Outcomes of patients with R/R CNS disease, non-CNS EM disease, and the rest of the cohort after tisagenlecleucel infusion
| Outcome . | CNS disease (n = 40) . | Non-CNS EM disease (n = 15) . | BM-only disease (n = 129) . | P . |
|---|---|---|---|---|
| CRS | .30 | |||
| Grade 1 | 15 | 3 | 27 | |
| Grade 2 | 5 | 4 | 23 | |
| Grade 3 | 3 | 2 | 14 | |
| Grade 4 | 2 | 3 | 14 | |
| Grade 5 | 0 | 0 | 1 | |
| Unknown | 1 | 0 | 0 | |
| ICANS | .39 | |||
| Grade 1 | 9 | 2 | 8 | |
| Grade 2 | 2 | 0 | 5 | |
| Grade 3 | 1 | 0 | 7 | |
| Grade 4 | 2 | 0 | 2 | |
| Treatment of toxicity* | .14 | |||
| Tocilizumab | 10 | 5 | 31 | |
| Steroids | 6 | 2 | 18 | |
| Other | 2 | 0 | 4 | |
| Response | .35 | |||
| No CR | 4 | 4 | 15 | |
| CR (% MRD negative) | 35 (97%) | 10 (90%) | 111 (95%) | |
| Died before day 28 | 1 | 1 | 3 | |
| Relapsed post-CAR | 15 | 6 | 45 | .91 |
| Site of relapse | ||||
| CNS | 5 | 0 | 0 | |
| CNS + BM | 2 | 0 | 2 | |
| BM only | 8 | 2 | 37 | |
| BM + other EM | 0 | 2 | 4 | |
| Other EM disease | 0 | 2 | 2 | |
| CD19-negative relapse | 5/15 | 1/6 | 20/38 | .025 |
| Unknown | 2 | 3 | 3 | |
| Time from infusion to relapse, d | 101 (30-577) | 95 (30-245) | 90 (28-645) | .87 |
| SCT post CAR | 6 | 1 | 38 | .06 |
| Rationale for SCT post-CAR | .52 | |||
| Preemptive | 1 | 0 | 5 | |
| Loss of BCA | 0 | 0 | 11 | |
| Refractory/relapse | 4 | 1 | 16 | |
| MLL rearranged | 1 | 0 | 2 | |
| Alive/dead | 31/9 | 9/6 | 93/36 | .40 |
| Cause of death | .87 | |||
| Relapse | 7 | 5 | 26 | |
| Infection | 2 | 0 | 4 | |
| CRS | 0 | 0 | 1 | |
| Neurotoxicity | 0 | 0 | 1 | |
| Transplant related | 0 | 1 | 3 | |
| Cardiac related | 0 | 0 | 1 | |
| Loss of BCA | 10 | 8 | 39 | .14 |
| Time from infusion to loss of BCA, d | 174 (36-266) | 84.5 (29-396) | 93 (28-545) | .23 |
| Outcome . | CNS disease (n = 40) . | Non-CNS EM disease (n = 15) . | BM-only disease (n = 129) . | P . |
|---|---|---|---|---|
| CRS | .30 | |||
| Grade 1 | 15 | 3 | 27 | |
| Grade 2 | 5 | 4 | 23 | |
| Grade 3 | 3 | 2 | 14 | |
| Grade 4 | 2 | 3 | 14 | |
| Grade 5 | 0 | 0 | 1 | |
| Unknown | 1 | 0 | 0 | |
| ICANS | .39 | |||
| Grade 1 | 9 | 2 | 8 | |
| Grade 2 | 2 | 0 | 5 | |
| Grade 3 | 1 | 0 | 7 | |
| Grade 4 | 2 | 0 | 2 | |
| Treatment of toxicity* | .14 | |||
| Tocilizumab | 10 | 5 | 31 | |
| Steroids | 6 | 2 | 18 | |
| Other | 2 | 0 | 4 | |
| Response | .35 | |||
| No CR | 4 | 4 | 15 | |
| CR (% MRD negative) | 35 (97%) | 10 (90%) | 111 (95%) | |
| Died before day 28 | 1 | 1 | 3 | |
| Relapsed post-CAR | 15 | 6 | 45 | .91 |
| Site of relapse | ||||
| CNS | 5 | 0 | 0 | |
| CNS + BM | 2 | 0 | 2 | |
| BM only | 8 | 2 | 37 | |
| BM + other EM | 0 | 2 | 4 | |
| Other EM disease | 0 | 2 | 2 | |
| CD19-negative relapse | 5/15 | 1/6 | 20/38 | .025 |
| Unknown | 2 | 3 | 3 | |
| Time from infusion to relapse, d | 101 (30-577) | 95 (30-245) | 90 (28-645) | .87 |
| SCT post CAR | 6 | 1 | 38 | .06 |
| Rationale for SCT post-CAR | .52 | |||
| Preemptive | 1 | 0 | 5 | |
| Loss of BCA | 0 | 0 | 11 | |
| Refractory/relapse | 4 | 1 | 16 | |
| MLL rearranged | 1 | 0 | 2 | |
| Alive/dead | 31/9 | 9/6 | 93/36 | .40 |
| Cause of death | .87 | |||
| Relapse | 7 | 5 | 26 | |
| Infection | 2 | 0 | 4 | |
| CRS | 0 | 0 | 1 | |
| Neurotoxicity | 0 | 0 | 1 | |
| Transplant related | 0 | 1 | 3 | |
| Cardiac related | 0 | 0 | 1 | |
| Loss of BCA | 10 | 8 | 39 | .14 |
| Time from infusion to loss of BCA, d | 174 (36-266) | 84.5 (29-396) | 93 (28-545) | .23 |
ICANS, immune effector cell-associated neurotoxicity syndrome; MLL, mixed-lineage leukemia; MRD, minimal residual disease.
Toxicity refers to either CRS or ICANS.