急性髓系白血病（AML）作为一类异质性的血液系统恶性肿瘤，其治疗一直是血液学领域的重大挑战与焦点。尽管化疗方案不断优化，但对于多数患者而言，异基因造血干细胞移植（HSCT）仍是当前唯一有望实现长期生存乃至治愈的基石性手段。然而，移植技术本身并非停滞不前，其适应症、技术流程及支持疗法在过去数十年间经历了革命性的演变。与此同时，靶向治疗与免疫治疗的崛起，正与移植技术形成深刻的协同与互补，共同重塑着AML的治疗范式。2025年11月13-16日，2025国际细胞与免疫治疗大会（CTI 2025）在浙江杭州召开。会议期间，《肿瘤瞭望-血液时讯》特邀法国医学科学院院士Norbert Claude Gorin教授基于国际视野，特别是欧洲血液和骨髓移植学会（EBMT）的丰富经验，系统回顾AML领域在移植技术、新型疗法方面取得的关键进展，并展望通过全球合作，特别是中欧间日益紧密的协作，以期为提升AML患者预后带来广阔前景。
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造血干细胞移植技术的三大演进：拓展治愈边界
目前，对于大多数急性髓系白血病（AML）患者而言，异基因造血干细胞移植（HSCT）仍是唯一可能实现治愈的关键治疗手段。回顾过去三十年，HSCT领域取得了跨越式发展。首要的进展体现在患者年龄限制的显著放宽，从最初仅适用于55岁以下患者，扩展至如今75岁以下的患者也能受益，这主要归功于毒性更低的降低强度预处理（RIC）方案的广泛应用。
其次，干细胞来源发生了根本性变革，从需在全身麻醉下采集的骨髓，转变为通过白细胞分离术即可相对便捷获取的外周血干细胞，极大提升了供受双方的可操作性及安全性。
第三，供者选择范围得到了前所未有的拓展，突破了以往必须依赖人类白细胞抗原（HLA）全相合同胞供者的局限，单倍型相合移植技术的成熟使得几乎所有直系亲属成员都可能成为潜在供者，这一技术自2005-2006年起在中国与美国同步发展并迅速推广，是足以覆盖几乎所有需求患者的巨大进步。值得注意的是，约半数AML患者确诊时年龄已超过70岁，上述技术进步无疑具有重大的临床意义。
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靶向与免疫治疗崛起：革新AML治疗格局
在移植技术不断优化的同时，AML的治疗武器库也迎来了新的成员。靶向治疗能够特异性作用于仅或主要表达于AML细胞表面的特定抗原或分子（如FLT3、IDH等），与化疗等手段联合应用，可显著提升诱导缓解的深度与效率。此外，免疫治疗虽在髓系恶性肿瘤中的应用挑战更大，但目前已涌现出多种颇具前景的策略，包括T细胞衔接器、靶向CD33及CD123等的单克隆抗体及其与化疗药物偶联的抗体药物偶联物（ADC），以及已进入临床探索阶段的嵌合抗原受体T细胞（CAR-T）疗法。
全球研发格局亦值得关注，约48%的CAR-T细胞临床试验在中国开展，另有48%集中于美国，欧洲则占4%，这预示着未来治疗创新将更具全球性与多样性。展望未来，AML的治疗格局预计将在未来五年内持续革新，当前患者的总体治愈率约为50%，未来十年内有望提升至65%左右。
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中欧协作与全球数据共享：构建共赢的未来
从国际合作视角看，作为欧洲血液和骨髓移植学会（EBMT）全球委员会的首任主席（该委员会成立于2017年），我与中国血液学界有着深厚渊源。早自1977年完成首例自体移植起，便有多位中国青年学者赴我所在的机构接受系统培训，从自体移植逐步延伸至异基因移植领域，特别是后者的开展尤为不易。
出任现职后，我已近二十次访华，并将中国确立为全球委员会的首要合作方向，这不仅是基于中国十四亿人口的巨大健康需求，更因其已涌现出一批技术精湛、达到国际先进水平的移植中心。推动中国团队融入EBMT体系是核心任务之一，迄今已有39个中国中心成功加入，其临床数据业已汇入EBMT这个涵盖超过80万例移植病例的全球最大注册研究数据库，极大地丰富了全球研究的多样性与代表性。
尤为可喜的是，目前有六至七位优秀的中国青年研究者正与我们基于该数据库开展深度科研合作，已共同在干细胞移植领域发表了约十篇高质量学术论文，真正实现了知识共享、互利共赢的合作初衷。
For most patients with acute myeloid leukemia (AML), allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potentially curative key treatment. Over the past three decades, the field of HSCT has seen leaps in development. The primary progress is reflected in the significant relaxation of patient age limits. Initially applicable only to patients under 55, it now benefits patients up to 75 years old, largely thanks to the widespread use of less toxic reduced-intensity conditioning (RIC) regimens.
