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确定造血移植与细胞治疗的全球优先事项:EBMT 全球委员会的声明文件

英文原题:Defining global priorities in hematopoietic transplantation and cellular therapy: a statement paper from EBMT Global Committee.

PubMed 2026/08/11(内容时间) Bone Marrow Transplant Q1 · IF 5.1(JCR 2025)

研究概要

大多数受访者(67.1%)报告在JACIE或FACT认证的中心工作。

中文摘要

造血细胞移植、细胞与基因治疗以及 CAR-T 细胞已彻底改变了血液系统疾病的结局。然而,在其可获得性、可及性和可负担性方面仍存在重大差异,尤其是在低中等收入国家(LMICs)。EBMT 全球委员会旨在解决这些不平等问题。向 EBMT 成员分发了一份结构化调查,以确定区域和主题优先事项、评估当前基础设施,并评估参与全球倡议的意愿。对回复进行了描述性分析,并作为本声明文件的基础。共收集到来自 43 个国家的 145 份回复。大多数受访者(67.1%)报告在 JACIE 或 FACT 认证中心工作。EBMT 外展的最高优先区域是东欧(非欧盟)(43.9%)、拉丁美洲(38.2%)和非洲(33.5%),其次是中东(29.5%)。主要主题优先事项是教育和培训(74.3%)、科学合作(56.0%)和登记系统发展(43.4%)。大多数受访者表示愿意积极贡献,尤其是在教育活动方面(59.7%)。定性回复强调需要实用指南、导师计划、远程医疗、疑难病例的虚拟讨论/临床查房,以及对 LMIC 能力建设的支持。EBMT 社区强烈支持扩大全球角色,重点是在服务不足地区开展教育、能力建设和合作。

展开英文摘要原文

Hematopoietic cell transplantation, cellular and gene therapies, and (CAR)-T cells have revolutionized outcomes in hematologic disorders. However, major disparities persist in their availability, accessibility, and affordability, particularly in low- and middle-income countries (LMICs). The EBMT Global Committee aims to address these inequalities. A structured survey was distributed to EBMT members to identify regional and thematic priorities, assess current infrastructure, and assess willingness to contribute to global initiatives. Responses were analyzed descriptively and served as the basis for the current statement paper. A total of 145 responses from 43 countries were collected. Most respondents (67.1%) reported working in JACIE- or FACT-accredited centers. The highest priority regions for EBMT outreach were Eastern Europe (non-EU) (43.9%), Latin America (38.2%), and Africa (33.5%), followed by the Middle East (29.5%). The leading thematic priorities were education and training (74.3%), scientific collaboration (56.0%), and registry development (43.4%). Most respondents expressed willingness to actively contribute, particularly in educational activities (59.7%). Qualitative responses emphasized the need for practical guidelines, mentorship programs, telemedicine, virtual discussion/clinical rounds on difficult cases, and support for LMIC capacity building. The EBMT community strongly supports an expanded global role focused on education, capacity building, and collaboration in underserved regions.

论文信息

作者
Nagler A、Gorin NC、Chabannon C、Puigventós G、Heemskerk R、Sánchez-Ortega I、Rintala T、Mahé Y
第一作者单位
Division of Hematology, Sheba Medical Center, Tel Hashomer, Israel. arnon.nagler@sheba.health.gov.il.Israel
通讯作者单位
IRCCS Ospedale San Raffaele, Vita-Salute San Raffaele University, Haematology and Bone Marrow Transplantation, Milano, Italy. greco.raffaella@hsr.it.Italy
期刊
Bone marrow transplantation2026 Aug 11
原文标识
PubMed 42581093 · DOI 10.1038/s41409-026-03008-7