆科肿瘤注射用盐酸美法仑（50mg）成功获批

6月17日，国家药品监督管理局（NMPA）正式公示，李氏大药厂控股有限公司（简称：李氏大药厂或李氏集团，股份编号：00950.HK）全资子公司兆科（广州）肿瘤药物有限公司（简称：李氏-兆科肿瘤）自主研发的注射用盐酸美法仑（50mg）获批，且视同通过仿制药质量和疗效一致性评价。本品为血液肿瘤领域高端改良型仿制药，临床主要用于多发性骨髓瘤患者造血干细胞移植前的高剂量预处理治疗，以及不适合口服剂型治疗的多发性骨髓瘤患者的姑息治疗，是临床多发性骨髓瘤综合治疗的核心关键药物。传统制剂在溶解性与稳定性方面存在一定局限，影响了患者的治疗效果。李氏-兆科肿瘤本次获批的产品采用环糊精包裹技术，改善了药物的溶解性与稳定性。该产品溶解后在25℃条件下可保存1小时，5℃冷藏条件下可保存24小时，混合溶液在25℃下可保存4小时。更长的稳定时限可支持减慢滴注速度、延长给药时间，较长的稳定时限有助于临床给药方案的灵活调整，为临床操作提供充裕的时间窗口。作为高壁垒复杂注射剂，本品研发及产业化难度极高。产品杂质控制标准严苛、冻干工艺开发难度大，且药物具有细胞毒性，原料称量、药液配制、无菌灌装等全生产流程均需采取严格的安全防护措施。同时，美法仑原料药溶液状态稳定性差，为严格控制杂质增长，生产各步骤需精准管控药液温度与工序时长，对无菌生产体系与精细化工艺管控能力提出极高要求。研发过程中，项目团队结合委托生产企业产线设备条件，多轮迭代优化整套生产工艺，逐一攻克杂质精准控制、高等级无菌保障、细胞毒性安全生产等核心技术难点，最终实现产品质量符合仿制药质量和疗效一致性评价要求，圆满完成高品质产业化落地。本次注射用盐酸美法仑的顺利获批，为相关临床场景提供了新的用药选择，进一步完善了李氏大药厂与兆科药业的血液肿瘤产品管线，同时丰富了国内多发性骨髓瘤临床治疗的药物可及性。兆科药业项目团队专注于注射用等多种针剂产品的研发与生产，特别是复杂型针剂领域，相继攻克细胞毒性管控、高标准无菌生产、杂质精准控制等多项技术难关。该产品是继已上市的磺达肝癸钠注射液、舒更葡糖钠注射液等产品之后，依托李氏-兆科合肥基地复杂注射剂研发平台产业化落地的又一个高难度特殊注射剂项目。伴随集团在高端复杂注射剂平台的持续投入，包括正在开发的注射用西那普肽磷脂表面活性剂等多款高技术壁垒特色注射剂的陆续产业化，将进一步夯实李氏-兆科合肥基地在高端特色注射剂领域的核心竞争力，彰显公司持续深耕创新制剂、聚焦临床刚需、攻坚高端技术壁垒的研发实力。 *本文为李氏大药厂产品研发进展信息，不构成任何用药建议。往期回顾 ◆ 父爱如山，温暖相伴 ◆ 夏至 | 盛夏伊始，不负韶光 ◆ 全力以赴，共攀高峰——李氏大药厂曲特恪®A组精英服务商交流会 ◆ 乘势而上，共赢新局｜李氏大药厂全国重点集采经销商沟通会圆满落幕关于李氏大药厂 - 亞太地區最具創新力的上市企業 - 李氏大药厂控股有限公司（简称：李氏大药厂或李氏集团，股份编号: 00950.HK）成立于1994年，是一家结合了研发驱动和市场导向的生物医药集团公司，总部位于香港。李氏大药厂于2002年7月在香港联合交易所（简称：“港交所”）创业板上市，并于2010年5月成功转至港交所主板上市。李氏大药厂在全球拥有三大综合性生产研发基地和两大中心，即：李氏-兆科合肥基地（位于安徽省合肥市高新区）、李氏-兆科南沙基地（位于广东省广州市南沙区）、美国加州基地和集团研发中心、集团营销中心，目前员工总数超过1100人。李氏大药厂立足中国大陆，放眼国际，通过坚实和稳固的投入建设，全面覆盖整合了药物研发、临床研究、药政注册、生产制造、销售与市场推广等各个环节。集团集中于心血管科、妇科与产科、儿科、罕见病、肿瘤科、皮肤科、精神科等多个重要疾病领域开发不同阶段的专利新药和高端仿制药。李氏大药厂与20余家国际公司建立了广泛的合作关系，目前在中国大陆、香港、澳门及台湾地区推广及销售25种包含专利新药、仿制药物及授权引进的国外已上市进口药品。李氏大药厂现有超过40个处于不同开发阶段的产品，既有自主研发产品，也有通过来自美国、欧洲及日本等公司通过授权引进进行研发、生产以及商业化的产品。李氏大药厂的使命是致力于研发新药，治疗顽疾，改善生活质量，为患者带来福音，目标成为亚洲成功的生物制药集团，提供治疗疾病和改善生活质量的创新产品。下滑查看更多免责声明：本公众号发布或转载企业宣传资讯，仅供读者参考之用，并不构成收购、购买或认购依据，并不构成任何个人化的投资劝诱或建议。因本公布全部或任何部分内容而产生或因倚赖该等内容而引致的任何损失不承担任何责任。企业文化公共关系部出品图片来源：国家药品监督管理局网站撰稿：高坤坤排版：樊扬波校对：章青云审核：李刚与“李”同行招商热线 020-39062882 公众号小程序视频号小红书

