庆祝 9000 名接受 CAR T 细胞治疗的患者在 EBMT 登记处登记:收集造血细胞疗法方面的真实世界数据

IF 2.2 4区 医学 Q3 HEMATOLOGY Best Practice & Research Clinical Haematology Pub Date : 2024-06-01 DOI:10.1016/j.beha.2024.101557
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引用次数: 0

摘要

欧洲血液和骨髓移植学会(EBMT)长期以来一直关注造血细胞移植的评估工作。三十多年前,其成员建立了欧洲大陆登记处。如今,登记在册的患者已超过 70 万,收集到的移植信息超过 80 万条。这些海量信息有助于开展多项回顾性研究,评估不同时期和不同类别疾病的治疗方法的变化,为各 EBMT 附属中心的治疗效果设定基准,并越来越多地用于建立合成比较基准,以评估血液学领域的创新疗法。CAR-T 细胞疗法利用的人力和技术资源也用于提供造血干细胞移植;它们会产生副作用,需要实施风险缓解计划;它们是活体药物,会在受者体内持续存在,因此需要长期随访;在药典中引入 CAR-T 细胞可能会对 BMT 的实践产生重大影响;基于上述原因,甚至在 CAR-T 细胞首次在欧洲获批之前,EBMT 就参与了一个项目,旨在用细胞疗法表格对 EBMT 登记表进行补充,目的是登记接受 CAR-T 细胞治疗的患者,并收集其短期、中期和长期疗效的信息。其目的是为 EBMT 研究人员提供一种工具,用于对收集到的信息进行初步分析,并支持对转交给市场授权持有人和其他相关方的个人数据进行二次利用,以履行其对卫生当局的义务,并进一步评估 CAR-T 细胞在不同情况和适应症下的实际医疗价值。EBMT 注册表于 2019 年获得了欧洲药品管理局的肯定,五年后已包含 9000 多名接受治疗患者的信息。本文介绍了启动这项新活动的历程、在改进真实世界数据收集方面应吸取的经验教训,以及现有信息在患者就医方面给我们带来的启示。
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Celebrating the registration of 9.000 patients treated with CAR T cells in the EBMT registry: Collection of real-world data in the context of hematopoietic cellular therapies

The European society for Blood and Marrow Transplantation (EBMT) has a long-standing interest in the evaluation of hematopoietic cell transplantation. More than three decades ago, its members established a continental registry. Today, more than 700,000 patients have been registered, and information has been gathered on more than 800,000 transplants. This huge amount of information has allowed conducting multiple retrospective studies, evaluating changes in practices over time and for different categories of diseases, benchmarking outcome across EBMT affiliated centers, and increasingly serves to build synthetic comparators to evaluate the introduction of therapeutic innovations in the field of hematology. CAR-T cells therapies draw on human and technical resources that are also used to deliver HCT; they elicit side effects that require the implementation of risk mitigation plans; they are living drugs that persist in the body of the recipient and thus deserve prolonged follow-up; the introduction of CAR-T cells in the pharmacopeia is likely to significantly impact on the practice of BMT; for all these reasons and even before the first approvals of CAR-T Cells in Europe, EBMT engaged in a project aiming at complementing the EBMT Registry with a Cellular Therapy Form, with the objective to register CAR-T cells treated patients and collect information on their short-, middle- and long-term outcome. The goal is to provide EBMT investigators with a tool for primary analyses of the collected information and to support secondary use of data transferred at the individual level to Marketing Authorization Holders and other interested parties, to fulfill their obligations to health authorities and further evaluate the actual medical values of CAR-T Cells in different contexts and indications. The EBMT Registry received a positive opinion from the European Medicines agency in 2019, and five years later contains information on more than 9.000 treated patients. This article describes the journey to start this new activity, lessons to be drawn in view of improving the collection of real-world data, and what existing information tells us in terms of patient access.

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来源期刊
CiteScore
4.20
自引率
0.00%
发文量
42
审稿时长
35 days
期刊介绍: Best Practice & Research Clinical Haematology publishes review articles integrating the results from the latest original research articles into practical, evidence-based review articles. These articles seek to address the key clinical issues of diagnosis, treatment and patient management. Each issue follows a problem-orientated approach which focuses on the key questions to be addressed, clearly defining what is known and not known, covering the spectrum of clinical and laboratory haematological practice and research. Although most reviews are invited, the Editor welcomes suggestions from potential authors.
期刊最新文献
Editorial Board From clones to immunopeptidomes: New developments in the characterization of permissive HLA-DP mismatches in hematopoietic cell transplantation Relevance of donor-specific HLA antibodies in hematopoietic cell transplantation HLA structure and function in hematopoietic-cell transplantation Special issue 37.3: “Genetics and function of HLA and immune-related genes in hematopoietic cell transplantation and cellular immunotherapy”
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