Phase II Double-Blinded Placebo-Controlled Randomized Trial of CSF-1R Inhibitor, Axatilimab for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation (ABRAXAS)

This phase II trial studies how well adding axatilimab to standard of care (SOC) therapy works in preventing graft versus host disease (GVHD) following allogeneic hematopoietic stem cell transplantation (HCT) in patients with hematologic cancer. Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical, donor. This is often a sister or brother, but could be an unrelated donor. Sometimes the transplanted cells from a donor can attack the body's normal cells, causing GVHD. Symptoms of GVHD can include yellowing of the skin, mucous membranes, and eyes, skin rash or blisters, dry mouth, or dry eyes. Typically, drugs such as cyclophosphamide, tacrolimus, and mycophenolate mofetil are given after the transplant to help stop GVHD from happening, but these current therapies may negatively affect patient quality of life and newer treatment strategies are needed. Axatilimab is a monoclonal antibody. 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), which may prevent GVHD from developing. Adding axatilimab to SOC therapy may be more effective in preventing GVHD following allogeneic HCT in patients with hematologic cancer.

开始日期2026-06-01

申办/合作机构

Mayo Clinic

100 项与艾克利单抗相关的临床结果

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100 项与艾克利单抗相关的转化医学

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100 项与艾克利单抗相关的专利（医药）

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项与艾克利单抗相关的文献（医药）

2026-06-04·BLOOD

CSF-1R inhibition and lenalidomide synergize to promote myeloma control after autologous stem cell transplantation

Article

作者: Sekiguchi, Tomoko ; Ho, Kenneth ; Lyons, Justina M. ; Adams, Rachael C. ; Minnie, Simone A. ; Green, Damian J. ; Comstock, Melissa L. ; Nemychenkov, Nicole S. ; Legg, Samuel R. W. ; Spencer, Andrew ; Hill, Geoffrey R. ; Koyama, Motoko ; Boiko, Julie R. ; Schmidt, Christine R. ; Ensbey, Kathleen S.

Abstract:

Autologous stem cell transplantation (ASCT) with maintenance lenalidomide remains the mainstay of consolidation therapy for eligible patients with multiple myeloma, but preventing disease relapse remains a critical unmet need. Here, we investigated whether immunosuppressive myeloid populations in bone marrow correlated with ASCT outcomes. We identified a subset of CD64+CD169+CD163+ macrophages that expressed CSF-1R, PD-L1, and CD155 and were expanded in patients who relapsed after ASCT. Using a preclinical ASCT model with suboptimal endogenous antimyeloma activity, we demonstrated that although neither CSF-1R inhibition nor lenalidomide monotherapy significantly improved outcomes, their combination synergistically attenuated disease progression and prolonged survival. Single-cell RNA sequencing revealed that lenalidomide expanded natural killer (NK)–like CD8+ T cells but paradoxically also increased the frequency of Csf1r+ macrophages. Cell-cell communication analyses identified Csf1r+ macrophages as suppressors of these NK- and effector-like phenotypically exhausted (Tphex) CD8 T-cell populations through CD94/NKG2A and PD-L1/PD-1, respectively. CSF-1R blockade depleted these immunosuppressive macrophages, which correlated with decreased expression of inhibitory receptors and enhanced expression of activation markers in Tphex. Given the US Food and Drug Administration approval of axatilimab for chronic graft-versus-host disease, combining CSF-1R blockade with lenalidomide maintenance represents a readily testable strategy to improve progression-free survival after ASCT.

2026-06-01·INTERNATIONAL JOURNAL OF HEMATOLOGY

Advances in treatment of chronic graft-versus-host disease

Review

作者: Morishita, Takanobu ; Inamoto, Yoshihiro

Chronic graft-versus-host disease (cGVHD) is a leading cause of late morbidity and mortality after allogeneic hematopoietic cell transplantation. Corticosteroids remain the standard first-line therapy; however, many patients develop steroid-refractory or steroid-dependent disease, underscoring the need for more effective and better-tolerated treatments. Ruxolitinib has emerged as the most evidence-supported option for steroid-refractory cGVHD, with the phase 3 REACH3 trial demonstrating higher response rates, durable disease control, and clinically meaningful improvements in symptom burden compared with best available therapy. Belumosudil and axatilimab have also shown encouraging efficacy and safety in heavily pretreated populations. The addition of novel agents to standard corticosteroid-based therapy has been explored in clinical trials. Interest in combination strategies, such as ruxolitinib with extracorporeal photopheresis or belumosudil, is increasing, though prospective studies are required to define their role. Key challenges include optimizing long-term safety, mitigating infectious complications, and preserving the graft-versus-leukemia effect. This review summarizes current therapeutic strategies and discusses evolving treatment algorithms, emphasizing practical considerations in therapy selection. Approaches targeting specific pathogenic mechanisms, combining agents with distinct mechanisms of action, and incorporating biomarker-driven strategies are expected to further improve outcomes and quality of life for patients with cGVHD.

