Patient characteristics across dose cohorts
| Characteristic . | Dose, × 106 CAR T cells per kg . | P∗ . | |||
|---|---|---|---|---|---|
| 0-1.3 n = 48 . | 1.3-1.7 n = 46 . | 1.7-2.4 n = 43 . | 2.4-5.1 n = 43 . | ||
| Sex | .7 | ||||
| Female | 19 (40) | 15 (33) | 22 (51) | 16 (37) | |
| Male | 29 (60) | 31 (67) | 21 (49) | 27 (63) | |
| Age at diagnosis, y | 13 (8-16) | 8 (3-16) | 6 (2-12) | 5 (3-8) | <.001 |
| Age at infusion, y | 17 (13-20) | 13 (9-18) | 11 (6-16) | 10 (7-13) | <.001 |
| Race/ethnicity | .3 | ||||
| Asian | 2 (4.3) | 0 (0) | 3 (7.1) | 2 (4.8) | |
| Black | 3 (6.5) | 3 (6.8) | 0 (0) | 1 (2.4) | |
| Hispanic | 18 (39) | 21 (48) | 16 (38) | 12 (29) | |
| >1 race | 1 (2.2) | 1 (2.3) | 2 (4.8) | 0 (0) | |
| Non-Hispanic White | 22 (48) | 19 (43) | 21 (50) | 27 (64) | |
| Unknown | 2 | 2 | 1 | 1 | |
| Initial cytogenetic risk | .002 | ||||
| Favorable | 2 (5.0) | 4 (12) | 7 (21) | 10 (33) | |
| Intermediate | 15 (38) | 12 (35) | 12 (36) | 10 (33) | |
| Unfavorable | 23 (57) | 18 (53) | 14 (42) | 10 (33) | |
| Unknown | 8 | 12 | 10 | 13 | |
| N of relapses pre-CAR | 1.50 (1.00-2.00) | 1.00 (1.00-2.00) | 1.00 (1.00-2.00) | 1.00 (1.00-2.00) | >.9 |
| Relapsed or refractory indication | .6 | ||||
| Refractory | 8 (17) | 10 (22) | 7 (16) | 6 (14) | |
| Relapsed | 40 (83) | 36 (78) | 36 (84) | 37 (86) | |
| N of additional lines of therapy pre-CAR | 2.00 (2.00-3.00) | 3.00 (1.00-3.75) | 2.00 (1.00-3.00) | 2.00 (1.50-3.00) | .3 |
| Prior CD19-directed therapy | 11 (23) | 9 (20) | 11 (26) | 6 (14) | .4 |
| Pre-CAR SCT | 13 (27) | 14 (30) | 9 (21) | 9 (21) | .3 |
| Preinfusion disease burden overall | .9 | ||||
| High | 25 (54) | 23 (51) | 21 (50) | 21 (49) | |
| Low | 9 (20) | 8 (18) | 10 (24) | 14 (33) | |
| No detectable disease | 12 (26) | 14 (31) | 11 (26) | 8 (19) | |
| Unknown | 2 | 1 | 1 | 0 | |
| CAR viability, % | 86 (83-92) | 88 (83-93) | 88 (85-92) | 90 (86-94) | .020 |
| Unknown | 0 | 0 | 0 | 1 | |
| Determination of CAR expression (% CAR+viable cells) | 11.6 (8.6-15.4) | 14.1 (11.1-17.7) | 16.0 (12.0-20.9) | 15.7 (9.9-19.1) | .017 |
| Unknown | 1 | 0 | 1 | 2 | |
| Time from diagnosis to CAR T-cell infusion, mo | 28 (17-58) | 34 (12-54) | 30 (12-56) | 39 (12-64) | .8 |
| Characteristic . | Dose, × 106 CAR T cells per kg . | P∗ . | |||
|---|---|---|---|---|---|
| 0-1.3 n = 48 . | 1.3-1.7 n = 46 . | 1.7-2.4 n = 43 . | 2.4-5.1 n = 43 . | ||
| Sex | .7 | ||||
| Female | 19 (40) | 15 (33) | 22 (51) | 16 (37) | |