一致性评价上市批准

2026-06-26

·BioSpace

CHMP recommendation advances Johnson & Johnson’s TECVAYLI®▼ (teclistamab) plus daratumumab as a potential standard of care for relapsed/refractory multiple myeloma

Recommendation supported by unprecedented Phase 3 data, demonstrating that TECVAYLI ® in combination with daratumumab achieved statistically significant improvements in progression-free and overall survival versus standard of care 1 Pending approval, this novel complementary immunotherapy combination is positioned to delay disease progression for patients treated as early as second line, with 83% of patients alive at three years 1 BEERSE, BELGIUM, June 26, 2026 (GLOBE NEWSWIRE) -- Johnson & Johnson today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of an indication extension of TECVAYLI ® (teclistamab) in combination with daratumumab for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior therapy. Advancing complementary immunotherapies to improve patient outcomes Teclistamab and daratumumab work in a mechanistically complementary manner by engaging multiple tumour and immune directed pathways. 1,2 When combined, daratumumab modulates the immune system to create a more favourable immune microenvironment and enhances T-cell fitness and activation, amplifying teclistamab-mediated killing of myeloma cells. 1,2 This combination improves patient outcomes by using immunotherapies earlier in the treatment journey when patients’ immune systems are more robust. 1, 2 Expert and company perspectives support the earlier use of teclistamab combination in multiple myeloma care “Unprecedented data show a meaningful extension in overall survival and minimal progression events observed after the first six months. More than 90% of patients receiving the combination who were progression-free at six months remained progression-free at three years, highlighting the potential for durable long-term disease control.” said Ester in ’t Groen, EMEA Therapeutic Area Head, Haematology, Johnson & Johnson. “This CHMP opinion marks an important step towards establishing the off-the-shelf immunotherapy combination of teclistamab plus daratumumab as a new standard of care earlier in the treatment pathway for multiple myeloma.” “At Johnson & Johnson our ambition is to leverage the full potential of our comprehensive multiple myeloma portfolio, to strengthen patient outcomes at every stage of the treatment continuum,” said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Johnson & Johnson. "By advancing innovative immunotherapies such as teclistamab and combining them with a well-established standard of care like daratumumab, we are building on our deep scientific expertise to deliver more integrated, combination-based approaches that can continue to raise expectations for patient care.” Teclistamab plus daratumumab SC achieves meaningful and sustained improvements in patient outcomes The CHMP recommendation is supported by data from the Phase 3 MajesTEC-3 study ( NCT05083169 ), evaluating the efficacy and safety of teclistamab plus daratumumab subcutaneous (SC) formulation versus investigator’s choice of daratumumab SC and dexamethasone with either pomalidomide or bortezomib (DPd/DVd) in patients who have received 1–3 prior lines of therapy. 3 At nearly three years of follow-up, results show an 83.4% reduction in the risk of disease progression or death in patients treated with teclistamab plus daratumumab SC, compared to standard of care (hazard ratio [HR], 0.17; 95% confidence interval [CI], 0.12-0.23; p <0.0001). 1 More than 90% of patients who remained progression-free at six months (n=249) remained progression-free at three years, demonstrating sustained disease control over time. 1 Overall survival (OS) favoured teclistamab plus daratumumab SC (HR, 0.46; 95% CI, 0.32-0.65; p <0.0001), with treatment benefit observed across all prespecified subgroups. 1 ,2 At three years, OS rates were 83.3% for the combination and 65.0% for standard of care. 1 Both progression-free survival and OS benefits were clinically meaningful and statistically significant. 1 Manageable safety pro with teclistamab combination Teclistamab plus daratumumab SC demonstrated a safety pro with the well-known profiles of the individual therapies. 