Table 2.
Study (reference) | Patient number | scFv | Costimulatory domain | Median age (range) (y) | CAR-T cell doses | Disease-related outcomes |
---|---|---|---|---|---|---|
Maude et al. (2014) [28] | 30 ALL (25 children, 5 adults) | FMC63 | 4-1BB | Pediatric ALL: 11 (5–22) Adult ALL: 57 (36–60) |
0.76‐20.6 × 106 CAR + T cells/kg | 6-month EFS: 67% 6-month OS: 78% CR: 90% (MRD-negative in 88% of those who achieved CR) |
Brentjens et al. (2011) [81] | 8 CLL adults | SJ25C1 | CD28 | Adults CLL: 58 (67-48) | Cohort receiving no LDC: 1.2–3.0 × 107 CAR + T cells/kg Cy cohort: 0.4‐1.0 × 107 CAR + T cells/kg |
ORR: 1/8 (PR); two others with ≥2 months of SD, all in Cy cohort |
Brudno et al. (2016) [80] | 5 ALL | FMC63 | CD28 | Adults ALL: 25 (20-68) | 5.2‐7.0 × 106 CAR + T cells/kg | CR: 80% (4/5, all MRD-negative) |
Kochenderfer et al. (2015) [82] | 5 CLL adults | FMC63 | CD28 | Adults CLL: 61(48-68) | 1 × 106 CAR + T cells/kg | CR: 60% |
Lee et al. (2014) [76] | 20 B-ALL | FMC63 | CD28 | Children and young adults: 15 (1-30) | 1 × 106 CAR-transduced T cells per kg (dose 1) 3 × 106 CAR-transduced T cells per kg (dose 2) |
CR: 70% (MRD-negative in 86% of those who achieved CR) PD: 20% & SD: 10% (day 28) |
Schuster et al. (2017) [18] | 28 lymphomas | FMC63 | 4-1BB | Adult patients | Median total CTL019-cell: 5.00 × 108 (range, 1.79 × 108 to 5.00 × 108) Median CTL019-cell: 5.79 × 106 cells/kg (range, 3.08 × 106 to 8.87 × 106 cells/kg) |
CR at 6 months: (57%; 95% CI, 37 to 76) CR: 57% median of 28.6 months (range, 3.5 to 37.9) |
EFS: event-free survival; CLL: chronic lymphocytic leukemia; CR: complete response; PR: partial response; SD: stable disease; PD: progressive disease.