2026-03-04·EXPERT OPINION ON BIOLOGICAL THERAPY

An evaluation of axatilimab for the treatment of chronic graft-versus-host disease

Review

作者: Banigallapati, Sriram ; Chhabra, Saurabh

INTRODUCTION:

Chronic graft-versus-host disease (cGVHD), a frequent, debilitating autoimmune-like syndrome affecting allogeneic transplant recipients begins with inflammatory response to per-transplant tissue injury which evolves into chronic inflammation, T- and B-cell dysregulation, and aberrant tissue repair and fibrotic reaction. Monocytes and macrophages contribute to multiorgan inflammation and fibrosis that are hallmarks of cGVHD.

AREAS COVERED:

Axatilimab is a high-affinity anti-CSF-1 R humanized immunoglobulin G4 monoclonal antibody that blocks ligand binding to CSF-1 R and downregulates development and differentiation of pathogenic monocyte-derived macrophages. A phase 1/2 study, and subsequently a phase 2 randomized trial that evaluated axatilimab in patient with refractory cGVHD, reported an ORR of 50-74%. Toxicity was dose-dependent. Based on the Phase 2 results, the lowest dosage, 0.3 mg/kg every 2 weeks, was identified as appropriate for the indication. Here, we examine clinical development of axatilimab leading up to its approval by the Food and Drug Administration for treatment of cGVHD after failure of at least two prior therapies.

EXPERT OPINION:

Axatilimab has demonstrated safety, tolerability, and efficacy in clinical trials; however, many questions remain unanswered including long-term safety data, efficacy when compared to other cGVHD therapies, risks and benefits of axatilimab in combination, and role in earlier lines of cGVHD treatment.

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项与艾克利单抗相关的新闻（医药）

2026-07-01

·BioSpace

Step Pharma announces the appointment of Neil Gallagher as Non-Executive Director

Neil Gallagher, MD, PhD, joins with over 25 years clinical development experience, having contributed to multiple commercial therapeutics across haematology and oncology His appointment will strengthen Step Pharma’s board as it advances dencatistat across three clinical programmes for essential thrombocythaemia (ET), lymphoma and solid tumours. Saint-Genis-Pouilly, France, 1 July 2026 – Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces the appointment of Neil J. Gallagher, MD, PhD, as Non-Executive Director effective immediately. He will support the Company as it continues to advance its first-in-class CTPS1 inhibitor, dencatistat, as a treatment for essential thrombocythaemia, lymphoma and solid tumours. Neil has worked across haematology and oncology for over 25 years, having held various senior executive positions at several global pharmaceutical companies including Amgen, AstraZeneca, Novartis and AbbVie, where as Chief Medical Officer he led the development organisation through multiple regulatory approvals in immunology, oncology, neuroscience and eye care. He currently serves as a Non-Executive Director at several public and private oncology companies, including Foghorn Therapeutics (NASDAQ: FHTX) and iOnctura, and as a Board Advisor to Nouscom. Over the course of his executive career, Dr Gallagher has built an exceptional track record of bringing innovative oncology therapies to patients, including venetoclax during his time as CMO at AbbVie and overseeing the FDA approvals of revumenib and axatilimab as President, Head of R&D at Syndax Pharmaceuticals. Neil received his MD from Trinity College Dublin, Ireland, alongside a PhD in Cancer Biology from the University of Birmingham, UK. He is also a Member of the Royal College of Obstetricians and Gynaecologists (MRCOG). Andrew Parker, Chief Executive Officer, Step Pharma, commented: “Neil's deep expertise in successfully leading complex development programmes through to regulatory approval across haematology and oncology will be invaluable as we continue to advance our first-in-class CTPS1 inhibitor, dencatistat, for essential thrombocythaemia, lymphoma and solid tumours. His appointment comes at a pivotal moment for the Company, and we are delighted to welcome him to the Step Pharma board.” Neil Gallagher, newly appointed member of the board, Step Pharma, added : “I am delighted to join the Step Pharma board at such an exciting point in the Company’s evolution. The advancement of dencatistat into three parallel programmes reflects the strength of Step Pharma’s innovative ‘pipeline in a product’ strategy. This is a truly efficient approach to clinical development that has the potential to deliver new treatment options across multiple indications where unmet need remains high. I look forward to applying my experience in haematology and oncology drug development to help make this a reality for patients.” -Ends- About Step Pharma Step Pharma’s goal is to bring about a step change in how cancer is treated with targeted therapies that kill cancer cells and leave healthy cells unharmed. The Company is the world leader in CTPS1 inhibition, a new approach with the potential to yield highly selective, safe and effective treatments for both blood cancers and solid tumours. Step Pharma was founded in 2014 by Kurma Partners, the Imagine Institute and Sygnature Discovery, based on the scientific discoveries of Prof. Alain Fischer and Dr Sylvain Latour. Step Pharma is based in Saint-Genis-Pouilly, France, and is supported by a strong syndicate of investors including Kurma Partners, V-Bio Ventures, Bpifrance (Fonds Biothérapies Innovantes et Maladies Rares and InnoBio2 Fund), Pontifax, Hadean Ventures, Sunstone Life Science Ventures, Inserm Transfert Initiative, Idinvest, Sygnature Discovery and the Imagine Institute. More information on the company can be found at . For more information please contact: Step Pharma Andrew Parker, Chief Executive Officer contact@step-ph.com Media Relations ICR Healthcare Amber Fennell, Namrata Taak, Phillip Marriage Tel. +44 (0) 20 3709 5700 steppharma@icrhealthcare.com

