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造血细胞移植和嵌合抗原受体T细胞疗法治疗血液系统疾病、实体瘤及免疫紊乱的适应证:2025年欧洲血液与骨髓移植学会实践建议

Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations.

作者信息

Greco R, Ruggeri A, McLornan D P, Snowden J A, Alexander T, Angelucci E, Averbuch D, Bazarbachi A, Hazenberg M D, Kalwak K, Kenyon M, Mekelenkamp H, Neven B, Pedrazzoli P, Peric Z, Risitano A M, Sánchez-Ortega I, Ciceri F, Sureda A

机构信息

Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Hospital, Vita-Salute San Raffaele University, Milan, Italy.

Department of Haematology and Stem Cell Transplantation, University College London Hospitals NHS Trust, London, UK.

出版信息

Bone Marrow Transplant. 2025 Sep 9. doi: 10.1038/s41409-025-02701-3.

DOI:10.1038/s41409-025-02701-3
PMID:40926035
Abstract

For over two decades, the EBMT has updated recommendations on indications for haematopoietic cell transplantation (HCT) practice based on clinical and scientific developments in the field. This is the ninth special EBMT report on indications for HCT for haematological diseases, solid tumours and immune disorders. Our aim is to provide guidance on HCT indications according to prevailing clinical practice in EBMT countries and centres. In order to inform patient decisions, these recommendations must be considered with the risk of the disease, risk of HCT procedure and non-HCT strategies, including evolving cellular therapies, and their availability on site. HCT techniques are constantly evolving and we make no specific recommendations, but encourage harmonisation of practice, where possible, to ensure experience across indications can be meaningfully aggregated via registry outputs. We also recommend working according to JACIE certification standards to maintain quality in clinical and laboratory practice, including benchmarking of survival outcomes [1-3]. Since the last edition, innovative cellular and gene therapies have entered in activity across indications affecting clinical decision making. As the number and type of regulatory authority-approved cellular therapies grow, recommendations for best practice and quality of patient care were developed to support clinicians and will be regularly updated.

摘要

二十多年来,欧洲血液与骨髓移植协会(EBMT)一直根据该领域的临床和科学进展,更新造血细胞移植(HCT)实践适应症的建议。这是关于血液系统疾病、实体瘤和免疫疾病HCT适应症的第九份EBMT特别报告。我们的目标是根据EBMT国家和中心的现行临床实践,为HCT适应症提供指导。为了让患者做出决策,这些建议必须结合疾病风险、HCT程序风险和非HCT策略(包括不断发展的细胞疗法)及其在当地的可及性来考虑。HCT技术在不断发展,我们不给出具体建议,但鼓励尽可能统一实践,以确保通过登记结果能够有意义地汇总各适应症的经验。我们还建议按照欧洲细胞治疗认证基金会(JACIE)认证标准开展工作,以维持临床和实验室实践的质量,包括生存结果的基准比较[1-3]。自上一版以来,创新的细胞和基因疗法已在影响临床决策的各个适应症中开展应用。随着监管机构批准的细胞疗法数量和类型的增加,已制定了最佳实践和患者护理质量的建议,以支持临床医生,并将定期更新。

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Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM.基于白消安或苏消安预处理方案的儿童急性淋巴细胞白血病异基因造血干细胞移植的可比结局:FORUM研究结果
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Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.
贝替贝洛ogene 自体造血干细胞基因治疗依赖输血的严重基因型β-地中海贫血患者(HGB-212):一项非随机、多中心、单臂、开放标签、单次剂量、3 期临床试验。
Lancet. 2024 Nov 30;404(10468):2175-2186. doi: 10.1016/S0140-6736(24)01884-1. Epub 2024 Nov 8.
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