1 , 3 , 4 All cases of cytokine release syndrome were Grade 1/2, did not lead to treatment discontinuation, and were manageable and resolved. 1 Rates of Grade 3/4 treatment-emergent adverse events (TEAEs) were comparable to standard of care regimens (95.1% vs. 96.6%) with cytopenia and infection most commonly observed. 1 Grade ≥3 infections decreased over time with the use of immunoglobulin supplementation and infection prophylaxis, along with a switch to monthly dosing. 2 Discontinuations due to TEAEs were low and similar between study arms (4.6% vs. 5.5%). 1 About the MajesTEC-3 Study MajesTEC-3 ( NCT05083169 ) is an ongoing, Phase 3 randomised study evaluating the safety and efficacy of teclistamab plus daratumumab subcutaneous (SC) (n=291) versus investigator’s choice of daratumumab SC and dexamethasone with either pomalidomide or bortezomib (n=296) (DPd/DVd) in patients with relapsed or refractory multiple myeloma (RRMM) who have received 1–3 prior lines of therapy. 5 The primary endpoint is progression-free survival (PFS) and secondary endpoints include complete response or better (≥CR), overall response rate (ORR), minimal residual disease (MRD) negativity (10⁻⁵ by next-generation sequencing), overall survival (OS), time to worsening of symptoms (MySIm-Q), and safety. 5 The MajesTEC-3 study is a part of the MajesTEC clinical programme, which includes exploring the potential of teclistamab as a combination regimen. 5 About Teclistamab Teclistamab received European Commission (EC) approval in August 2022 for the treatment of patients with RRMM who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy. 6 In August 2023, the EC approved a Type II variation application for teclistamab, providing the option for a reduced dosing frequency of 1.5mg/kg every two weeks in patients who have achieved a complete response (CR) or better for a minimum of six months. 7 Teclistamab is an off-the-shelf (or ready-to-use) bispecific antibody. 3 , 8 Teclistamab, a subcutaneous injection, redirects T-cells through two cellular targets (BCMA and CD3) to activate the body’s immune system to fight cancer. 1,3 Teclistamab is currently being evaluated in several combination studies. 5 , 9,10 ,11 To date, more than 26,000 patients have been treated worldwide with teclistamab. 1 2 For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using teclistamab, please refer to the Summary of Product Characteristics at: . ▼ In line with EMA regulations for new medicines and those given conditional approval, teclistamab is subject to additional monitoring. 3 About Daratumumab and Daratumumab SC Johnson & Johnson is committed to exploring the potential of daratumumab for patients with multiple myeloma across the spectrum of the disease. In August 2012 , Janssen Biotech, Inc., a Johnson & Johnson company, and Genmab A/S entered a worldwide agreement, which granted Johnson & Johnson an exclusive licence to develop, manufacture and commercialise daratumumab. Since launch, daratumumab has become a foundational therapy in the treatment of multiple myeloma, having been used in the treatment of more than 748,000 patients worldwide. 1 3 Daratumumab was the first CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma. 5, 1 4 Daratumumab SC was also the first oncology injectable approved for administration by patients living with multiple myeloma or their caregivers from the fifth dose, if determined to be appropriate by their healthcare professional and following proper training. 5, 15 Daratumumab SC is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE ® drug delivery technology. 5 CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease. 5 ,16 Daratumumab binds to CD38 and inhibits tumour cell growth causing myeloma cell death. 5 Daratumumab may also have an effect on normal cells. 5 Data across ten Phase 3 clinical trials, in both the frontline and relapsed settings across all newly diagnosed multiple myeloma patients, have shown that daratumumab-based regimens resulted in significant improvement in progression-free survival and/or overall survival. 