高管变更上市批准

2026-07-01

·BioSpace

Syndax Pharmaceuticals Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4) - July 1, 2026

NEW YORK, July 01, 2026 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that on July 1, 2026, the Company granted inducement awards to purchase up to 250,700 shares of common stock to eight new employees under the Company's 2023 Inducement Plan. The stock options will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the vesting commencement date and 1/48th of the underlying shares vesting monthly thereafter over 36 months, subject to the employee's continued service relationship with Syndax through the applicable vesting dates. About Syndax Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company advancing innovative cancer therapies. Highlights of the Company's pipeline include Revuforj ®  (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit  or follow the Company on  X  and  LinkedIn . Syndax Contact Sharon Klahre Syndax Pharmaceuticals, Inc. sklahre@syndax.com Tel 781.684.9827

2026-06-17

·头颈健康之声

2026年3月儿童肿瘤内科治疗进展综述：精准医疗时代的范式转移与生存质量重塑

点击蓝字关注我们 2026年3月儿童肿瘤内科治疗进展综述：精准医疗时代的范式转移与生存质量重塑 2026年3月，全球儿童肿瘤学领域在内科治疗方面迎来了多项具有分水岭意义的研究成果。这些进展集中发表于《新英格兰医学杂志》（NEJM）、《兰开特》（Lancet）系列、《临床肿瘤学杂志》（JCO）、《美国医学会杂志》（JAMA）系列以及《肿瘤学年鉴》（Annals of Oncology）等顶级期刊，并深度整合了第52届欧洲血液与骨髓移植学会（EBMT）年会的关键报告。当前，儿童肿瘤治疗的逻辑正发生根本性转变：从单纯追求治愈率（目前已达70-80%）转向在维持高治愈率的同时，通过"去化疗"模式减少远期毒性，利用精准分子靶向解决难治病例，以及通过全周期生存管理提升患儿的长期生活质量。本月最受关注的进展包括：费城染色体阳性急性淋巴细胞白血病（Ph+ ALL）一线治疗中"去化疗"模式的成功验证；儿童单倍体造血干细胞移植中供者选择标准的重新界定；以及通过肠道微生物组干预解决移植后难治性移植物抗宿主病（GVHD）的突破性三期临床数据。在实体瘤领域，神经母细胞瘤的二线免疫化疗方案、髓母细胞瘤的新型基质微环境靶点、以及肝母细胞瘤的分子分型演进，均展示了内科治疗在精准化层面的深度拓展。此外，学术界开始反思全球范围内临床试验可及性的不平等问题，并尝试利用人工智能（AI）规范生物标志物的开发。本综述将对这些进展进行详尽剖析，旨在为儿科肿瘤临床医生与研究者提供深度学术参考。血液系统肿瘤：精准干预与移植范式的重塑费城染色体阳性急性淋巴细胞白血病的"去化疗"革命在传统的儿童白血病治疗中，费城染色体阳性急性淋巴细胞白血病（Ph+ ALL）一直被视为高危亚型，其分子标志物为 BCR-ABL1融合基因。既往标准方案依赖于高强度化疗联合酪氨酸激酶抑制剂（TKI），并辅以造血干细胞移植。然而，在2026年3月的EBMT年会上，浙江大学医学院附属第一医院黄河教授团队发布的报告（IS01-4）展示了一种极具颠覆性的"无化疗"策略 1。该方案通过第二代TKI达沙替尼（Dasatinib）联合序贯CD19和CD22嵌合抗原受体T细胞（CAR-T）疗法。研究结果显示，在新诊断的Ph+ ALL患者中，中位随访23.9个月时，2年总生存率（OS）和2年无白血病生存率（LFS）均高达 98% 1。这一数据显著优于传统移植方案的长期预后，且避免了移植相关的非复发死亡风险。在该临床试验中，患者首先接受为期2周的达沙替尼配合长春新碱和地塞米松的诱导治疗，随后进入CAR-T序贯治疗阶段。诱导后的完全分子学缓解（CMR）率仅为 31%，但在CD19 CAR-T治疗后激增至 85%，在序贯CD22 CAR-T后进一步巩固。这种序贯回输策略有效降低了单一靶点缺失导致的抗原逃逸风险 2。