Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda.
Clinical Monitoring Research Program, Leidos Biomedical Research Inc., NCI Campus at Frederick, Frederick, MD.
J Immunother. 2018 Sep;41(7):350-358. doi: 10.1097/CJI.0000000000000241.
Neurotoxicity associated with CAR-T cell therapy can be life-threatening. With rapid development of CAR-T therapies, a systematic method is needed to identify and monitor symptoms of neurotoxicity, elucidate potential etiologies, and compare toxicity across trials. This paper presents a systematic evaluation developed and used to prospectively assess neurotoxicity in our phase I anti-CD22 CAR-T-cell trial and describes the symptoms of neurotoxicity identified using this methodology. Central nervous system (CNS) studies included routine lumbar punctures performed for disease evaluation pretherapy and posttherapy and a baseline brain MRI. Brief cognitive evaluations, assessing 4 domains (attention, working memory, cognitive flexibility, and processing speed), were administered preinfusion and postinfusion. A newly developed CAR-T-specific neurological symptom checklist (NSC) was completed by caregivers at 3 designated time-points. Serial serum cytokine levels were compared with neurotoxicity symptoms and severity. The majority of the first 22 consecutively treated subjects (ages, 7-30) demonstrated stable or improved cognitive test scores following therapy and no irreversible neurotoxicity, despite CAR-T-related antileukemic response, cytokine release syndrome, and trafficking of CAR-T cells to the CSF. The NSC allowed us to document the type and timing of symptoms and explore the etiology of neurotoxicity associated with CD22 CAR-T therapy. Cytokine profiling demonstrated that more concerning symptoms of neurotoxicity, such as hallucination and disorientation, were significantly associated with higher serum cytokine levels, supporting the hypothesis of inflammation-driven neurotoxicity. Systematic assessments of neurotoxicity were feasible in acutely ill children and young adults and served to characterize and monitor the symptoms associated with CAR-T therapy. We recommend these evaluations be incorporated into future immunotherapy protocols.
细胞疗法相关的神经毒性可能危及生命。随着嵌合抗原受体 T 细胞(CAR-T)疗法的快速发展,需要一种系统的方法来识别和监测神经毒性症状,阐明潜在的病因,并比较试验间的毒性。本文提出了一种系统评价方法,用于前瞻性评估我们的抗 CD22 CAR-T 细胞 I 期试验中的神经毒性,并描述了使用该方法确定的神经毒性症状。中枢神经系统(CNS)研究包括在治疗前和治疗后进行常规的腰椎穿刺以评估疾病,以及基线脑 MRI。在输注前和输注后进行简短的认知评估,评估 4 个领域(注意力、工作记忆、认知灵活性和处理速度)。护理人员在 3 个指定时间点完成新开发的 CAR-T 特异性神经症状检查表(NSC)。比较了血清细胞因子水平与神经毒性症状和严重程度的关系。前 22 名连续接受治疗的患者(年龄 7-30 岁),尽管存在 CAR-T 相关的抗白血病反应、细胞因子释放综合征和 CAR-T 细胞向 CSF 转移,但大多数患者在治疗后认知测试评分稳定或改善,没有不可逆的神经毒性。NSC 使我们能够记录症状的类型和时间,并探讨与 CD22 CAR-T 治疗相关的神经毒性的病因。细胞因子谱分析表明,更严重的神经毒性症状,如幻觉和定向障碍,与更高的血清细胞因子水平显著相关,支持炎症驱动的神经毒性假说。对急性病儿童和青少年进行神经毒性的系统评估是可行的,并有助于对与 CAR-T 治疗相关的症状进行特征描述和监测。我们建议将这些评估纳入未来的免疫治疗方案。
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