Single-cell expression and TCR data from CD19-specific CAR T cells in a phase I/II clinical trial

By leveraging single-cell transcriptome and T cell receptor (TCR) sequencing, we aimed to track the transcriptional signatures of CAR T cell clonotypes throughout the course of treatment and furthermore identify molecular patterns leading to potent CAR T cell cytotoxicity. The data presented in this study encompass blood and bone marrow samples from patients ≤ 21 years of age with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) participating in the SJCAR19 phase I/II clinical trial (NCT03573700). In brief, patients enrolled in the clinical trial received either 1 x 10^6 (dose level 1) or 3 x 10^6 (dose level 2) per kilogram of body weight following successful generation of autologous CAR T cell products and lymphodepleting chemotherapy. Peripheral blood was drawn from each participant every week until week 4 post-infusion, at week 6 or 8, and month 3 or 6 if feasible. At week 4 post-infusion, blood marrow was also collected from participants. Total T cells (CD3+) were...

本研究借助单细胞转录组与T细胞受体（TCR）测序技术，旨在追踪治疗全程中嵌合抗原受体T细胞（chimeric antigen receptor T cell, CAR T）克隆型的转录特征，并进一步识别介导强效CAR T细胞细胞毒性的分子模式。本研究收录的样本，来自参与SJCAR19Ⅰ/Ⅱ期临床试验（NCT03573700）的复发或难治性B细胞急性淋巴细胞白血病（B-ALL）患者，患者年龄均不超过21岁，样本类型涵盖外周血与骨髓。简言之，入组临床试验的患者在成功制备自体CAR T细胞产品并完成淋巴清除化疗后，将按每公斤体重接受1×10^6个细胞（剂量水平1）或3×10^6个细胞（剂量水平2）的输注。所有受试者均在输注后每周采集外周血，直至输注后第4周；若条件允许，还会在输注后第6或8周、第3或6个月进行采样。输注后第4周时，同时采集受试者的骨髓样本。研究将对总T细胞（CD3+）进行...