17,18,19,20,21,22,23,24,25,26 For further information on daratumumab, please see the Summary of Product Characteristics at: . About Multiple Myeloma Multiple myeloma is a complex blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow. 2 7 ,2 8 In multiple myeloma, these malignant plasma cells continue to proliferate, accumulating in the body and crowding out normal blood cells, as well as often causing bone destruction and other serious complications. 2 9 , 30 In the European Union, it is estimated that more than 35,000 people were diagnosed with multiple myeloma in 2022, and more than 22,700 patients died. 31 Patients living with multiple myeloma experience relapses which become more frequent with each line of therapy, while remissions become progressively shorter. 32 , 33 ,3 4 Whilst some patients with multiple myeloma initially have no symptoms, others can have common signs and symptoms of the disease, which can include bone fracture or pain, low red blood cell counts, fatigue, high calcium levels, infections, or kidney damage. 3 5 About Johnson & Johnson At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at . Follow us at . Cautions Concerning Forward-Looking Statements This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of teclistamab and daratumumab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s most recent Annual Report on Form 10-K, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. Key Search Terms MajesTEC-3 Phase 3 clinical trial TECVAYLI Teclistamab DARZALEX Daratumumab Teclistamab and daratumumab combination TECVAYLI and DARZALEX combination Relapsed and or refractory multiple myeloma (RRMM) Early relapse multiple myeloma Second line multiple myeloma treatment First relapse therapy for multiple myeloma Treatment resistance in multiple myeloma Progression-free survival (PFS) in multiple myeloma Overall survival (OS) in multiple myeloma Multiple myeloma Bispecific antibody therapy CD38-targeted therapy BCMA-targeted bispecific therapy Combination immunotherapy EMEA haematology pipeline Multiple myeloma innovation Multiple myeloma treatment END References: 1 Costa L, et al. Teclistamab plus Daratumumab in Relapsed or Refractory Multiple Myeloma. N Engl J Med. 2026;394(8):739-752. 2 Mateos MV, et al. Phase 3 Randomized Study of Teclistamab plus Daratumumab Versus Investigator’s Choice of Daratumumab and Dexamethasone with either Pomalidomide or Bortezomib (DPd/DVd) in patients (pts) with Relapsed Refractory Multiple Myeloma (RRMM): Results of the MajesTEC-3 Study. Oral Presentation #06. American Society of Hematology (ASH) Annual Meeting; 06-09 December, 2025. 3 European Medicines Agency. TECVAYLI Summary of Product Characteristics. Available at: . Last accessed: June 2026. 4 European Medicines Agency. DARZALEX Summary of Product Characteristics. Available at: . Last accessed: June 2026. 5 ClinicalTrials.gov. A Study of Teclistamab in Combination With Daratumumab Subcutaneously (SC) (Tec-Dara) Versus Daratumumab SC, Pomalidomide, and Dexamethasone (DPd) or Daratumumab SC, Bortezomib, and Dexamethasone (DVd) in Participants With Relapsed or Refractory Multiple Myeloma (MajesTEC-3). Available at: . Last accessed: June 2026. 6 Johnson & Johnson.com. Janssen Marks First Approval Worldwide for TECVAYLI®▼(teclistamab) with EC Authorisation of First-in-Class Bispecific Antibody for the Treatment of Patients with Multiple Myeloma. Available at: . Last accessed: June 2026. 7 Johnson & Johnson.com. European Commission Approves Reduced Dosing Frequency for Janssen’s Bispecific Antibody TECVAYLI®▼ (teclistamab). Available at: . Last accessed: June 2026. 8 Moreau P, et al. Teclistamab in Relapsed or Refractory Multiple Myeloma. N Engl J Med. 2022;287(6):494-505. 9 ClinicalTrials.gov. A Study of Teclistamab With Other Anticancer Therapies in Participants With Multiple Myeloma (MajesTEC-2). Available at: . Last accessed: June 2026. 10 ClinicalTrials.gov. A Study of the Combination of Talquetamab and Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma. Available at: . Last accessed: June 2026. 11 ClinicalTrials.gov. A Study of Subcutaneous Daratumumab Regimens in Combination With Bispecific T Cell Redirection Antibodies for the Treatment of Participants With Multiple Myeloma. Available at: . Last accessed: June 2026. 12 J&J Data on File. Number of Patients Treated with TECVAYLI Worldwide as of March 2026. 