CAR-T疗法治疗B细胞血液恶性肿瘤的快速卫生技术评估

胡紫馨 谢金平 王苑如 邵蓉

中图分类号 R733.7;R956;R979.1 文献标志码 A 文章编号 1001-0408（2021）23-2895-06

DOI 10.6039/j.issn.1001-0408.2021.23.14

摘 要 目的：评价嵌合抗原受体T细胞（CAR-T）疗法治疗B细胞血液恶性肿瘤的有效性、安全性和经济性，为临床决策提供循证依据。方法：利用快速卫生技术评估（HTA）方法进行评价，系统检索PubMed、Embase、Cochrane Library、中国知网、万方数据库和国外HTA官方网站，数据库的检索时限均为建库起至2021年3月20日。按照纳排标准对文献进行纳入和资料提取、质量评价后，对CAR-T疗法治疗B细胞血液恶性肿瘤的有效性、安全性和经济性进行描述性分析。结果：共纳入2篇HTA报告、5篇系统评价/Meta分析和5篇经济学研究。在有效性方面，CAR-T疗法治疗B细胞血液恶性肿瘤具有良好的有效性，治疗急性淋巴细胞白血病的总缓解率（ORR）在63.5%以上、完全缓解率（CR）为77.1%（95%CI：62.8%～87.1%），治疗慢性淋巴细胞白血病的ORR为70.0%（95%CI：53.0%～80.0%）、CR为25.5%（95%CI：13.9%～42.1%），治疗B细胞淋巴瘤的ORR在44.4%以上。在安全性方面，CAR-T疗法治疗过程中细胞因子释放综合征发生率在20%以上，1/3或以上（个别研究认为发生率为9%）的患者发生了神经毒性，感染发生率为12.2%～33.3%，移植物抗宿主病的发生率为23.4%（95%CI：8.6%～49.8%）。在经济性方面，纳入研究多认为CAR-T疗法具有经济学优势，但均是在美国、日本等发达国家评價的结果。结论：CAR-T作为新兴的血液恶性肿瘤治疗产品，具有良好的有效性，发生的不良反应等级较低、基本可控;而经济性尚需结合在国内实际开展的相关研究进一步评价。

关键词 嵌合抗原受体T细胞免疫疗法;B细胞血液恶性肿瘤;快速卫生技术评估

Chimeric Antigen Receptor T Cells Therapy for the Treatment of B-lymphoblastic Hematologic Malignancy： A Rapid Health Technology Assessment

HU Zixin，XIE Jinping，WANG Wanru，SHAO Rong（Research Center of National Drug Policy & Ecosystem， China Pharmaceutical University， Nanjing 211198， China）

ABSTRACT   OBJECTIVE： To evaluate the effectiveness， safety and economy of chimeric antigen receptor T cells （CAR-T） therapy for the treatment of B-lymphoblastic hematologic malignancy， and to provide evidence-based reference for clinical decision. METHODS： Rapid health technology assessment （HTA） was adopted. PubMed， Embase， Cochrane Library， CNKI， Wanfang databases and foreign HTA official websites were systematically searched during the inception-Mar. 20th， 2021. After inclusion， data extraction and quality evaluation of literatures according to the inclusion and exclusion criteria， descriptive analysis was performed for the effectiveness， safety and economy of CAR-T therapy for the treatment of B-lymphoblastic hematologic malignancy. RESULTS： A total of 2 HTA reports， 5 systematic reviews/Meta-analysis， and 5 economics studies were included. In terms of effectiveness， CAR-T therapy showed good efficacy in the treatment of B-lymphoblastic hematologic malignancy; overall remission rate （ORR） of CAR-T therapy in the treatment of acute lymphoblastic leukemia was more than 63.5%， and the complete remission rate （CR） was 77.1% （95%CI： 62.8%-87.1%）; ORR of CAR-T therapy in the treatment of chronic lymphoblastic leukemia was 70.0% （95%CI： 53.0%-80.0%）， and the CR was 25.5% （95%CI： 13.9%-42.1%）; ORR of CAR-T therapy in the treatment of B-cell lymphoma was more than 44.4%. In terms of safety， the incidence of cytokine release syndrome was more than 20% during the treatment of CAR-T therapy， and 1/3 or more （9% believed in some studies） patients suffered from neurotoxicity; the incidence of infection was 12.2%-33.3%， and the incidence of graft-versus-host disease was 23.4% （95%CI： 8.6%-49.8%）. In terms of economy， most of the included studies believed that CAR-T therapy possessed economic advantages， which were the results of evaluation in developed countries such as the United States and Japan. CONCLUSIONS： CAR-T， as a new product of treatment for hematological malignancy， shows good effectiveness and low level of ADR， which is basically controllable; its economy needs to be further evaluated by relevant researches combined with domestic reality.

KEYWORDS   Chimeric antigen receptor T cells therapy; B-lymphoblastic hematologic malignancy; Rapid health technology assessment

B细胞血液恶性肿瘤主要包括B系急性淋巴细胞白血病（acute lymphoblastic leukemia，ALL）、B系慢性淋巴细胞白血病（chronic lymphoblastic leukemia，CLL）和B细胞淋巴瘤。……