Table 2.
Reference | Drug | Study Population and Median Age | N | CR (%) MRD-CR (%) |
Survival | CRS (%) Grade ≥ 3 (%) |
ICANS (%) Grade ≥ 3 (%) |
---|---|---|---|---|---|---|---|
PRWCC [35] | Tisagenlecleucel | Pediatric and young adults 12 y |
185 | 85% 80% |
12-month OS: 72% 12-month EFS: 50% |
63% 21% |
21% 7% |
Pasquini et al. [36] | Tisagenlecleucel | Pediatric 13.2 y |
255 | 85.5% 99.1% (115/116) |
12-month OS: 60.9% 12-month EFS: 52.4% |
55% 16.1% |
27% 9% |
Brissot et al. [37] | CD19-targeting CAR T-cells | Adults 23.8 y |
118 | 91% NA |
12-month OS: 88.9% in CR 12-month OS: 61.9% in non-CR |
88% NA |
NA NA |
Anagnostou et al. [38] | CD19-targeting CAR T-cells | Pediatric and adults NA |
953 | 80% 72% |
12-month OS: 58% | 82% 26% |
29% 12% |
N: number; CR: complete remission; MRD-: minimal residual disease negative; CRS: cytokine release syndrome; ICANS: immune effector cell-associated neurologic syndrome; OS: overall survival; EFS: event-free survival; NA: not available.