下表详细对比了该去化疗方案与传统模式的关键指标：评估维度传统高强度化疗+TKI+移植达沙替尼联合序贯CAR-T (去化疗) 2年总生存率 (OS) 73% (视风险等级) 98% 1 2年无白血病生存率 (LFS) 波动较大 98% 3 微小残留病 (MRD) 阴性率移植前约为65% CD19 CAR-T后达 86% (p＜0.01) 2 主要毒副作用严重骨髓抑制、感染、GVHD 1-2级CRS (细胞因子释放综合征) 1 远期生活质量生长发育受限、不育风险高显著改善，靶向性强这种模式的成功提示，Ph+ ALL的内科治疗正进入"分子手术"时代。通过精准打击融合基因产生的异常蛋白和恶性B细胞表面抗原，化疗药物在白血病治疗中的角色正在从"主角"转变为"过渡性工具" 1。儿童单倍体移植供者选择的新法则：年龄与亲缘关系的权衡随着单倍体相合造血干细胞移植（Haplo-HCT）的广泛开展，如何从众多家属供者中筛选最佳对象成为临床痛点。2026年3月发表于《JCO》的一项由Nicole Liberio领导的大型队列研究，为儿童患者的供者选择提供了具有法律效力般的证据支持 4。该研究分析了1,069名19岁以下接受HCT的患儿，结果显示供者年龄是预测急慢性GVHD的最强因素。与18岁以下的同胞或家属供者相比，18-35岁的供者导致II-IV级急性GVHD（aGVHD）的风险增加了2.5倍（OR 2.5），而35岁以上供者的这一风险比例达到 2.7倍 4。更具洞察力的发现在于供者身份对慢性GVHD（cGVHD）的影响。研究明确指出，母亲作为供者是cGVHD的独立危险因素。即便在排除年龄干扰后，母亲供者的cGVHD风险仍比同胞供者高出3.35倍（HR 3.35），也显著高于父亲供者（HR 2.3）5。这可能与母亲体内存留的针对胎儿抗原的微嵌合细胞在移植后被激活有关。下表总结了基于JCO最新研究的儿童单倍体移植供者优先级建议：优先级供者特征 aGVHD (II-IV级) 风险 cGVHD (2年) 风险循证医学依据第一选择 <18岁同胞供者 15% (参考值) 16% (参考值) 4 第二选择 18-35岁男性供者 (如年轻父亲或堂表亲) 23% (OR 2.5) 26% (HR 2.1) 6 第三选择 >35岁男性供者 (如父亲) 31% (OR 1.7) 30% (HR 2.2) 4 应谨慎考虑母亲供者 (无论年龄) 高 (HR 2.05-2.53) 极高 (HR 3.10-3.35) 5 此外，针对少数裔患者寻找匹配供者的难题，研究强调了单倍体供者的可及性优势（约 70% 的患者有潜在供者），并指出在PTCy（移植后环磷酰胺）方案的保护下，错配不全相合供者（MMUD）已能获得与全相合供者（MUD）相当的生存结果，这为临床实践提供了更大的灵活度 7。 GVHD 治疗的生物突破：微生物组与靶向新药在EBMT 2026大会的总统论坛上，肠道微生物群药物MaaT013（Xervyteg）的ARES三期临床研究数据引发轰动。针对类固醇耐药且经鲁索替尼（Ruxolitinib）治疗失败的重度胃肠道急性GVHD患者，MaaT013展示了突破性的疗效 10。 MaaT013是一种全相合、多供者汇集的微生物组干预药物，旨在重建受损的肠道生态。在66名重症患者中，该药实现了超过 46% 的客观缓解率（ORR），且大部分为完全缓解（CR）或非常好的部分缓解（VGPR）10。这标志着aGVHD治疗从单纯的免疫抑制（如鲁索替尼、激素）转向了微环境修复。针对慢性GVHD，本月同样有重要新进展： Belumosudil (ROCK2抑制剂)：在12岁以下儿童中的回顾性分析（OS16-01）显示，该药对难治性cGVHD具有显著疗效，且安全性良好，特别是在改善纤维化病变方面表现突出 10。 Axatilimab (CSF-1R抗体)：与鲁索替尼联合使用的二期研究（OS16-04）更新显示，该方案能更早地干预cGVHD的进展，尤其是在皮肤和肺部受累的患者中 13。内皮激活指数 (EASIX)：一项1月发表但在3月获得临床广泛应用的中国研究（Shaoyan Hu等）证明，在儿童单倍体移植第7天的改良EASIX得分可精准预测患者的非复发死亡率（NRM）及生存获益，为早期干预提供了生物标志物依据 14。 CAR-T 治疗的远期挑战与幸存者健康管理 CAR-T细胞疗法已成为复发/难治性B-ALL的二线或三线标准治疗。2026年3月，学术界对该疗法的关注重点正从"获取缓解"向"维持缓解"及"幸存者质量"转移。关于缓解的持久性，B细胞 aplasia（B细胞缺乏状态）被确认为CAR-T细胞功能性持续的间接标志 3。然而，长期B细胞缺乏带来的免疫缺陷和感染风险需要精密的监测。更为敏感的话题是性健康和生育力：发表于本月的纵向研究显示，超过50%的青少年和年轻成人（AYA）肿瘤幸存者存在长期性功能障碍，且随时间推移未见显著改善 15。尽管目前CAR-T对生育能力的具体影响尚无大规模数据，但已有CAR-T治疗后产下健康胎儿的案例报道，这为患儿家庭提供了希望，同时也凸显了早期生殖内分泌转诊的重要性 15。实体瘤：化疗免疫联合与分子机制的深度挖掘神经母细胞瘤：化疗免疫方案的二线进阶与耐药机制神经母细胞瘤（NB）作为儿科实体瘤的"无冕之王"，其高危病例的复发率始终居高不下。2026年3月13日在线发表于《Pediatric Blood Cancer》的研究，详细评估了托泊替康/环磷酰胺联合地妥昔单抗（T/C/DIN）在复发/难治性高危神经母细胞瘤（r/r HRNB）中的表现 16。在54名入组患儿中，总客观缓解率达到 44%，这一数据对于已经历多轮治疗的r/r HRNB患者而言具有重要的临床意义 16。研究发现，既往对伊立替康/替莫唑胺/地妥昔单抗（I/T/DIN）方案有反应的患者，切换至T/C/DIN方案后的缓解率更高（61% vs 31%）。