State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Tianjin Institutes of Health Science, Tianjin, China.
J Immunother Cancer. 2024 Nov 27;12(11):e010080. doi: 10.1136/jitc-2024-010080.
B-cell maturation antigen(BCMA)-directed chimeric antigen receptor (CAR)-T-cell therapy has significantly improved the treatment of relapsed or refractory multiple myeloma (MM). Nevertheless, the uncommon phenomenon of biphasic CAR-T cell expansion in vivo and its related severe toxicities have not been methodically described and studied. Herein, we report a case of patients with MM who experienced two CAR-T cell expansion peaks and subsequently developed multiple severe toxicities following BCMA CAR-T cell infusion. The first expansion peak occurred on Day 7, accompanied by grade 3 cytokine release syndrome. The second peak occurred on Day 28, associated with severe immune effector cell-associated hematotoxicity (ICAHT), immune effector-cell associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS), and polymicrobial infections. Both ICAHT and IEC-HS were refractory to our standard treatments; however, human umbilical cord mesenchymal stem cell infusion exhibited some efficacy in improving cytopenia. Despite the administration of a broad-spectrum anti-infective regimen, cytomegalovirus viremia remained uncontrollable, resulting in the development of central nervous system infection, neurological symptoms, and ultimately death. Additionally, we also employed high-dimensional 33-color spectral flow cytometry to describe the dynamic changes in immune cell components and functions between the two expansion peaks. Collectively, this case provides novel insights into the biphasic CAR-T expansion and related immune effector cell-associated toxicities.
B 细胞成熟抗原 (BCMA)- 定向嵌合抗原受体 (CAR)-T 细胞疗法显著改善了复发或难治性多发性骨髓瘤 (MM) 的治疗效果。然而,体内 CAR-T 细胞的双相扩增这一罕见现象及其相关的严重毒性尚未得到系统描述和研究。在此,我们报告了一例 MM 患者在接受 BCMA CAR-T 细胞输注后经历了两次 CAR-T 细胞扩增峰,并随后出现多种严重毒性。第一次扩增峰发生在第 7 天,伴有 3 级细胞因子释放综合征。第二次扩增峰发生在第 28 天,与严重的免疫效应细胞相关血液毒性 (ICAHT)、免疫效应细胞相关噬血细胞性淋巴组织细胞增生症样综合征 (IEC-HS) 和多种微生物感染相关。ICAHT 和 IEC-HS 均对我们的标准治疗方案产生抵抗,但人脐带间充质干细胞输注在改善血细胞减少症方面显示出一定疗效。尽管采用了广谱抗感染方案,但巨细胞病毒血症仍无法控制,导致中枢神经系统感染、神经症状,并最终导致死亡。此外,我们还采用了高维 33 色荧光流式细胞术来描述两次扩增峰之间免疫细胞成分和功能的动态变化。总之,该病例为双相 CAR-T 扩增及相关免疫效应细胞相关毒性提供了新的见解。