Table 1.

Pediatric and YA trials of CD19-directed CAR T-cell therapy

ReferenceT-cell engagerPopulationCR/MRD CRPersistence EFS/OS
8 (single center) CD19 scFv/4-1BB CD3ζ 25 pediatric/YA patients (age 5-22 y) 90%/82% (infused patients) 6-mo EFS, 67% (infused)/OS, 78% (infused) 
5 adult patients (age 26-60 y) 3 patients BMT in CR, 1 patient DLI due to MRD 
15  CD19 scFv/ CD28 CD3ζ 21 pediatric/YA patients (age 5-27 y) 70%/60% (all enrolled patients) 9.7-mo OS, 52% 
LFS of 12 patients achieving MRD was 78.8%; intent was BMT; 10/12 MRD proceeded to BMT; 2 ineligible for BMT relapsed 
16  CD19 scFv/4-1BB CD3ζ 45 pediatric/YA patients (age 1-25 y) 89% (all enrolled), 93% (infused); all patients with MRD CR 1-y EFS, 50.8% (infused)/OS, 65.9% (infused) 
11 patients received BMT 
9 (ELIANA multicenter trial) CD19 scFv/4-1BB CD3ζ 92 enrolled, 75 infused pediatric/YA patients (age 3-23 y) 66% (all enrolled) 1-y EFS, 50% (infused)/OS, 76% (infused) 
81% (infused); all CRs required 2 time points 4 wk apart and were MRD 8 patients BMT (6 in MRD remission, 2 with MRD+ disease) 
ReferenceT-cell engagerPopulationCR/MRD CRPersistence EFS/OS
8 (single center) CD19 scFv/4-1BB CD3ζ 25 pediatric/YA patients (age 5-22 y) 90%/82% (infused patients) 6-mo EFS, 67% (infused)/OS, 78% (infused) 
5 adult patients (age 26-60 y) 3 patients BMT in CR, 1 patient DLI due to MRD 
15  CD19 scFv/ CD28 CD3ζ 21 pediatric/YA patients (age 5-27 y) 70%/60% (all enrolled patients) 9.7-mo OS, 52% 
LFS of 12 patients achieving MRD was 78.8%; intent was BMT; 10/12 MRD proceeded to BMT; 2 ineligible for BMT relapsed 
16  CD19 scFv/4-1BB CD3ζ 45 pediatric/YA patients (age 1-25 y) 89% (all enrolled), 93% (infused); all patients with MRD CR 1-y EFS, 50.8% (infused)/OS, 65.9% (infused) 
11 patients received BMT 
9 (ELIANA multicenter trial) CD19 scFv/4-1BB CD3ζ 92 enrolled, 75 infused pediatric/YA patients (age 3-23 y) 66% (all enrolled) 1-y EFS, 50% (infused)/OS, 76% (infused) 
81% (infused); all CRs required 2 time points 4 wk apart and were MRD 8 patients BMT (6 in MRD remission, 2 with MRD+ disease) 

BMT, bone marrow transplantation; CR, complete remission; LFS, leukemia-free survival; OS, overall survival; YA, young adult.

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