即便是在I/T/DIN治疗期间出现疾病进展（PD）的患者，接受T/C/DIN方案后仍有 38% 的客观缓解率，这证明了化疗骨架替换在免疫联合方案中的协同价值 16。在分子机制层面，研究揭示了NB间充质细胞（NB-MES）在免疫逃逸中的关键作用。NB-MES细胞不仅表现出对自然杀伤（NK）细胞杀伤的强抵抗性，还能通过Adrenergic-to-Mesenchymal Transition（AMT）过程下调GD2表达，从而对抗GD2抗体产生耐药性 17。这一发现为未来的临床试验提供了新思路：可能需要通过表观遗传调节药物将肿瘤细胞锁定在易受免疫攻击的"肾上腺素能"状态。下表总结了本月关于高危神经母细胞瘤化疗免疫方案的临床疗效数据：方案构成研究对象样本量 (n) 客观缓解率 (ORR) 主要毒副作用关键文献 T/C/DIN 复发/难治性高危NB 54 44% 3/4级贫血 (37%)、中性粒细胞减少 16 I/T/DIN (诱导后巩固) 高危NB (ASCT后) 40 42% (2年EFS) 胃肠道毒性、发热性中性粒细胞减少 19 Fimepinostat 复发实体瘤/脑瘤 N/A 缺乏显著临床活性 N/A 19 中枢神经系统肿瘤：髓母细胞瘤的新靶点与个体化放疗髓母细胞瘤（MB）的治疗正处于放疗减毒与靶向进攻的交汇点。2026年3月，哈佛医学院团队在《Cell》发布的最新成果引起了基础科研领域的广泛关注。研究识别出胎盘生长因子（PlGF）及其受体神经纤毛蛋白1（Nrp1）是所有四种分子亚型（WNT、SHH、Group 3、Group 4）髓母细胞瘤共同的生长命门 20。该研究揭示了MB细胞与基质微环境的独特交互：MB细胞释放Shh蛋白，诱导邻近基质细胞产生PlGF，PlGF再反向作用于肿瘤细胞上的Nrp1受体，驱动肿瘤的扩散和进展。在多个小鼠模型中，阻断该通路的抗体表现出显著的抑瘤效果，且不影响正常发育，这使其有望成为替代高毒性化疗的潜力候选药物 20。在临床治疗层面，一项针对5岁以下MB患儿的Phase 3试验（Standard Regimen Comparison）对比了"Head Start 4"与"HIT-SKK"方案。该研究的核心目标是通过大剂量的诱导化疗联合自救外周血干细胞支持，力争使患儿彻底免于颅脊放射（CSI），从而最大程度保护其认知功能和发育水平 22。肝脏肿瘤：肝母细胞瘤与肝细胞癌的分子连续体肝母细胞瘤（HB）通常多见于3岁以下幼儿，但近年来一类具有"肝细胞癌特征"的肝母细胞瘤（HBC）受到高度关注。2026年3月12日发表的一项跨中心研究揭示了这类罕见肝脏肿瘤的分子图谱 24。 HBC被证实起源于肝干细胞，其生存率远低于普通型HB（5年OS 58% vs 85%）24。更重要的是，研究发现HBC处于HB向肝细胞癌（HCC）演进的"分子连续体"上。这一发现具有显著的临床指导意义：对于HBC患儿，传统的化疗方案可能并不足够，这类患儿在接受肝移植治疗后预后更好。此外，针对WNT/β-catenin通路和mTOR通路的靶向抑制剂（如Everolimus）正在作为HBC和复发HB的补充方案进行探索 24。下表总结了目前儿科肝脏肿瘤的分类特征与预后：肿瘤亚型典型发病年龄 5年总生存率 (OS) 分子特征关键内科策略典型肝母细胞瘤 (HB) <3岁约85% WNT通路激活顺铂为基础的化疗肝母细胞瘤伴癌特征 (HBC) AYA/较大儿童约58% 肝干细胞起源强化化疗、考虑早期移植 24 肝细胞癌 (HCC) 青少年/成人较低异质性高、免疫冷肿瘤索拉非尼、PD-L1抑制剂探索 26 其他实体瘤的内科进展：肾母细胞瘤与肉瘤肾母细胞瘤（Wilms瘤）的治疗已非常成熟，但对于复发难治病例，多靶点激酶抑制剂卡博替尼（Cabozantinib）正在进行二期临床试验评估，旨在为化疗失败的患儿提供新的挽救手段 28。在骨肿瘤领域，骨肉瘤和尤文肉瘤的生存率瓶颈亟待突破。目前的研究方向集中在利用cDNA监测肿瘤微小残留病（MRD），以及探索针对ALK/ROS1和FGFR突变的精准靶向药物（如Ensartinib, Erdafitinib）28。虽然这些突变在肉瘤中频率较低，但在复发病例中，通过Pediatric MATCH试验匹配相应的靶向药已成为新的标准流程 28。支持治疗与生存质量：内科干预的温度顺铂相关听力损失的化学保护：PEDMARK的里程碑顺铂引起的老年性耳聋般的永久性听力损失，曾是实体瘤患儿一生的阴影。2026年3月，Fennec Pharmaceuticals宣布了PEDMARK（硫代硫酸钠）的最新进展。在SIOPEL 6等三期临床试验的支持下，PEDMARK被证实能显著降低顺铂诱导的耳毒性风险 29。 PEDMARK通过在顺铂给药6小时后中和残留的顺铂毒性碎片，从而保护耳蜗毛细胞。目前的适应症已扩展至1个月及以上的局部性、非转移性实体瘤患儿 29。然而，临床医生需关注该药引起的电解质紊乱（如高钠血症、低钾血症），在用药前后需严格监测生化指标 29。精准诊断指引：JCO Somatic Testing 2026 内科治疗的精准性有赖于检测的规范化。2026年3月，ASCO联合多家机构更新了《实体瘤体细胞检测指南》。指南强调，对于晚期和复发性儿童肿瘤，应常规开展组织无关性（Tissue-Agnostic）检测，重点关注NTRK融合、RET融合及HER2扩增等可靶向位点 30。对于血液系统肿瘤，指南特别指出了B细胞前体ALL中体细胞基因组监测对指导精准免疫治疗的关键作用 30。