Medical Clinic, Department for Haematology, Oncology and Tumorimmunology, Charité Universitätsmedizin Berlin, Berlin, Germany; EBMT Transplant Complications Working Party, Department of Haematology, Saint Antoine Hospital; INSERM UMR-S 938, Sorbonne University, Paris.
EBMT Transplant Complications Working Party, Department of Haematology, Saint Antoine Hospital; INSERM UMR-S 938, Sorbonne University, Paris, France; EBMT Paris study office, Department of Haematology, Saint Antoine Hospital; INSERM UMR-S 938, Sorbonne University, Paris.
Haematologica. 2024 Nov 1;109(11):3557-3565. doi: 10.3324/haematol.2023.284810.
Chimeric antigen receptor (CAR) T cells are in standard clinical use to treat relapsed or refractory hematologic malignancies, such as non-Hodgkin lymphoma, multiple myeloma and acute lymphoblastic leukemia. Owing to the rapidly progressing field of CAR T-cell therapy and the lack of generally accepted treatment guidelines, we hypothesized significant differences between European centers in prevention, diagnosis and management of short- and long-term complications. To capture the current CAR T-cell management among European Society for Blood and Marrow Transplantation (EBMT) centers and to determine the medical need and specific areas for future clinical research the EBMT Transplant Complications Working Party performed a survey among 227 EBMT CAR T-cell centers. We received complete servey answers from 106 centers (47%) addressing questions in the areas of product selection, CAR T-cell logistics, management of cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome as well as management in later phases including prolonged cytopenias. We identified common patterns in complication management, but also significant variety in clinical management of the centers in important aspects. Our results demonstrate a high medical need for treatment harmonization and future clinical research in the following areas: treatment of steroid-refractory and very severe cytokine release syndrome/neurotoxicity, treatment of cytopenia, early discharge and outpatient management, as well as immunoglobulin substitution.
嵌合抗原受体 (CAR) T 细胞已被广泛应用于治疗复发或难治性血液系统恶性肿瘤，如非霍奇金淋巴瘤、多发性骨髓瘤和急性淋巴细胞白血病。由于 CAR T 细胞治疗领域的快速发展，以及缺乏普遍接受的治疗指南，我们假设欧洲各中心在预防、诊断和管理短期和长期并发症方面存在显著差异。为了了解欧洲血液和骨髓移植学会 (EBMT) 中心目前的 CAR T 细胞管理情况，并确定医学需求和未来临床研究的具体领域，EBMT 移植并发症工作组对 227 个 EBMT CAR T 细胞中心进行了调查。我们收到了 106 个中心（47%）的完整调查答复，这些中心回答了产品选择、CAR T 细胞物流、细胞因子释放综合征和免疫效应细胞相关神经毒性综合征管理以及包括延长细胞减少期在内的后期管理等领域的问题。我们在并发症管理方面发现了共同的模式，但在重要方面，各中心的临床管理也存在显著差异。我们的研究结果表明，在以下领域需要进行治疗协调和未来的临床研究：治疗类固醇难治性和非常严重的细胞因子释放综合征/神经毒性、治疗细胞减少症、早期出院和门诊管理以及免疫球蛋白替代治疗。