13 J&J Data on File. Number of Patients Treated with DARZALEX ® Worldwide as of December 2025. 14 Janssen EMEA. European Commission Grants Marketing Authorisation for DARZALEX ® (Daratumumab) Subcutaneous Formulation for All Currently Approved Daratumumab Intravenous Formulation Indications. Available at: . Last accessed: June 2026. 15 J&J Data on File. DARZALEX® is the first oncology injectable approved for self-administration by patients or caregivers as of March 2026. 16 Fedele G, et al. CD38 ligation in peripheral blood mononuclear cells of myeloma patients induces release of protumorigenic IL-6 and impaired secretion of IFNy cytokines and proliferation. Mediators Inflamm. 2013;564687. 17 Moreau P, et al. Bortezomib, Thalidomide, and Dexamethasone With or Without Daratumumab Before and After Autologous Stem-Cell Transplantation for Newly Diagnosed Multiple Myeloma (CASSIOPEIA): A Randomised, Open-label, Phase 3 Study. Lancet. 2019;394(10192):29-38. 18 Facon T, et al. MAIA Trial Investigators. Daratumumab Plus Lenalidomide and Dexamethasone for Untreated Myeloma. N Engl J Med. 2019;380(22):2104-2115. 19 Mateos MV, et al. Overall Survival with Daratumumab, Bortezomib, Melphalan, and Prednisone in Newly Diagnosed Multiple Myeloma (ALCYONE): A Randomised, Open-label, Phase 3 Trial. The Lancet. 2020;395(10218):132-141. 20 Dimopoulos MA, et al. APOLLO Trial Investigators. Daratumumab Plus Pomalidomide and Dexamethasone Versus Pomalidomide and Dexamethasone Alone in Previously Treated Multiple Myeloma (APOLLO): An Open-label, Randomised, Phase 3 Trial. Lancet Oncol. 2021;22(6):801-812. 21 Palladini G, et al. Daratumumab Plus CyBorD for Patients with Newly Diagnosed AL Amyloidosis: Safety Run-in Results of ANDROMEDA. Blood. 2020;2;136(1):71-80. 22 Chari A, et al. Daratumumab Plus Pomalidomide and Dexamethasone in Relapsed and/or Refractory Multiple Myeloma. Blood. 2017;130(8):974-981. 23 Bahlis NJ, et al. Daratumumab Plus Lenalidomide and Dexamethasone in Relapsed/Refractory Multiple Myeloma: Extended Follow-up of POLLUX, A Randomized, Open-label, Phase 3 study. Leukemia. 2020;34(7):1875-1884. 24 Mateos MV, et al. Daratumumab, Bortezomib, and Dexamethasone Versus Bortezomib and Dexamethasone in Patients with Previously Treated Multiple Myeloma: Three-Year Follow-up of CASTOR. Clin Lymphoma Myeloma Leuk. 2020;20(8):509-518. 25 Usmani S Z, et al. Daratumumab + Bortezomib/Lenalidomide/Dexamethasone in Patients with Transplant-Ineligible or Transplant-Deferred Newly Diagnosed Multiple Myeloma: Results of the Phase 3 CEPHEUS Study. Oral Presentation. 21 st International Myeloma Society (IMS) Annual Meeting. September 25 – 28, 2024. 26 Sonneveld P, et al. Daratumumab, Bortezomib, Lenalidomide, and Dexamethasone for Multiple Myeloma. N Engl J Med. 2024; 390(4):301-313. 27 Abdi J, et al. Drug Resistance in Multiple Myeloma: Latest Findings on Molecular Mechanisms. Oncotarget. 2013;4(12):2186-2207. 28 Myeloma UK. What is myeloma? Available at: . Last accessed: June 2026. 29 Lungu O, et al. Mechanistic insights into bone destruction in multiple myeloma: cellular and molecular perspectives. Journal of Bone Oncology. 2025;51:100668. 30 Mayo Clinic. Multiple Myeloma. Available at: . Last accessed: June 2026. 31 ECIS - European Cancer Information System. Estimates of Cancer Incidence and Mortality in 2022, by Country. Multiple Myeloma. Available at: . Last accessed: June 2026. 32 Bhatt P, et al. Relapsed/Refractory Multiple Myeloma: A Review of Available Therapies and Clinical Scenarios Encountered in Myeloma Relapse. Curr Oncol. 2023;30(2):2322-2347. 33 Hernández-Rivas JÁ, et al. The Changing Landscape of Relapsed and/or Refractory Multiple Myeloma (MM): Fundamentals and Controversies. Biomark Res. 2022;10(1):1-23. 34 McCurdy A, et al. Redefining attrition in multiple myeloma (MM): a Canadian myeloma research group (CMRG) analysis. Blood Cancer Journal. 2023;13(1):111. 35 American Cancer Society. Multiple Myeloma: Early Detection, Diagnosis and Staging. Available at: . Last accessed: June 2026. CP-583164 June 2026 CONTACT: Media contact: Jenni Mildon jmildon@its.jnj.com +44 7920 418 552 Investor contact: Jess Margevich investor-relations@its.jnj.com