全球儿科肿瘤学研究的现状与公平性发表于《JAMA Network Open》的一项具有国际影响力的研究（2026年3月），揭示了全球儿科肿瘤临床试验布局的严重失衡。在分析的5,645项儿科癌症试验中，超过80%的试验由高收入国家（HIC）赞助开展，而低收入国家（LIC）仅贡献了不足 2% 31。这种鸿沟导致了一个严重的临床悖论：虽然目前的研究推动了医学前沿（如CAR-T、靶向药），但这些昂贵的方案在疾病负担最重的低资源地区完全无法落地。数据还显示，低中收入国家的临床试验多集中在"支持治疗"而非"肿瘤根治"，这种研究结构的差异反映了基础医疗条件的限制 31。学术界呼吁建立更加包容的全球科研共享机制，促进前沿药物在发展中国家的临床验证。下表直观展示了临床试验全球分布的差异：国家收入分类 (World Bank) 试验占比 (%) 研究重点方向结果发表率 (10年) 关键来源高收入国家 (HIC) 72% 早期药物研发、肿瘤定向治疗较高 31 中高收入国家 (UMIC) 17% 临床二/三期试验较高 31 低中收入国家 (LMIC) 9% 支持治疗、晚期临床管理较低 31 低收入国家 (LIC) 2% 基础临床管理、流行病学极低 31 总结与展望 2026年3月的研究进展为儿科肿瘤内科治疗勾勒出一幅宏大的蓝图：精准化与去毒化正成为不可逆转的双重趋势。血液系统肿瘤通过TKI联合免疫细胞疗法的模式，已经实现了部分亚型的"化疗自由"；移植医学通过对供者免疫特征的精细解构，极大降低了长期并发症的风险。实体瘤领域虽然依旧面临耐药性的挑战，但通过对微环境和分子连续体的深入理解，我们正逐步打破"一刀切"的治疗僵局。未来的发展方向将集中在三个维度：内科治疗的极致前移。如Ph+ ALL所示，将最具威力的靶向和免疫药用于一线，可能比复发后再用获益更大、毒性更小。生存质量的内科化干预。从听力保护、生育力保存到神经认知功能的维持，支持治疗正在从"配角"进化为与抗癌治疗同等地位的"生命伴侣"。人工智能赋能的动态诊断。通过AI对MRD、影像学及多组学数据的整合，实现对疗效的实时监测和方案的动态微调。儿科肿瘤学的最终目标不仅仅是让患儿"活下来"，更是让他们作为一个完整、健康的人"走下去"。2026年3月的这些科学基石，正让我们离这个目标越来越近。引用的著作 1. 2026 EBMT丨浙大一院血液骨髓移植中心1项大会报告、2项特邀报告 ..., 访问时间为四月10,2026，https://ihemato.com/Detail?id=1165 2. Phase II Study of BCMA Chimeric Antigen Receptor T-Cell Therapy in Patients With Newly Diagnosed Multiple Myeloma Ineligible for or Not Proceeding to Autologous Stem-Cell Transplantation (CAREMM-001) - ResearchGate, https://www.researchgate.net/publication/... 3. Maintenance after CAR T? Are we there yet? Reducing the risk of relapse after loss of anti-CD19 CAR T - cell persistence in ALL | Request PDF - ResearchGate, 4. Optimizing Haploidentical Donor Selection for Pediatric Hematopoietic Cell Transplant - ASCO Publications, https://ascopubs.org/doi/pdf/10.1200/JCO-25-01591 5. Optimizing Haploidentical Donor Selection for Pediatric ... ，https://ascopubs.org/doi/10.1200/JCO-25-01591 6. Optimizing Haploidentical Donor Selection for Pediatric Hematopoietic Cell Transplant | Request PDF - ResearchGate。 7. NMDP(SM) Advances Cell Therapy Research in Clinical Practice and Patient Outcomes at EBMT 2026 - BioSpace。 8. NMDP(SM) Advances Cell Therapy Research in Clinical Practice and Patient Outcomes at EBMT 2026 | Markets Insider。 9. Re-Evaluating Donor Selection Priorities for Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide | JCO Oncology Practice - ASCO Publications。 10. What's new in GVHD? Insights from the EBMT Annual Meeting 2026 | VJHemOnc。 11. March 31, 2026: MaaT Pharma Announces 2025 Annual Results and Provides Business Updates。 