Secondly, the source of stem cells has undergone a fundamental shift. It has moved from bone marrow, which requires collection under general anesthesia, to peripheral blood stem cells, which can be relatively easily obtained via leukapheresis. This has greatly enhanced the operability and safety for both donors and recipients.
Thirdly, the range of donor options has expanded unprecedentedly, breaking through the previous limitation of relying solely on human leukocyte antigen (HLA)-matched sibling donors. The maturation of haploidentical transplantation technology means that almost any immediate family member can potentially be a donor. This technology, developed and rapidly promoted concurrently in China and the United States since around 2005-2006, represents a monumental advancement sufficient to cover almost all patients in need. It is noteworthy that approximately half of AML patients are diagnosed over the age of 70, making these technological progressions undoubtedly of great clinical significance.
While transplantation technology continues to optimize, the AML treatment arsenal has also welcomed new additions. Targeted therapies can specifically act on particular antigens or molecules (such as FLT3, IDH) expressed solely or predominantly on AML cell surfaces. Used in combination with chemotherapy, they can significantly improve the depth and efficiency of induction remission. Furthermore, although applying immunotherapy to myeloid malignancies presents greater challenges, several promising strategies have already emerged. These include T-cell engagers, monoclonal antibodies targeting CD33 and CD123, along with their antibody-drug conjugates (ADCs), and chimeric antigen receptor T-cell (CAR-T) therapies, which have entered clinical exploration.
The global R&D landscape is also noteworthy. Approximately 48 percent of CAR-T cell clinical trials are conducted in China, with another 48 percent concentrated in the United States and 4 percent in Europe. This indicates that future treatment innovations will be more global and diverse. Looking ahead, the AML treatment landscape is expected to continue evolving over the next five years. While the current overall cure rate for patients is about 50 percent, it is projected to increase to around 65 percent within the next decade.
From an international cooperation perspective, as the inaugural President of the Global Committee of the European Society for Blood and Marrow Transplantation (EBMT) – established in 2017 – I have deep-rooted connections with the Chinese hematology community. Dating back to 1977 when I performed my first autologous transplant, several young Chinese scholars have received systematic training at my institution, gradually extending from autologous to allogeneic transplantation, with the latter being particularly challenging to establish.
Since assuming my role, I have visited China nearly twenty times and have identified China as the primary collaboration direction for the Global Committee. This decision is based not only on the vast health needs of China's 1.4 billion population but also on the emergence of a group of technically proficient transplantation centers in China that have reached international advanced standards. One core mission has been facilitating the integration of Chinese teams into the EBMT system. To date, 39 Chinese centers have successfully joined, and their clinical data has been incorporated into the EBMT registry, the world's largest database with over 800,000 transplant cases. This极大地 (has greatly) enriched the diversity and representativeness of global research.
It is particularly gratifying that currently, six to seven outstanding young Chinese researchers are engaging in deep scientific collaboration with us using this database. We have already co-authored about ten high-quality academic papers in the field of stem cell transplantation, truly realizing the initial goal of knowledge sharing and mutual benefit.
总 结
AML的治疗征程，是一部从依赖单一强效手段到构建整合性治疗体系的演进史。从HSCT技术的三次重大飞跃——年龄限制突破、干细胞来源革新、供者选择拓宽，到靶向与免疫治疗为代表精准医疗时代的到来，我们正见证着一种治疗范式的根本性转变：从“最大耐受剂量”的毒性攻坚，迈向“最大精准程度”的个体化调控。
这一进程不仅体现在技术层面，更深刻地蕴含于全球医学合作模式的升级中。中欧在AML领域的合作，恰是这一趋势的生动注脚。它超越了单纯的技术引进，发展为基于平等、互信的数据共享与协同创新。中国庞大的临床资源与欧洲悠久的学术积淀相结合，共同贡献于全球血液学知识体系，最终使各国患者受益。
展望未来，随着更多创新疗法的涌现与全球合作网络的深化，我们坚信，攻克AML这一目标的实现将不仅依赖于实验室里的科学突破，更有赖于跨越国界的智慧汇聚与共同努力。这场战役的终极胜利，必将属于开放、协作的整个人类科学共同体。
会场花絮
专家简介
前插行后插行
Norbert Claude Gorin 教授
法国医学科学院院士
巴黎索邦大学血液学和细胞治疗教授
欧洲血液和骨髓移植学会（EBMT）全球委员会主席
法国国家医学科学院院士
中国工程院2023年外籍院士