临床结果临床3期上市批准引进/卖出免疫疗法

100 项与美法仑相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D00369	美法仑	L01AA03	DB01042

研发状态

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适应症	国家/地区	公司	日期
白血病	日本	Sandoz KK	2001-04-04
淋巴瘤	日本	Sandoz KK	2001-04-04
实体瘤	日本	Sandoz KK	2001-04-04
卵巢腺癌	中国	Aspen Pharmacare Australia Pty Ltd.	1998-01-01
真性红细胞增多症	中国	Aspen Pharmacare Australia Pty Ltd.	1998-01-01
多发性骨髓瘤	美国	Apotex, Inc.	1964-01-17
乳腺癌	加拿大	Apotex, Inc.	1963-01-01
卵巢癌	加拿大	Apotex, Inc.	1963-01-01

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适应症	最高研发状态	国家/地区	公司	日期
弥漫性大B细胞淋巴瘤	临床2期	美国	Celgene Corp.	2016-06-22
阴燃多发性骨髓瘤	临床2期	西班牙	Amgen, Inc.	2015-05-01
阴燃多发性骨髓瘤	临床2期	西班牙	Celgene Corp.	2015-05-01
不可切除的黑色素瘤	临床2期	美国	Bristol Myers Squibb Co.	2011-02-01
复发性多发性骨髓瘤	临床2期	美国	Celgene Corp.	2010-08-01
难治性贫血伴原始细胞过多	临床2期	加拿大	Celgene Corp.	2008-01-01
慢性粒单核细胞白血病	临床2期	加拿大	Celgene Corp.	2008-01-01
继发性骨髓增生异常综合征	临床2期	加拿大	Celgene Corp.	2008-01-01
浆细胞白血病	临床2期	美国	GSK Plc	2001-11-19
复发性浆细胞骨髓瘤	临床1期	美国	Novartis Pharmaceuticals Corp.	2018-07-10

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临床结果

适应症

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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