12. REGISTRATION DOCUMENT - MaaT Pharma。 13. EBMT 2026 | Top abstracts: What's hot in GvHD?。 14. Modified EASIX score on day 7 predicts survival and non-relapse mortality in pediatric acute leukemia undergoing haploidentical stem cell transplantation - ResearchGate。 15. Long-Term Survivorship After Chimeric Antigen Receptor T-Cell Therapy for Hematologic Malignancies | Journal of Clinical Oncology - ASCO Publications。 16. Efficacy and Tolerability of Topotecan/Cyclophosphamide/Dinutuximab in Relapsed and Refractory High-Risk Neuroblastoma: A Multi-Institutional Retrospective Study - ResearchGate。 17. Efficacy and Tolerability of Topotecan/Cyclophosphamide ... - PubMed。 18. Topotecan injection - PrescriberPoint。 19. Rochelle Bagatell's research works | University of Pennsylvania and other places - ResearchGate。 20. New Target in Childhood Brain Cancer | Harvard Medical School。 21. New Developments in the Pathogenesis, Therapeutic Targeting, and Treatment of Pediatric Medulloblastoma - PubMed。 22. Standard Regimen Comparison for Medulloblastoma · Info for Participants - Clinical Trials。 23. The Management of Children with Pre-School Medulloblastoma -- A Review of Four Decades of Multi-Disciplinary and Multi-Institutional Collaboration. 24. Study reveals how a rare pediatric liver cancer emerges and its response to therapy, https://blogs.bcm.edu/2026/03/12/from-the-labs-study-reveals-how-a-rare-pediatric-liver-cancer-emerges-and-its-response-to-therapy/ 25. Pediatric Hepatoblastoma: From Developmental Molecular Mechanisms to Innovative Therapeutic Strategies - MDPI。 26. Exploring the optimal regimen in advanced hepatocellular carcinoma: a protocol of individual patient data network meta-analysis - Frontiers。 27. Liver Cancer clinical trials at University of California Health。 28. UCSF Hepatoblastoma Clinical Trials for 2026 --- San Francisco Bay Area。 29. Fennec Pharmaceuticals Announces Investigator-Sponsored Study to Be Conducted by University of Arizona Cancer Center - BioSpace。 30. ARCHIVED Somatic Tumor Testing 2025-11-15 (updated 2026-01-01 to 04-01) | Carelon Clinical Guidelines。 31. The Global Clinical Trial Landscape for Children and Adolescents With Cancer - PubMed。关注头颈健康之声内容丨李祥攀编辑丨陈蓉审核丨李祥攀/陈平/许秋妮了解更多肿瘤科普知识,请关注武汉大学人民医院头颈肿瘤科微信公众号 “头颈健康之声” 地址：武汉市洪山区白沙四路与光霞路交叉路口武汉大学人民医院洪山院区5号楼4楼健康热线：027-88041911转82550、82551

