Summary of efficacy end points (liso-cel–treated set)
| . | Outpatients (n = 57) . | Inpatients (n = 25) . | Total (N = 82) . |
|---|---|---|---|
| Best overall response,∗ n (%) | |||
| CR | 33 (58) | 11 (44) | 44 (54) |
| PR | 14 (25) | 8 (32) | 22 (27) |
| SD | 3 (5) | 3 (12) | 6 (7) |
| PD | 7 (12) | 3 (12) | 10 (12) |
| ORR | |||
| CR + PR, n (%) | 47 (82) | 19 (76) | 66 (80) |
| 95% CI† | 70.1-91.3 | 54.9-90.6 | 70.3-88.4 |
| CR rate | |||
| CR, n (%) | 33 (58) | 11 (44) | 44 (54) |
| 95% CI† | 44.1-70.9 | 24.4-65.1 | 42.3-64.7 |
| Median DOR (95% CI),‡ mo | 11.1 (3.9 to NR) | NR (2.1 to NR) | 14.75 (5.0 to NR) |
| Median follow-up (95% CI),§ mo | 17.7 (16.95-22.8) | 17.3 (7.75-23.3) | 17.35 (16.95-22.7) |
| 6-mo probability of continued response, % (95% CI)‡ | 54 (38.9-67.3) | 58 (33.2-76.3) | 55 (42.5-66.4) |
| 12-mo probability of continued response, % (95% CI)‡ | 50 (34.6-63.1) | 51 (26.0-70.9) | 50 (37.5-61.8) |
| Median DOR in patients achieving CR (95% CI),‡ mo | NR (11.1 to NR) | NR (8.7 to NR) | NR (16.6 to NR) |
| Median follow-up (95% CI),§ mo | 17.4 (16.95-22.8) | 17.3 (7.7-23.5) | 17.7 (16.95-22.8) |
| 6-mo probability of continued response, % (95% CI)‡ | 75 (56.0-86.6) | 91 (50.8-98.7) | 79 (63.5-88.5) |
| 12-mo probability of continued response, % (95% CI)‡ | 68 (49.1-81.6) | 79.5 (39.3-94.5) | 71.5 (55.2-82.7) |
| Median PFS (95% CI),‡ mo | 6.05 (2.9 to NR) | 4.3 (2.8 to NR) | 5.8 (3.0-15.6) |
| Median follow-up (95% CI),§ mo | 18.6 (17.8-23.7) | 18.0 (8.5-24.0) | 18.2 (17.8-23.7) |
| 6-mo probability of PFS, % (95% CI)‡ | 51 (37.3-62.9) | 44 (24.5-61.9) | 49 (37.6-59.0) |
| 12-mo probability of PFS, % (95% CI)‡ | 42 (28.6-54.0) | 38.5 (19.5-57.3) | 41 (30.1-51.4) |
| Median OS (95% CI),‡ mo | NR (9.2 to NR) | 22.2 (8.0 to NR) | NR (10.6 to NR) |
| Median follow-up (95% CI),§ mo | 22.0 (18.2-23.7) | 18.3 (11.1-24.05) | 22.0 (18.0-23.6) |
| 6-mo probability of OS, % (95% CI)‡ | 85 (72.3-92.2) | 78 (55.6-90.4) | 83 (72.5-89.7) |
| 12-mo probability of OS, % (95% CI)‡ | 62 (46.5-73.7) | 60 (36.6-76.8) | 61 (48.9-71.5) |
| . | Outpatients (n = 57) . | Inpatients (n = 25) . | Total (N = 82) . |
|---|---|---|---|
| Best overall response,∗ n (%) | |||
| CR | 33 (58) | 11 (44) | 44 (54) |
| PR | 14 (25) | 8 (32) | 22 (27) |
| SD | 3 (5) | 3 (12) | 6 (7) |
| PD | 7 (12) | 3 (12) | 10 (12) |
| ORR | |||
| CR + PR, n (%) | 47 (82) | 19 (76) | 66 (80) |
| 95% CI† | 70.1-91.3 | 54.9-90.6 | 70.3-88.4 |
| CR rate | |||
| CR, n (%) | 33 (58) | 11 (44) | 44 (54) |
| 95% CI† | 44.1-70.9 | 24.4-65.1 | 42.3-64.7 |
| Median DOR (95% CI),‡ mo | 11.1 (3.9 to NR) | NR (2.1 to NR) | 14.75 (5.0 to NR) |
| Median follow-up (95% CI),§ mo | 17.7 (16.95-22.8) | 17.3 (7.75-23.3) | 17.35 (16.95-22.7) |
| 6-mo probability of continued response, % (95% CI)‡ | 54 (38.9-67.3) | 58 (33.2-76.3) | 55 (42.5-66.4) |
| 12-mo probability of continued response, % (95% CI)‡ | 50 (34.6-63.1) | 51 (26.0-70.9) | 50 (37.5-61.8) |
| Median DOR in patients achieving CR (95% CI),‡ mo | NR (11.1 to NR) | NR (8.7 to NR) | NR (16.6 to NR) |
| Median follow-up (95% CI),§ mo | 17.4 (16.95-22.8) | 17.3 (7.7-23.5) | 17.7 (16.95-22.8) |
| 6-mo probability of continued response, % (95% CI)‡ | 75 (56.0-86.6) | 91 (50.8-98.7) | 79 (63.5-88.5) |
| 12-mo probability of continued response, % (95% CI)‡ | 68 (49.1-81.6) | 79.5 (39.3-94.5) | 71.5 (55.2-82.7) |
| Median PFS (95% CI),‡ mo | 6.05 (2.9 to NR) | 4.3 (2.8 to NR) | 5.8 (3.0-15.6) |
| Median follow-up (95% CI),§ mo | 18.6 (17.8-23.7) | 18.0 (8.5-24.0) | 18.2 (17.8-23.7) |
| 6-mo probability of PFS, % (95% CI)‡ | 51 (37.3-62.9) | 44 (24.5-61.9) | 49 (37.6-59.0) |
| 12-mo probability of PFS, % (95% CI)‡ | 42 (28.6-54.0) | 38.5 (19.5-57.3) | 41 (30.1-51.4) |
| Median OS (95% CI),‡ mo | NR (9.2 to NR) | 22.2 (8.0 to NR) | NR (10.6 to NR) |
| Median follow-up (95% CI),§ mo | 22.0 (18.2-23.7) | 18.3 (11.1-24.05) | 22.0 (18.0-23.6) |
| 6-mo probability of OS, % (95% CI)‡ | 85 (72.3-92.2) | 78 (55.6-90.4) | 83 (72.5-89.7) |
| 12-mo probability of OS, % (95% CI)‡ | 62 (46.5-73.7) | 60 (36.6-76.8) | 61 (48.9-71.5) |
All percentages are rounded to whole numbers except those with .5%.
PD, progressive disease; SD, stable disease.
Best disease response recorded from the time of liso-cel infusion until disease progression, end of study, or the start of another anticancer therapy. Best response is assigned according to the following order: CR, PR, SD, PD, not evaluable, or not available. PDs are counted even if determined by clinical assessment only.
Two-sided 95% exact Clopper-Pearson CIs.
Kaplan-Meier method was used to obtain 2-sided 95% CIs.
Reverse Kaplan-Meier method was used to obtain median follow-up and its 95% CIs.