CAR-T（CAR-T）细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T（CAR-T）细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
代表美国移植与细胞治疗学会：异基因造血细胞移植与 CAR-T 细胞治疗在慢性淋巴细胞白血病患者中作用的临床实践建议
英文原题：Clinical Practice Recommendations on the Role Of Allogeneic Hematopoietic Cell Transplantation and Chimeric Antigen Receptor T-Cell Therapy in Patients With Chronic Lymphocytic Leukemia on Behalf of the American Society for Transplantation and Cellular Therapy.
查看英文原题
Clinical Practice Recommendations on the Role Of Allogeneic Hematopoietic Cell Transplantation and Chimeric Antigen Receptor T-Cell Therapy in Patients With Chronic Lymphocytic Leukemia on Behalf of the American Society for Transplantation and Cellular Therapy.
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中文摘要
CAR-T 细胞疗法（CAR-T 细胞）是复发和/或难治性（R/R）慢性淋巴细胞白血病（CLL）的一种新治疗选择。包括共价或非共价Bruton酪氨酸激酶抑制剂（BTK）以及B细胞白血病/淋巴瘤2蛋白（BCL-2）抑制剂venetoclax在内的新型疗法已在一线和R/R治疗中取代了化学免疫治疗（CIT）方案，并将异基因造血细胞移植（allo-HCT）推至更晚的治疗阶段。更新2016年关于allo-HCT治疗CLL的临床实践建议对于指导当代临床实践是必要的。由18位在不同CLL治疗模式方面具有多元化专业知识的医师和1位方法学家组成的小组参与了这项工作。任何获得70%投票的建议即被视为共识。对于至少接受过2线治疗（包括共价BTK抑制剂和BCL-2抑制剂）后无应答或复发的患者，推荐CAR-T 细胞疗法。
此外，对于随后在三线或更晚阶段接受非共价BTK抑制剂的患者，无论应答如何，均推荐CAR-T 细胞疗法。对于allo-HCT后复发的CLL，假设患者适合该操作，也推荐CAR-T 细胞疗法。在适合作为候选者的CLL患者中，如果疾病对CAR-T 细胞疗法为R/R，且在异基因移植前证实有客观缓解，则推荐allo-HCT。对于克隆相关Richter转化（RT）患者，在证实对一线CIT或其他治疗有客观缓解后，也推荐allo-HCT。对于R/R RT，推荐CAR-T 细胞疗法。
我们强调，只要有可用临床试验，就应让患者入组，以继续推动该领域进展并改善 R/R CLL 的预后。我们承认，本文未涵盖一些独特的临床情形，这些情形可能需要采取个案化处理。
展开英文摘要原文
Chimeric antigen receptor T-cell therapy (CAR T-cell) is a new treatment option for relapsed and/or refractory (R/R) chronic lymphocytic leukemia (CLL). Novel therapies including Bruton's tyrosine kinase inhibitors (BTK), covalent or noncovalent, and an inhibitor of the B-cell leukemia/lymphoma 2 protein (BCL-2), venetoclax, have replaced chemoimmunotherapy (CIT) regimens in the front-line and the R/R setting, and have relegated allogeneic hematopoietic cell transplantation (allo-HCT) to later treatment stages.
Updating the 2016 clinical practice recommendations on allo-HCT in CLL is necessary to help guide contemporary clinical practice. A panel of 18 physicians with diverse expertise across different CLL treatment modalities and one methodologist participated in this effort. Any recommendation receiving 70% votes was considered a consensus. CAR T-cell therapy is recommended for patients not responding or relapsing after at least 2 lines of therapy consisting of a covalent BTK inhibitor and a BCL-2 inhibitor.
In addition, CAR T-cell therapy is recommended for patients who subsequently received a noncovalent BTK inhibitor in the third-line or later setting, regardless of response. CAR T-cell therapy is also recommended in CLL relapsing after an allo-HCT, assuming that patients are fit for the procedure.
In those CLL patients who are candidates, allo-HCT is recommended if disease is R/R to CAR T-cell therapy provided that an objective response is demonstrated prior to the allograft. Allo-HCT is also recommended in patients with clonally-related Richter transformation (RT) after demonstrating an objective response to front-line CIT or other treatments. CAR T-cell therapy is recommended in R/R RT.
We emphasize the importance of enrolling patients in clinical trials whenever available to continue to advance the field and improve prognosis of R/R CLL.
We acknowledge that there are unique clinical scenarios not covered herein which may require a case-by-case approach.
论文信息
- 作者
- Kharfan-Dabaja MA、Kumar A、Pinilla-Ibarz J、Brown JR、Shadman M、Awan FT、Kenderian SS、Siddiqi T
- 单位
- Division of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapy Program, Mayo Clinic Comprehensive Cancer Center, Mayo Clinic, Jacksonville, Florida. Electronic address: KharfanDabaja.Mohamed@mayo.edu.United States
- 文献类型
- 临床实践指南
- 期刊
- Transplantation and cellular therapy2025 Aug
- 原文标识
- PubMed 40514010 · DOI 10.1016/j.jtct.2025.06.002