100 项与艾克利单抗相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D11952	-	-	DB16388

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适应症	国家/地区	公司	日期
慢性移植物抗宿主病	美国	Incyte Corp.	2024-08-14

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适应症	最高研发状态	国家/地区	公司	日期
特发性肺纤维化	临床3期	美国	Syndax Pharmaceuticals, Inc.	2022-09-16
BCR/ABL阴性非典型性慢性髓系白血病	临床2期	美国	Incyte Corp.	2024-08-02
慢性粒单核细胞白血病	临床2期	美国	Incyte Corp.	2024-08-02
骨髓增生异常综合征	临床2期	美国	Incyte Corp.	2024-08-02
真性红细胞增多症	临床2期	美国	Incyte Corp.	2024-08-02
原发性骨髓纤维化	临床2期	美国	Incyte Corp.	2024-08-02
难治性慢性粒细胞白血病	临床2期	美国	Incyte Corp.	2024-08-02
难治性髓系肿瘤	临床2期	美国	Incyte Corp.	2024-08-02
原发性血小板增多症	临床2期	美国	Incyte Corp.	2024-08-02
晚期恶性实体瘤	临床2期	美国	Incyte Corp.	2024-05-21

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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


NCT04710576 (AGAVE-201, EHA2026)	临床1期	慢性移植物抗宿主病	239	Axatilimab 0.3 mg/kg Q2W	顧衊構夢窪餘蓋範餘窪(鬱夢衊繭獵廠糧蓋襯夢) = 醖遞淵選鏇艱顧鑰構糧鹹膚襯獵鑰蓋構蓋夢構 (鬱獵觸膚夢簾蓋願顧淵 )	积极	2026-05-12
NCT06388564 (EHA2026)	临床2期	慢性移植物抗宿主病	66	Axatilimab+Ruxolitinib	鏇顧簾夢淵夢選艱艱襯(簾獵糧鑰願糧糧觸獵衊) = 獵網願築窪窪齋積觸網鬱憲襯艱淵網網鬱襯夢 (窪製壓鏇構築築構醖築 ) 更多	积极	2026-05-12
				Ruxolitinib	鏇顧簾夢淵夢選艱艱襯(憲網鏇遞遞憲艱觸鏇積) = 鹹餘鹹憲壓願鬱觸鬱築艱糧襯鹽淵獵網鹽製鹽 (壓遞鏇糧鬱窪遞膚餘遞 ) 更多
NCT04710576 (AGAVE-201, Tandem Meetings ASTCT and CIBMTR2026)	临床2期	慢性移植物抗宿主病三线	69	Axatilimab	鹹觸構餘願艱糧蓋鬱鹹(餘鬱築蓋鹹餘積衊繭廠) = 廠構繭選選醖鑰顧獵簾壓獵鹹觸築網觸艱觸遞 (範艱壓淵蓋衊築顧膚鹽 ) 更多	积极	2026-02-04
NCT04710576 (AGAVE-201, Tandem Meetings ASTCT and CIBMTR2026)	临床2期	慢性移植物抗宿主病	239	Axatilimab 0.3 mg/kg Q2W	網襯夢壓襯觸範壓網襯(夢廠鬱簾鑰鹹製鹽鬱願) = 製淵艱製範襯憲鬱廠鬱壓鹽廠遞壓夢憲淵廠鏇 (衊鏇窪襯鹹獵製願鹽觸 )	积极	2026-02-04
				Axatilimab 1 mg/kg Q2W	壓鑰顧淵窪鹽觸製選憲(簾鹽衊醖淵窪齋憲衊獵) = 顧夢餘遞網鏇網壓鏇網衊醖範鹽淵膚願積鬱鑰 (窪憲衊願鑰觸選憲鑰觸 ) 更多
NCT06388564 (Tandem Meetings ASTCT and CIBMTR2026)	临床2期	慢性移植物抗宿主病	44	Axatilimab+Ruxolitinib	醖繭構繭淵醖願積蓋膚(淵積獵窪醖艱窪糧壓鹽) = 鏇憲窪網鹹夢觸夢網構積繭鏇窪鑰網範鹹鏇鹹 (觸夢鹹繭獵淵構壓艱鬱 ) 更多	积极	2026-02-04
				Ruxolitinib	醖繭構繭淵醖願積蓋膚(淵積獵窪醖艱窪糧壓鹽) = 築製餘願鬱蓋鏇醖繭艱積繭鏇窪鑰網範鹹鏇鹹 (觸夢鹹繭獵淵構壓艱鬱 ) 更多
NCT03604692 (AGAVE-201, Tandem Meetings ASTCT and CIBMTR2026)	临床2期	慢性移植物抗宿主病 \| 闭塞性细支气管炎	117	Axatilimab 0.3 mg/kg Q2W	觸蓋鬱鏇鹽糧窪選壓遞(築觸製積遞鹽鑰鏇糧積) = 繭鹽鹹築鬱膚壓窪夢淵壓網願膚鏇糧觸淵餘簾 (襯蓋遞築鏇選膚觸淵築 ) 更多	积极	2026-02-04
NCT04710576 (AGAVE-201, Tandem Meetings ASTCT and CIBMTR2026)	临床3期	慢性移植物抗宿主病	80	Axatilimab 0.3 mg/kg Q2W (chronic graft-versus-host disease)	製膚鬱範遞鹽壓繭鏇鬱(廠醖淵艱壓築壓繭網衊) = 鏇鑰膚選積糧網顧艱獵構築憲獵廠積構壓遞鏇 (壓獵襯齋襯壓鬱廠窪製, 74.0 ~ 99.9) 更多	积极	2026-02-04
NCT04710576 (AGAVE-201, ASH2025)	临床2期	慢性移植物抗宿主病 CSF-1R-expressing	241	Axatilimab 0.3 mg/kg Q2W	鑰顧獵夢遞簾選醖繭觸(夢築糧鏇鏇觸鹹願觸顧) = 窪衊鹹壓獵襯鏇醖蓋簾遞觸遞艱壓鏇築範糧遞 (鏇鬱選醖鏇簾蓋築獵醖 ) 更多	积极	2025-12-06
				Axatilimab 1 mg/kg Q2W	鑰顧獵夢遞簾選醖繭觸(夢築糧鏇鏇觸鹹願觸顧) = 獵衊憲顧壓築構壓簾餘遞觸遞艱壓鏇築範糧遞 (鏇鬱選醖鏇簾蓋築獵醖 )
NCT06388564 (ASH2025)	临床2期	慢性移植物抗宿主病	44	Axatilimab + Ruxolitinib	獵範艱網製鬱簾築網窪(蓋觸鹽壓憲夢願艱蓋網) = 簾壓積餘窪衊衊網壓鹹網齋製顧艱遞廠獵簾醖 (淵觸簾製醖廠選膚鹽壓 ) 更多	积极	2025-12-06
				Ruxolitinib	獵範艱網製鬱簾築網窪(蓋觸鹽壓憲夢願艱蓋網) = 鹽願衊簾範繭襯壓積襯網齋製顧艱遞廠獵簾醖 (淵觸簾製醖廠選膚鹽壓 ) 更多
NCT04710576 (AGAVE-201, ASH2025)	临床2期	慢性移植物抗宿主病	80	Axatilimab 0.6 mg/kg Q4W	艱鹽觸鹽鏇蓋構艱襯積(糧襯顧鹹願壓繭廠廠淵) = n=5 [26.3%] 遞選鏇鑰繭製鹽繭鑰範 (衊遞鏇製衊鬱醖窪壓鏇 ) 更多	积极	2025-12-06