脐带血移植作为造血干细胞移植的重要方式，近年来面临单倍体相合移植等疗法的竞争，进入发展挑战期。但该疗法在特定人群治疗中仍具有不可替代的价值，同时其在细胞基因治疗、再生医学领域的应用潜力不断凸显。欧洲血液与骨髓移植学会（EBMT）作为该领域的权威机构，通过构建标准化教育体系、开展专业培训与考核，推动全球移植领域诊疗水平同质化发展。意大利Annalisa Ruggeri教授结合相关临床实践与EBMT工作，探讨了脐带血移植的临床价值、发展前景及行业规范化教育的重要意义，为该领域的持续进步提供参考。

Annalisa Ruggeri教授：脐带血移植当前正面临由竞争环境带来的诸多挑战，这在相当程度上源于人类白细胞抗原（HLA）不相合亲缘供者移植可行性的提升——尤其是采用单倍体相合供体联合移植后环磷酰胺的治疗模式。尽管如此，脐带血移植在特定临床场景中仍是一种不可或缺的重要手段，尤其适用于儿科患者及遗传性疾病患者。对于此类人群，需尽可能降低移植物抗宿主病（GVHD）对生活质量的影响，而脐带血移植在该方面具有其独特优势。

本次我的演讲聚焦于“焕发脐带血移植的新活力”，其核心在于，随着扩增技术（例如UM171）的应用，脐带血移植的临床疗效已得到显著提升：中性粒细胞植入时间与血小板恢复时间均大幅缩短，从而降低了非复发死亡率，为该技术的持续发展注入了新动力。

脐带血移植也是特定高危患者群体的重要治疗选择，例如具有极高复发风险的微小残留病（MRD）阳性急性白血病患者。研究已明确表明，脐带血移植中因HLA丢失所驱动的复发率极低，这为该类患者实现长期疾病控制提供了重要机遇。此外，脐带血移植也可作为细胞与基因治疗的独特平台。目前已有数项重要临床试验取得进展，例如由MD安德森癌症中心研究人员主导的、采用脐带血来源CAR-NK细胞治疗患者的研究；在再生医学领域，基于脐带血来源平台的移植也正应用于组织修复治疗中。有理由相信，脐带血移植在细胞与基因治疗中的应用仍存在广阔的探索空间。

伴随着脐带血移植适应证的不断拓展与治疗策略的持续演进，对临床医生的专业知识、技术规范及跨领域协作能力也提出了更高要求。在此背景下，持续的专业教育与全球实践标准的协同显得尤为重要。EBMT为此构建了系统的教育支持体系，致力于推进全球移植领域实践的规范化与标准化。其中，即将举办的第二届EBMT资格认证考试便是该体系的关键环节之一，旨在为移植专科医生及护理人员提供权威的能力评估与资质认证途径。鼓励处于职业生涯早期的同仁，通过学会提供的在线课程模块进行系统学习，并在此基础上参加考试，从而在验证个人专业能力的同时，促进不同国家、不同中心之间实践经验的交流与分享。教育始终是EBMT的核心使命。除了认证考试与在线课程，学会还通过组织系列线上研讨会、开展临床病例多学科讨论、设立月度文献评析专栏等多种形式，为全球移植界同仁搭建了多层次、立体化的学习与交流平台。积极参与这些教育活动，不仅有助于临床医生个人专业素养的持续提升，更是汇聚全球智慧、推动移植学科整体进步的重要方式。

Cord blood transplantation is currently navigating a challenging period, largely due to competition from alternative donor sources. In particular, the feasibility of transplantation using HLA-mismatched related donors—specifically haploidentical donors with post-transplant cyclophosphamide—has provided a viable and increasingly preferred option. Nevertheless, cord blood transplantation remains an important tool in certain specific clinical scenarios, such as pediatric patients or individuals with inherited disorders, where the impact of graft-versus-host disease (GVHD) on quality of life necessitates its avoidance.

The title of my presentation focused on rejuvenating cord blood transplantation. This perspective is driven by recent advances in ex vivo expansion technologies. For instance, the use of UM171 has been shown to markedly reduce the time to neutrophil engraftment and platelet recovery, thereby decreasing non-relapse mortality. These developments have opened new possibilities for the broader application of cord blood transplantation.

Furthermore, cord blood transplantation serves as a valuable option for patients with minimal residual disease (MRD)-positive acute leukemia, who face a very high risk of disease relapse. In this context, we have clearly demonstrated that the incidence of relapse driven by HLA loss is remarkably low following cord blood transplantation, offering these patients a significant opportunity for long-term disease control. In addition, cord blood transplantation provides a unique platform for cell and gene therapy. Several important clinical trials are currently underway, demonstrating the safe administration of cord blood-derived natural killer (NK) CAR cells—pioneered by investigators at MD Anderson Cancer Center in Houston—as well as applications in regenerative medicine, for example, in patients requiring tissue repair using cord-derived platforms. I strongly believe that we still have much to learn regarding the application of cord blood transplantation in the fields of cell and gene therapy.

The European Society for Blood and Marrow Transplantation (EBMT) offers several important tools to validate and standardize education across the board. This year marks the second edition of the EBMT examination, which I strongly recommend, in my role as education representative of the EBMT society. I encourage all early-career transplant physicians and nurses to engage with the online preparatory module, with the subsequent opportunity to take the EBMT examination. This process enables both validation of knowledge and sharing of experiences across different countries within the EBMT network. Education remains one of our primary goals and objectives. The EBMT provides numerous webinars, as well as ample opportunities for discussion through clinical case presentations and the "paper of the month" series. I truly believe that all transplant physicians can approach the EBMT to share and participate in these educational tools, which represent a collective learning pathway for all of us.

总结

尽管面临行业竞争的挑战，但脐带血移植凭借在特定人群治疗中的独特优势、扩增技术带来的疗效提升，以及在细胞基因治疗、再生医学领域的广阔潜力，依然具有不可替代的临床价值，其发展前景值得期待。EBMT通过构建完善的教育体系、开展标准化培训与考核，为全球移植领域从业者提供了学习交流的平台，有效推动了诊疗水平的同质化发展，保障了患者治疗安全。这项工作不仅夯实了脐带血移植及相关领域的发展基础，更彰显了全球医学协作、追求专业精进、以患者为中心的医学理念，为血液移植领域的持续创新与进步注入持久动力，也为其他医学领域的规范化发展提供了有益借鉴。