Table 2.
LD regimens used in completed pediatric trials of CD19 CAR T-cells
| Study | Patient age (median) | LD regimen (# of patients) | Total # of CTL019 cells/kg body weight (# of infusions) |
|---|---|---|---|
| Grupp et al (2013)41 | 7 | None | 1.2×107 (3) |
| 10 | Etoposide – cyclophosphamide (1) | 1.4×106 (1) | |
| Maude et al (2014)23 | 5–60 (14) | Fludarabine 30 mg/m2 daily × 4 days, cyclophosphamide 500 mg/m2 daily × 2 days (13) | 0.76×106–20.6×106 (1–2) |
| Etoposide 100 mg/m2 daily × 2 days, cyclophosphamide 440 mg/m2 daily × 2 days (5) | |||
| None (3) | |||
| Fludarabine 30 mg/m2 daily × 3 days, cyclophosphamide 300 mg/m2 daily × 3 days (2) | |||
| Cyclophosphamide 300 mg/m2 every 12 hours × 3 days (2) | |||
| Etoposide 150 mg/m2 daily × 1 day, cytarabine 300 mg/m2 daily × 1 day (1) | |||
| Cyclophosphamide 1,000 mg/m2 daily × 1 day (1) | |||
| Clofarabine 30 mg/m2 daily × 5 days (1) | |||
| Methotrexate 1,000 mg/m2 day 1, cytarabine 1,000 mg/m2 every 12 hours days 2 and 3 (1) | |||
| Cyclophosphamide 300 mg/m2 every 12 hours days 1–3, vincristine 2 mg day 3, Adriamycin 50 mg/m2 day 3 (1) | |||
| Maude et al (2016)44 | 4–24 (11) | LD – unknown (59) | 1×106–17.4×106 (1–3) |
| Maude et al (2018)45 | 3–23 (11) | Fludarabine – cyclophosphamide (71) | 0.2×106–5.4×106 (1) |
| None (3) | |||
| Cytarabine – etoposide (1) | |||
| Gardner et al | 1.3–25.4 (12.3) | Cyclophosphamide (27) | 0.5×106–10×106 (1–2) |
| (2017)52 | Fludarabine 30 mg/m2 daily × 4 days, cyclophosphamide 500 mg/m2 daily × 2 days (14) | ||
| None (2) | |||
| Lee et al (2015) (KTE-C19)42 | 4–27 (13.6) | Fludarabine 25 mg/m2 daily × 3 days, cyclophosphamide 900 mg/m2 daily × 1 day (28) | 1×106–3×106 (1) |
| FLAG (6) | |||
| Ifosfamide – etoposide (2) | |||
| Fludarabine 30 mg/m2 daily × 4 days, cyclophosphamide 1,200 mg/m2 daily × 2 days (3) |
Abbreviations: CAR, chimeric antigen receptor; FLAG, fludarabine, cytarabine, granulocyte colony stimulating factor; LD, lymphodepletion.