biostudies-literatureTalleur ACNCI NIH HHS5737-5749https://www.ebi.ac.uk/biostudies/studies/S-EPMC9647829biostudies-literatureUnknown6(21)T cells expressing CD19-specific chimeric antigen receptors (CD19-CARs) have potent antileukemia activity in pediatric and adult patients with relapsed and/or refractory B-cell acute lymphoblastic leukemia (B-ALL). However, not all patients achieve a complete response (CR), and a significant percentage relapse after CD19-CAR T-cell therapy due to T-cell intrinsic and/or extrinsic mechanisms. Thus, there is a need to evaluate new CD19-CAR T-cell products in patients to improve efficacy. We developed a phase 1/2 clinical study to evaluate an institutional autologous CD19-CAR T-cell product in pediatric patients with relapsed/refractory B-ALL. Here we report the outcome of the phase 1 study participants (n = 12). Treatment was well tolerated, with a low incidence of both cytokine release syndBlood advancesPreferential expansion of CD8+ CD19-CAR T cells postinfusion and the role of disease burden on outcome in pediatric B-ALL.PMC9647829P30 CA021765R01 CA237311Metais JYMadden RInaba HLockey TPui CHHurley CSuliman ATriplett BMSharma AQudeimat APatil SLBragg ACrawford JCAkel SWillis CTuggle-Brown MLangfitt DYoungblood BCheng CSchell SObeng EATalleur ACGeiger TLGottschalk SSrinivasan AZheng WMamcarz EMeagher MMHuang SLi YKarol SEZebley CCullins DVelasquez MPZhou SMThomas PGfalsePreferential expansion of CD8+ CD19-CAR T cells postinfusion and the role of disease burden on outcome in pediatric B-ALL.T cells expressing CD19-specific chimeric antigen receptors (CD19-CARs) have potent antileukemia activity in pediatric and adult patients with relapsed and/or refractory B-cell acute lymphoblastic leukemia (B-ALL). However, not all patients achieve a complete response (CR), and a significant percentage relapse after CD19-CAR T-cell therapy due to T-cell intrinsic and/or extrinsic mechanisms. Thus, there is a need to evaluate new CD19-CAR T-cell products in patients to improve efficacy. We developed a phase 1/2 clinical study to evaluate an institutional autologous CD19-CAR T-cell product in pediatric patients with relapsed/refractory B-ALL. Here we report the outcome of the phase 1 study participants (n = 12). Treatment was well tolerated, with a low incidence of both cytokine release synd2022-01-01T00:00:00Z2022 Nov2026-05-31T23:09:57.562Z2024-11-20T15:24:29.929ZS-EPMC96478293544693410.1182/bloodadvances.2021006293