Summary of CD19-directed CAR T-cell trials in pediatric R/R ALL
| Trial . | Costimulatory domain . | Population . | N* . | CR rate (%) . | Subsequent HSCT n (%)† . | Outcome . | CAR T-cell persistence . |
|---|---|---|---|---|---|---|---|
| Penn/CHOP phase 1/2a37,39 | 4-1BB | R/R ALL: refractory, relapse after HSCT, ineligible for HSCT | 6039 | 9339 | 7 (13)39 | 60% RFS at 12 months (95% CI, 48-75%)39 | 68% at 6 months (95% CI, 50-92%)‡37 |
| NCI phase 136,52 | CD28 | R/R B-ALL: second relapse or greater, refractory, ineligible for HSCT | 5152 | 60.852 | 21 (75)52 | 49.5% LFS at 18 months52 | Longest, 68 days§36 |
| Seattle phase 1/232 | 4-1BB | R/R ALL: second relapse or greater, refractory, MRD after HSCT, ineligible for HSCT | 43 | 93 | 11 (28) | 50.8% EFS at 12 months (95% CI, 36.9-69.9%) | Median, 3 months (range, 2.07-6.44 months)|| |
| MSKCC phase 153 | CD28 | R/R B-ALL: second relapse or greater, very early (CR1 <18 months) BM relapse, refractory, ineligible for HSCT | 25 | 75 | 15 (83) | 8 of 18 in remission, median follow-up, 28.6 months | Median, 7 days (range, 0-234 days) |
| ELIANA phase 233 | 4-1BB | R/R B-ALL: second relapse or greater, refractory, relapse after HSCT | 75 | 81 | 8 (13) | 80% RFS at 6 months, 59% RFS at 12 months | Median, 168 days (range, 20-617 days) |
| Trial . | Costimulatory domain . | Population . | N* . | CR rate (%) . | Subsequent HSCT n (%)† . | Outcome . | CAR T-cell persistence . |
|---|---|---|---|---|---|---|---|
| Penn/CHOP phase 1/2a37,39 | 4-1BB | R/R ALL: refractory, relapse after HSCT, ineligible for HSCT | 6039 | 9339 | 7 (13)39 | 60% RFS at 12 months (95% CI, 48-75%)39 | 68% at 6 months (95% CI, 50-92%)‡37 |
| NCI phase 136,52 | CD28 | R/R B-ALL: second relapse or greater, refractory, ineligible for HSCT | 5152 | 60.852 | 21 (75)52 | 49.5% LFS at 18 months52 | Longest, 68 days§36 |
| Seattle phase 1/232 | 4-1BB | R/R ALL: second relapse or greater, refractory, MRD after HSCT, ineligible for HSCT | 43 | 93 | 11 (28) | 50.8% EFS at 12 months (95% CI, 36.9-69.9%) | Median, 3 months (range, 2.07-6.44 months)|| |
| MSKCC phase 153 | CD28 | R/R B-ALL: second relapse or greater, very early (CR1 <18 months) BM relapse, refractory, ineligible for HSCT | 25 | 75 | 15 (83) | 8 of 18 in remission, median follow-up, 28.6 months | Median, 7 days (range, 0-234 days) |
| ELIANA phase 233 | 4-1BB | R/R B-ALL: second relapse or greater, refractory, relapse after HSCT | 75 | 81 | 8 (13) | 80% RFS at 6 months, 59% RFS at 12 months | Median, 168 days (range, 20-617 days) |
CHOP, Children’s Hospital of Philadelphia; EFS, event-free survival; LFS, leukemia-free survival; MSKCC, Memorial Sloan Kettering Cancer Center; NCI, National Cancer Institute; Penn, University of Pennsylvania; RFS, relapse-free survival; R/R, relapsed/refractory.
*Numbers of patients infused.
†Percentage of patients in remission.
‡Probability of persistence.
§Longest duration in any patient.
Measured by B-cell aplasia.