Published evidence for association between increased CAR T-cell expansion and clinical outcomes

StudyClinical settingPatients with CAR T-cell expansion dataCAR T-cell quantification assayAssociation with neurotoxicityAssociation with CRSAssociation with response to treatment
Hamilton et al1  Standard of care Axi-cel, n = 124 (LBCL) and n = 19 (FL); brexu-cel, n = 17 (MCL) Flow cytometry Yes, with grade ≥3 ICANS Yes, with grade ≥2 CRS Not with PFS or with ongoing response 
Wang et al2  ZUMA-2 Brexu-cel, n = 67 (MCL) qPCR Yes, with grade ≥3 NE Yes, with grade ≥3 CRS Yes, with ORR and with ongoing response 
Jacobson et al5  ZUMA-5 Axi-cel, n = 124 (FL) and n = 24 (MZL) qPCR Yes, with grade ≥3 NE in FL Yes, with grade ≥3 CRS in FL Yes, with ongoing response in FL 
Neelapu et al6  ZUMA-1 Axi-cel, n = 101 (LBCL) qPCR Yes, with grade ≥3 NE Not with grade ≥3 CRS Yes, with ORR and with ongoing response 
Abramson et al7  TRANSCEND NHL 001 Liso-cel, n = 245 (LBCL) qPCR Yes, with grade ≥3 NE Yes, with any grade CRS Yes, with ORR 
Filosto et al8  ZUMA-7 Axi-cel, n = 162 (LBCL) qPCR Yes, with grade ≥3 NE Yes, with grade ≥3 CRS Yes, with ORR but not with ongoing response 
Good et al9  Standard of care Axi-cel, n = 32 (LBCL) Flow cytometry and qPCR Yes, with grade ≥2 ICANS Yes, with grade 2 CRS Not with ORR or with ongoing response 
Blumenberg et al10  Standard of care Axi-cel, n = 60 (LBCL); tisa-cel, n = 58 (LBCL) Flow cytometry Not assessed Not assessed Yes, with PFS and with OS 
StudyClinical settingPatients with CAR T-cell expansion dataCAR T-cell quantification assayAssociation with neurotoxicityAssociation with CRSAssociation with response to treatment
Hamilton et al1  Standard of care Axi-cel, n = 124 (LBCL) and n = 19 (FL); brexu-cel, n = 17 (MCL) Flow cytometry Yes, with grade ≥3 ICANS Yes, with grade ≥2 CRS Not with PFS or with ongoing response 
Wang et al2  ZUMA-2 Brexu-cel, n = 67 (MCL) qPCR Yes, with grade ≥3 NE Yes, with grade ≥3 CRS Yes, with ORR and with ongoing response 
Jacobson et al5  ZUMA-5 Axi-cel, n = 124 (FL) and n = 24 (MZL) qPCR Yes, with grade ≥3 NE in FL Yes, with grade ≥3 CRS in FL Yes, with ongoing response in FL 
Neelapu et al6  ZUMA-1 Axi-cel, n = 101 (LBCL) qPCR Yes, with grade ≥3 NE Not with grade ≥3 CRS Yes, with ORR and with ongoing response 
Abramson et al7  TRANSCEND NHL 001 Liso-cel, n = 245 (LBCL) qPCR Yes, with grade ≥3 NE Yes, with any grade CRS Yes, with ORR 
Filosto et al8  ZUMA-7 Axi-cel, n = 162 (LBCL) qPCR Yes, with grade ≥3 NE Yes, with grade ≥3 CRS Yes, with ORR but not with ongoing response 
Good et al9  Standard of care Axi-cel, n = 32 (LBCL) Flow cytometry and qPCR Yes, with grade ≥2 ICANS Yes, with grade 2 CRS Not with ORR or with ongoing response 
Blumenberg et al10  Standard of care Axi-cel, n = 60 (LBCL); tisa-cel, n = 58 (LBCL) Flow cytometry Not assessed Not assessed Yes, with PFS and with OS 

Liso-cel, lisocabtagene maraleucel; MZL, marginal zone lymphoma; NE, neurological events; ORR, objective response rate; OS, overall survival; PFS, progression-free survival; qPCR, quantitative polymerase chain reaction; tisa-cel, tisagenlecleucel.

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