| Male | 29 (60) | 31 (67) | 21 (49) | 27 (63) | |
| Age at diagnosis, y | 13 (8-16) | 8 (3-16) | 6 (2-12) | 5 (3-8) | <.001 |
| Age at infusion, y | 17 (13-20) | 13 (9-18) | 11 (6-16) | 10 (7-13) | <.001 |
| Race/ethnicity | .3 | ||||
| Asian | 2 (4.3) | 0 (0) | 3 (7.1) | 2 (4.8) | |
| Black | 3 (6.5) | 3 (6.8) | 0 (0) | 1 (2.4) | |
| Hispanic | 18 (39) | 21 (48) | 16 (38) | 12 (29) | |
| >1 race | 1 (2.2) | 1 (2.3) | 2 (4.8) | 0 (0) | |
| Non-Hispanic White | 22 (48) | 19 (43) | 21 (50) | 27 (64) | |
| Unknown | 2 | 2 | 1 | 1 | |
| Initial cytogenetic risk | .002 | ||||
| Favorable | 2 (5.0) | 4 (12) | 7 (21) | 10 (33) | |
| Intermediate | 15 (38) | 12 (35) | 12 (36) | 10 (33) | |
| Unfavorable | 23 (57) | 18 (53) | 14 (42) | 10 (33) | |
| Unknown | 8 | 12 | 10 | 13 | |
| N of relapses pre-CAR | 1.50 (1.00-2.00) | 1.00 (1.00-2.00) | 1.00 (1.00-2.00) | 1.00 (1.00-2.00) | >.9 |
| Relapsed or refractory indication | .6 | ||||
| Refractory | 8 (17) | 10 (22) | 7 (16) | 6 (14) | |
| Relapsed | 40 (83) | 36 (78) | 36 (84) | 37 (86) | |
| N of additional lines of therapy pre-CAR | 2.00 (2.00-3.00) | 3.00 (1.00-3.75) | 2.00 (1.00-3.00) | 2.00 (1.50-3.00) | .3 |
| Prior CD19-directed therapy | 11 (23) | 9 (20) | 11 (26) | 6 (14) | .4 |
| Pre-CAR SCT | 13 (27) | 14 (30) | 9 (21) | 9 (21) | .3 |
| Preinfusion disease burden overall | .9 | ||||
| High | 25 (54) | 23 (51) | 21 (50) | 21 (49) | |
| Low | 9 (20) | 8 (18) | 10 (24) | 14 (33) | |
| No detectable disease | 12 (26) | 14 (31) | 11 (26) | 8 (19) | |
| Unknown | 2 | 1 | 1 | 0 | |
| CAR viability, % | 86 (83-92) | 88 (83-93) | 88 (85-92) | 90 (86-94) | .020 |
| Unknown | 0 | 0 | 0 | 1 | |
| Determination of CAR expression (% CAR+viable cells) | 11.6 (8.6-15.4) | 14.1 (11.1-17.7) | 16.0 (12.0-20.9) | 15.7 (9.9-19.1) | .017 |
| Unknown | 1 | 0 | 1 | 2 | |
| Time from diagnosis to CAR T-cell infusion, mo | 28 (17-58) | 34 (12-54) | 30 (12-56) | 39 (12-64) | .8 |
Data are presented as n (%) or median (interquartile range). Association with dose was tested with linear regression, with dose groups assigned scores of 1-4. Association between dose and ordinal variables was tested with χ2 test of linear-by-linear association. Association between dose and race/ethnicity was tested with χ2 test, with dose treated as ordinal and race/ethnicity as nominal.
Pearson χ2 test; χ2 test of linear-by-linear association; linear regression; χ2 test of ordinal association.