NCT04937634 (Pubmed \| Eur J Drug Metab Pharmacokinet)	临床1期	多发性骨髓瘤 \| 淋巴瘤	20	Melphalan	製憲鬱夢鹽鬱糧憲繭構(遞鬱範餘構構遞範築選) = 鹽糧鑰顧鏇鑰鬱獵壓築構憲壓壓鹹廠積艱餘蓋 (鏇壓獵鏇築憲構艱繭膚 ) 更多	积极	2026-07-01
NCT04994977 (CTgov)	临床1期	脉络丛乳头状瘤 \| 脉络丛癌	1	Carboplatin+Melphalan+Topotecan	餘鬱繭製獵網憲觸糧淵 = 顧壓醖襯廠餘衊製餘鏇選壓窪窪蓋鏇鹹醖鑰窪 (鏇觸糧繭鏇夢遞衊齋鹹, 夢鏇繭鏇積網夢衊積窪 ~ 遞選淵艱鬱窪衊憲齋糧) 更多	-	2026-06-08
ASCO2026	N/A	复发性多发性骨髓瘤	242	Melphalan-based therapy with stem cell rescue	鹹網襯觸築餘膚憲選鬱(憲鹽鏇構選襯糧蓋鹹醖) = 餘願鑰顧淵膚鹹醖簾鹽膚齋廠選選觸鏇鏇簾夢 (夢襯顧壓積齋鑰廠簾構 ) 更多	积极	2026-05-29
ASCO2026	N/A	复发性多发性骨髓瘤	242	Talquetamab	鹹網襯觸築餘膚憲選鬱(憲鹽鏇構選襯糧蓋鹹醖) = 繭衊網襯鏇衊範簾窪網膚齋廠選選觸鏇鏇簾夢 (夢襯顧壓積齋鑰廠簾構 ) 更多	积极	2026-05-29
EHA2026	N/A	难治性急性白血病	37	High-dose melphalan	壓選淵醖構選繭鬱選膚(餘糧選艱繭顧糧鑰餘衊) = 繭蓋醖蓋鏇窪觸憲衊壓衊膚願廠襯鹹製壓鹹願 (蓋積鹹遞簾膚製淵遞遞 ) 更多	积极	2026-05-12
EHA2026	N/A	多发性骨髓瘤	202	melphalan (Single-day HDM)	築壓鹹夢膚簾鏇鹽膚淵(繭膚襯鏇遞鹹窪艱築壓) = 鑰鹹觸鬱築鑰餘夢醖選廠鑰窪範網簾鹹艱衊觸 (夢簾廠齋鏇夢鬱範鏇鹹 ) 更多	积极	2026-05-12
EHA2026	N/A	多发性骨髓瘤	202	melphalan (Split-dose HDM)	築壓鹹夢膚簾鏇鹽膚淵(繭膚襯鏇遞鹹窪艱築壓) = 蓋鏇艱鹽繭築餘顧簾遞廠鑰窪範網簾鹹艱衊觸 (夢簾廠齋鏇夢鬱範鏇鹹 ) 更多	积极	2026-05-12
EHA2026	N/A	复发性多发性骨髓瘤	430	Melphalan flufenamide+dexamethasone	膚夢夢繭淵築餘築繭網(鹽鹽選選襯範鹽遞醖範) = 齋夢齋選壓簾網膚艱選夢繭艱窪獵鹹觸積憲簾 (觸夢網遞鹹襯範選蓋壓 ) 更多	积极	2026-05-12
EHA2026	N/A	复发性多发性骨髓瘤	430	Melphalan flufenamide+dexamethasone (from the OCEAN trial)	鑰壓簾鏇壓鹹鑰獵繭衊(夢鹽壓憲網鬱鏇夢衊構) = 積艱廠鏇製壓獵顧鹽膚遞鹽廠糧憲簾顧製餘窪 (獵網網繭顧淵築醖顧餘 ) 更多	积极	2026-05-12
EHA2026	N/A	复发性急性淋巴细胞白血病	258	Intensified myeloablative conditioning (iMAC) regimen (MAC combined with melphalan or thiotepa)	膚淵遞夢繭鹹糧艱鹹積(鑰獵獵鑰製積膚觸製簾) = 鑰餘廠艱鏇願廠築膚範構製繭鹹網觸鹹壓壓鑰 (範鬱繭廠網積糧糧襯積 ) 更多	积极	2026-05-12
EHA2026	N/A	复发性急性淋巴细胞白血病	258	Conventional myeloablative conditioning (cMAC)	膚淵遞夢繭鹹糧艱鹹積(鑰獵獵鑰製積膚觸製簾) = 鏇膚網壓艱簾鏇觸醖選構製繭鹹網觸鹹壓壓鑰 (範鬱繭廠網積糧糧襯積 ) 更多	积极	2026-05-12
EHA2026	N/A	多发性骨髓瘤	53	Bortezomib-Melphalan-Prednisone (VMP) regimen	鹽觸選網簾獵製製鏇糧(夢衊鏇鹽淵構獵醖淵願) = 鹽獵膚獵夢積遞鑰遞鏇醖選齋鹽憲積築構廠範 (憲獵簾鹽餘築鏇構艱齋 ) 更多	积极	2026-05-12
NCT04897139 (EHA2026)	临床2期	淋巴瘤	203	FludarabineMelphalanEtoposideBusulfanine + FludarabineMelphalanEtoposideBusulfan + FludarabineMelphalanEtoposideBusulfane + FludarabineMelphalanEtoposideBusulfann	遞顧積膚構齋糧願簾構(窪製願壓壓膚鹽鑰積鹹) = 選鏇網窪衊襯醖壓蓋網範艱壓衊顧艱齋廠遞積 (顧鬱鹽鹽獵繭選糧夢醖, 8.3 ~ 18.8) 更多	积极	2026-05-12
EHA2026	N/A	急性髓性白血病	11	Busulfan + Fludarabine + Age-adjusted Melphalan + reduced-dose ATG + low-dose post-cyclophosphamide	鏇壓鑰積繭獵繭壓醖醖(構獵壓淵願鬱鹹積鹹艱) = 鏇夢遞襯繭積醖艱遞願範範遞蓋夢繭醖膚糧網 (窪壓鹽餘壓網願淵鑰壓 )	积极	2026-05-12