Summary of primary, key secondary, and secondary efficacy outcomes (ITT set)
| . | Liso-cel (n = 92) . | SOC (n = 92) . | Liso-cel vs SOC . |
|---|---|---|---|
| Primary efficacy end point per IRC | |||
| EFS | Stratified HR (95% CI) | ||
| Patients with events, n (%) | 44 (48) | 71 (77) | — |
| Median (95% CI) EFS, mo∗ | NR (9.5-NR) | 2.4 (2.2-4.9) | 0.356 (0.243-0.522) |
| 12-mo EFS rate, % (95% CI)† | 57.1 (47.0-67.3) | 22.5 (13.9-31.2) | — |
| 18-mo EFS rate, % (95% CI)† | 52.6 (42.3-62.9) | 20.8 (12.2-29.5) | — |
| Key secondary efficacy end points | |||
| CR rate per IRC | Stratified 1-sided P value | ||
| CR, n (%); 95% CI‡ | 68 (74); 63.7-82.5 | 40 (43); 33.2-54.2 | <.0001 |
| PFS per IRC | Stratified HR (95% CI); P value§ | ||
| Patients with events, n (%) | 37 (40) | 52 (57) | — |
| Median (95% CI) PFS, mo∗ | NR (12.6-NR) | 6.2 (4.3-8.6) | 0.400 (0.261-0.615); <.0001 |
| 12-mo PFS rate, % (95% CI)† | 63.1 (53.0-73.3) | 31.2 (20.2-42.3) | — |
| 18-mo PFS rate, % (95% CI)† | 58.2 (47.7-68.7) | 28.8 (17.7-40.0) | — |
| OS | Stratified HR (95% CI); P value§ | ||
| Patients with events, n (%) | 28 (30) | 38 (41) | — |
| Median (95% CI) OS, mo∗ | NR (29.5-NR) | 29.9 (17.9-NR) | 0.724 (0.443-1.183); .0987 |
| 12-mo OS rate, % (95% CI)† | 83.4 (75.7-91.1) | 72.0 (62.7-81.3) | — |
| 18-mo OS rate, % (95% CI)† | 73.1 (63.9-82.3) | 60.6 (50.2-71.1) | — |
| Secondary efficacy end points per IRC | |||
| ORR | |||
| ORR, n (%); 95% CI‡ | 80 (87); 78.3-93.1 | 45 (49); 38.3-59.6 | — |
| DOR | Stratified HR (95% CI) | ||
| Patients with events, n/N (%) | 31/80 (39) | 25/45 (56) | — |
| Median (95% CI) DOR, mo∗ | NR (13.4-NR) | 9.1 (5.1-NR) | 0.579 (0.340-0.984) |
| Duration of CR | |||
| Patients with events, n/N (%) | 21/68 (31) | 21/40 (52.5) | — |
| Median (95% CI) DOR, mo∗ | NR (NR-NR) | 9.3 (5.1-NR) | 0.483 (0.262-0.890) |
| . | Liso-cel (n = 92) . | SOC (n = 92) . | Liso-cel vs SOC . |
|---|---|---|---|
| Primary efficacy end point per IRC | |||
| EFS | Stratified HR (95% CI) | ||
| Patients with events, n (%) | 44 (48) | 71 (77) | — |
| Median (95% CI) EFS, mo∗ | NR (9.5-NR) | 2.4 (2.2-4.9) | 0.356 (0.243-0.522) |
| 12-mo EFS rate, % (95% CI)† | 57.1 (47.0-67.3) | 22.5 (13.9-31.2) | — |
| 18-mo EFS rate, % (95% CI)† | 52.6 (42.3-62.9) | 20.8 (12.2-29.5) | — |
| Key secondary efficacy end points | |||
| CR rate per IRC | Stratified 1-sided P value | ||
| CR, n (%); 95% CI‡ | 68 (74); 63.7-82.5 | 40 (43); 33.2-54.2 | <.0001 |
| PFS per IRC | Stratified HR (95% CI); P value§ | ||
| Patients with events, n (%) | 37 (40) | 52 (57) | — |
| Median (95% CI) PFS, mo∗ | NR (12.6-NR) | 6.2 (4.3-8.6) | 0.400 (0.261-0.615); <.0001 |
| 12-mo PFS rate, % (95% CI)† | 63.1 (53.0-73.3) | 31.2 (20.2-42.3) | — |
| 18-mo PFS rate, % (95% CI)† | 58.2 (47.7-68.7) | 28.8 (17.7-40.0) | — |
| OS | Stratified HR (95% CI); P value§ | ||
| Patients with events, n (%) | 28 (30) | 38 (41) | — |
| Median (95% CI) OS, mo∗ | NR (29.5-NR) | 29.9 (17.9-NR) | 0.724 (0.443-1.183); .0987 |
| 12-mo OS rate, % (95% CI)† | 83.4 (75.7-91.1) | 72.0 (62.7-81.3) | — |
| 18-mo OS rate, % (95% CI)† | 73.1 (63.9-82.3) | 60.6 (50.2-71.1) | — |
| Secondary efficacy end points per IRC | |||
| ORR | |||
| ORR, n (%); 95% CI‡ | 80 (87); 78.3-93.1 | 45 (49); 38.3-59.6 | — |
| DOR | Stratified HR (95% CI) | ||
| Patients with events, n/N (%) | 31/80 (39) | 25/45 (56) | — |
| Median (95% CI) DOR, mo∗ | NR (13.4-NR) | 9.1 (5.1-NR) | 0.579 (0.340-0.984) |
| Duration of CR | |||
| Patients with events, n/N (%) | 21/68 (31) | 21/40 (52.5) | — |
| Median (95% CI) DOR, mo∗ | NR (NR-NR) | 9.3 (5.1-NR) | 0.483 (0.262-0.890) |
All percentages are rounded to whole numbers except those with “.5%.”
— denotes not available or not reported.
Median estimates of time to event were Kaplan-Meier product-limit estimates.
Based on Greenwood formula; 2-sided CI.
Based on Cochran-Mantel-Haenszel test; 2-sided CI.
Based on a stratified Cox proportional hazards model; 1-sided P value.